[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"countryName\":\"United States\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":662},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,25914,0,25,[9,40,70,99,125,156,179,199,226,248,282,312,337,359,393,422,446,474,498,521,541,561,588,615,638],{"id":10,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":18,"phases":4,"briefSummary":19,"conditions":20,"keywords":23,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":36,"locationsCount":39},"100053613",false,"NCT06915766","Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis","Intermediate-Size Patient Population Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis","Key Inclusion Criteria:\n\n* Able to provide written informed consent and commit to EAP assessments.\n* ≥18 years of age.\n* Able to swallow tablets.\n* Diagnosed with ASM, SM-AHN, MCL, BMM, ISM, or SSM according to the 2022 WHO Classification for SM\n* Not receiving adequate disease control on current therapy(ies).\n* Have clinically acceptable laboratory screening results.\n\nExclusion Criteria:\n\n* Patients who are eligible for and\u002For enrolled in an on-going bezuclastinib clinical trial.\n* Patients who discontinued investigational use of bezuclastinib in previous clinical trials due to toxicity or withdrawal of consent.\n* Pregnant or currently breastfeeding.\n* Prior or ongoing clinically significant illness or medical or physical condition\n\nOther protocol-defined criteria apply.","ALL","18 Years","EXPANDED_ACCESS","The purpose of this expanded access program (EAP) protocol is to provide investigational bezuclastinib to patients with a diagnosis of nonadvanced systemic mastocytosis (NonAdvSM) or advanced systemic mastocytosis (AdvSM) who have received and failed or been intolerant to at least one standard approved therapy and\u002For have no comparable or satisfactory alternative therapy options.",[21,22],"Systemic Mastocytoses, Indolent","Systemic Mastocytoses, Aggressive",[24,25,26,27,28,29,30],"Systemic Mastocytosis","NonAdvanced Systemic Mastocytosis","Advanced Systemic Mastocytosis","Systemic Mastocytosis with an associated hematologic neoplasm (SM-AHN)","Aggressive Systemic Mastocytosis (ASM)","Mast Cell Leukemia (MCL)","Bone Marrow Mastocytosis (BMM)","AVAILABLE","2026-07-10",{"date":34,"type":35},"2026-07-13","ACTUAL",{"name":37,"class":38},"Cogent Biosciences, Inc.","INDUSTRY",23,{"id":41,"slug":4,"hasResults":11,"nctId":42,"briefTitle":43,"officialTitle":44,"acronym":4,"eligibilityCriteria":45,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":46,"targetDuration":4,"studyType":49,"phases":50,"briefSummary":53,"conditions":54,"keywords":56,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":62,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":69},"100053587","NCT05874414","Combination of GNS561 and Trametinib in Patients With Advanced KRAS Mutated Cholangiocarcinoma","Phase 1b\u002F2a Study of GNS561 in Combination With Trametinib in Advanced KRAS Mutated Cholangiocarcinoma","Inclusion criteria:\n\n1. Histologically confirmed intrahepatic CCA with a documented KRAS mutation.\n2. Patients greater than or equal to 18 years of age.\n3. Patients must have disease progression that is not amenable to potentially curative treatment.\n4. Patients must have received one or two lines of chemotherapy.\n5. Patients must have at least one measurable disease by RECIST v1.1.\n6. Performance status (ECOG) 0-1.\n7. Adequate organ baseline function defined as follows: absolute neutrophil count ≥1000 cells\u002FμL, platelet count ≥75,000 cells\u002FμL, hemoglobin ≥9 g\u002FdL, aspartate aminotransferase or alanine aminotransferase less than or equal to 3 × upper limit of normal, estimated glomerular filtration rate ≥60 mL\u002Fmin, corrected QT interval by Fridericia's (QTcF) interval ≤470 msec.\n8. Women of childbearing potential must present with a negative serum pregnancy test and agree to use adequate contraception during the study and until 6 months after the end of treatment. Male patients with women partners of childbearing potential must agree with the contraception procedures of the study protocol.\n9. Patients must be able to understand and be willing to comply with the requirements of the study protocol.\n10. Patients participate voluntarily and sign informed consent form(s).\n\nExclusion criteria:\n\n1. Previous treatment with a MEK inhibitor or autophagy inhibitor.\n2. Previous treatment with three or more lines of prior chemotherapy.\n3. Extrahepatic CCA with recent (within 6 weeks) placement of a stent or episodes of unstable biliary stents (manifest as obstruction, migration of the stent, infevtion or mechanical failure of the stent) within 6 weeks according to investigator's judgement.\n4. Current evidence of uncontrolled, significant intercurrent illness including, but not limited to, the following conditions:\n\n   1. Cardiovascular disorders: congestive heart failure New York Heart Association ≥ class 2 or left ventricular ejection fraction (LVEF) \\\u003C50%, arrythmias or cardiac conduction abnormalities. Uncontrolled arterial hypertension or inadequately controlled arterial hypertension, at the discretion of the investigator, based on an average of = \\>3 BP readings over = \\>2 sessions.\n   2. Patients who have retinal condition (retinal tear, exudate, hemorrhage) or history of retinal vein occlusion or central serous retinopathy or retinal pigment epithelial detachment.\n   3. History of interstitial lung disease or pneumonitis.\n   4. Patients who have clinically significant pleural effusion or ascites.\n   5. Patients who have neurological condition (e.g., tremor, ataxia, hypotension, confusion), history of seizures or active central nervous system metastases.\n   6. Impairment of gastrointestinal function or gastrointestinal disease (e.g., diarrhea, active ulcer disease, history of gastrointestinal perforation\u002Fhemorrhage, malabsorption or other conditions that under the judgment of the principal investigator (PI) may impair absorption of study drugs).\n   7. Patients who are taking antineoplastic drugs for concomitant cancer or history of malignancy other than CCA within 5 years prior to screening, with the exception of malignancies with a negligible risk of metastasis or death (e.g., 5-year OS rate \\> 90%) such as adequately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, localized prostate cancer, ductal carcinoma in situ, or Stage I uterine cancer.\n   8. Any other condition that would, in the Investigator s judgment, contraindicate the patients' participation in the clinical study due to safety concerns or compliance with clinical study procedures (e.g., infection, unable to swallow medication, social\u002Fpsychological issues, etc).\n5. Known active viral hepatitis, including HBV and HCV.\n6. Patients with known allergic reaction to quinoline derivatives (e.g., quinine, chloroquine, mefloquine) and\u002For hypersensitivity to study drugs.\n7. Patients who have not recovered for certain AEs due to previous lines of therpay.\n8. Female patients who are pregnant or lactating at the time of enrollment.",{"count":47,"type":48},98,"ESTIMATED","INTERVENTIONAL",[51,52],"PHASE1","PHASE2","This is an open-label, multicenter Phase 1b\u002F2a study to evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of GNS561 in combination with trametinib in Advanced KRAS Mutated Cholangiocarcinoma after failure of standard-of-care first line therapy",[55],"Cholangiocarcinoma",[57,55,58,59,60],"GNS561","Trametinib","Phase1b\u002F2a","Bile Duct cancer","RECRUITING",{"date":34,"type":35},{"date":64,"type":35},"2023-08-21",{"date":66,"type":48},"2028-06",{"name":68,"class":38},"Genfit",11,{"id":71,"slug":4,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":75,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":77,"enrollmentInfo":78,"targetDuration":4,"studyType":49,"phases":80,"briefSummary":81,"conditions":82,"keywords":85,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":92,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":39},"100053451","NCT07284667","ACP-211 Monotherapy for Major Depressive Disorder With Inadequate Antidepressant Response","A Double-Blind, Placebo-Controlled, Parallel Group, Efficacy and Safety Study of ACP-211 Monotherapy in Adults With Major Depressive Disorder and Inadequate Response to Antidepressant Treatment","NORLIGHT","Inclusion Criteria:\n\n* Adults ≥18 and ≤65 years of age\n* Provides written informed consent\n* Clinical diagnosis of MDD\n* History of inadequate response to at least two antidepressants, with at least one inadequate response documented during the current episode\n* Currently treated with an approved antidepressant at a stable dose prior to Screening\n* MADRS total score ≥28, CGI-S score ≥4 , and QIDS-SR16 score ≥16 at Screening and Baseline\n* Females of childbearing potential must have a negative pregnancy test and agree to use acceptable contraception; males must agree to use barrier protection and refrain from sperm donation\n\nExclusion Criteria:\n\n* Current diagnosis of certain personality disorders or persistent depressive disorder\n* Recent substance use disorders, excluding caffeine or nicotine\n* Active suicidal risk or recent suicidal attempt\n* History of schizophrenia, psychotic disorders, bipolar disorder, or MDD with psychotic features\n* Current treatment requirement for PTSD, acute stress disorder, panic disorder, or OCD\n* History of neuroleptic malignant syndrome, serotonin syndrome, or epilepsy (except single febrile seizure in infancy)\n* Documented non-response to ADT, including ketamine or esketamine\n* Allergy or sensitivity to ketamine or esketamine\n* Significant cardiovascular disease\n* Positive history of hepatitis B, hepatitis C, or HIV infection\n* Unstable diabetes or uncontrolled medical conditions\n* Positive urine drug test for an illicit drug or cannabis\n* Received neuromodulation therapies (ECT,TMS, VNS, DBS) in the current depressive episode\n* Recent initiation or change in psychotherapy Additional inclusion\u002Fexclusion criteria apply. Participants will be evaluated at Screening to ensure that all criteria for study participation are met.","65 Years",{"count":79,"type":48},153,[52],"The goal of this clinical trial is to learn if ACP-211 can help treat adults with major depressive disorder (MDD) who have not improved with antidepressant therapy (ADT), including those with treatment resistant depression (TRD).\n\nThe main questions the study aims to answer are:\n\n* Does ACP-211 work better than a placebo (a look-alike capsule with no medicine) to reduce symptoms of depression?\n* What adverse events do participants have when taking ACP-211?",[83,84],"Major Depressive Disorder (MDD)","Depressive Disorder, Treatment-Resistant",[83,84,86,87,88,89,90,91],"Antidepressive Agents","Randomized Controlled Trial","Double-Blind Method","Clinical Trial, Phase II","Psychiatric Status Rating Scales","Ketamine",{"date":34,"type":35},{"date":94,"type":35},"2025-11-14",{"date":96,"type":48},"2027-09",{"name":98,"class":38},"ACADIA Pharmaceuticals Inc.",{"id":100,"slug":4,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":102,"acronym":4,"eligibilityCriteria":103,"healthyVolunteers":11,"sex":16,"minAge":104,"maxAge":105,"enrollmentInfo":106,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":109,"conditions":110,"keywords":112,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":118,"startDateStruct":119,"completionDateStruct":4,"leadSponsor":121,"locationsCount":124},"100054269","NCT00028080","Evaluation, Treatment, and Follow-up of Patients With Lyme Disease","* INCLUSION CRITERIA:\n* Clinical diagnosis of active Lyme disease at the time of the initial NIH evaluation based on the CDC case definition. Study physician will review history to confirm probable cases.\n* Subjects must maintain a private physician for non-protocol related medical complaints and for emergency medical treatment required for these or other of their disorders.\n\nEXCLUSION CRITERIA:\n\n* Post treatment Lyme disease syndrome.\n* Unacceptably poor compliance, which, in the opinion of the investigator, would interfere with one's ability to study or provide quality medical care for the patient.\n\nELIGIBILITY CRITERIA TO BE ASKED TO UNDERGO APHERESIS INCLUDE:\n\n* Age 18 years or above\n* Weight greater than 110 pounds\n* No known heart, lung, kidney disease, or bleeding disorders\n* Negative HIV, HCV and HBsAg serologies\n* Female subjects should not be pregnant or nursing\n* Adequate peripheral venous access\n\nPatients will have a CBC performed up to 2 weeks before the procedure. In order to be able to undergo the procedure, patient must fulfill all of the below:\n\n* Hemoglobin greater than 11 g\u002FdL for males and greater than 10 g\u002FdL for females\n* Platelets greater than 150 k\u002Fmm(3)\n* WBC greater than 3.5 x 10(3) uL\n* MCV above 80\n\nWomen who are able to conceive children must have a negative pregnancy test within 2 weeks before the procedure.","2 Years","99 Years",{"count":107,"type":48},500,"OBSERVATIONAL","This study is designed to establish a population of patients with Lyme disease for evaluation, treatment and follow-up to learn more about the infection.\n\nPatients with active Lyme disease may be eligible for this study.\n\nParticipants will have a medical history and physical examination and diagnostic evaluations as appropriate to their individual condition. Laboratory tests may include routing blood and urine tests, X-rays, or other imaging studies, body fluid or tissue cultures, skin biopsy and tests for allergic or immune responses. Treatment will include only medications approved by the Food and Drug Administration, given according to accepted dose schedules and ways of taking the medicines. All diagnostic tests and treatments will be according to standard medical practice for the disease. No experimental procedures will be offered under this protocol.\n\nPatients will be followed as needed for evaluation and treatment of their condition. In general, they will be asked to return at the end of therapy, then 3, 6 and 12 months later, and then every 6 to 12 months. More frequent visits may be required or less frequent visits may be allowed, depending on the individual s condition.\n\nParticipants may be asked to undergo the following additional procedures for research purposes:\n\n* Extra blood draws to study Lyme disease and other inflammatory conditions. Blood will be drawn from arm veins. The total amount drawn during any 6-week period will not exceed 450 cc (30 tablespoons) for adults and 7 cc (1\u002F2 tablespoon) per kilogram (2.2 pounds) of body weight for children under 18 years of age.\n* Leukapheresis to collect large numbers of white blood cells. Whole blood is collected through a needle in an arm vein, similar to donating blood. The blood circulates through a machine that separates it into its components, and the white cells are removed. The rest of the blood is returned to the body through the same needle. Only adults 18 years of age and older will be asked to undergo leukapheresis.",[111],"Lyme Disease",[113,111,114,115,116,117],"Borrelia Burgdorferi","Infection","Therapy","Natural History","Lyme",{"date":34,"type":35},{"date":120,"type":35},"2002-01-16",{"name":122,"class":123},"National Institute of Allergy and Infectious Diseases (NIAID)","NIH",1,{"id":126,"slug":4,"hasResults":11,"nctId":127,"briefTitle":128,"officialTitle":129,"acronym":4,"eligibilityCriteria":130,"healthyVolunteers":11,"sex":16,"minAge":131,"maxAge":132,"enrollmentInfo":133,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":135,"conditions":136,"keywords":138,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":148,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":155},"100053447","NCT07330258","A US Study That Observes How Parkinson's Disease Changes Over Time in Patients Who Still Have Movement Symptoms Despite Taking Parkinson's Medications","A Natural History Study of Treated Parkinson's Disease Patients Experiencing Motor Complications","Inclusion Criteria for Patient:\n\n* Individual of any sex ≥45 to ≤75 years of age at informed consent (at least 30% ≤60 years of age).\n* Diagnosis of clinically established Parkinson's disease (PD) as defined by the Movement Disorder Society (MDS) Clinical Diagnostic Criteria for PD ≥4 and \\\u003C12 years from time of PD diagnosis at informed consent.\n* Modified H\\&Y stage II-III in the practically defined OFF-medication state (≥12 hours from last dose of antiparkinsonian medications).\n* Score of ≥30 on MDS-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part III in the OFF-medication state.\n* Presence of motor fluctuations with ≥1 hour of absolute time in the OFF state per day as assessed by clinician\u002Fpatient at screening.\n* Receiving stable antiparkinsonian medication regimen for ≥4 weeks prior to screening with a levodopa daily dose ≥300 mg or a dosing frequency of ≥3 times per day.\n* Responsiveness to levodopa as determined by change in the following measures from the practically defined OFF state to ON state after taking typical first-daily dose of PD-medications: i. any degree of improvement (≥0.5 point) in modified H\\&Y stage OR. ii. ≥30% improvement in MDS-UPDRS part III score.\n* Montreal Cognitive Assessment (MoCA) score of ≥24.\n* Agree to participate and provide signed informed consent.\n\nExclusion Criteria for Patient:\n\n* Known history or presence of conditions that may provide an alternative to a PD diagnosis including but not limited to: multiple system atrophy, progressive supranuclear palsy, striatonigral degeneration, corticobasal syndrome\u002Fdegeneration, vascular Parkinsonism, drug-induced Parkinsonism, essential tremor, diffuse Lewy body disease, Lewy body dementia, Huntington's disease, Wilson's disease, Fahr's disease, Alzheimer's disease, cerebrovascular disease, brain tumor, trauma, and infection.\n* Known history or presence of significant vascular and\u002For cardiovascular disease limited to: stroke, transient ischemic attacks, poorly controlled hypertension, poorly controlled diabetes, unstable angina pectoris, or unstable myocardial infarction.\n* Known history or presence of significant psychosis or impulse control disorder, or untreated or sub optimally treated depression.\n* Known history or presence of human immunodeficiency virus, hepatitis B virus, hepatitis C virus, syphilis, or tuberculosis.\n* Current or previously active malignant disease within the past 5 years, except definitively treated cutaneous squamous cell carcinoma, basal cell carcinoma, or in situ uterine cervical carcinoma.\n* Currently pregnant, nursing, lactating, breastfeeding, or plan to be during study duration.\n* Known history or current use of percutaneous levodopa\u002Fcarbidopa intestinal gel, subcutaneous levodopa, or apomorphine pump.\n* Prior history of brain surgery, including but not limited to: deep brain stimulation (DBS), pallidotomy, focused ultrasound thalamotomy, or other experimental neurosurgical procedure.\n* Known history or current participation in cell or gene therapy procedures.\n* Current participation in any interventional clinical trial.\n\nInclusion Criteria for Care Partner:\n\n* ≥18 years of age at informed consent.\n* Identified by the PD patient as their primary care partner.\n* Agree to participate and the ability to provide signed informed consent independently, without the need for a legal representative.\n\nExclusion Criteria for Care Partner:\n\n* Not applicable.","45 Years","75 Years",{"count":134,"type":48},300,"This is an observational study in which data are collected and studied from Parkinson's disease patients who have movement symptoms despite taking standard Parkinson's medications. In observational studies, observations are made without any changes to the participant's healthcare or treatment plan. No investigational product will be administered in this study, as participants will be treated with the standard of care that medical experts currently consider most appropriate.\n\nParkinson's disease (PD) is a condition that affects the brain and causes problems with movement and other body functions. The symptoms of Parkinson's disease can worsen over time. People with Parkinson's disease may experience shaking (tremor), slow movements, stiff muscles, trouble walking, and problems with balance. They can also have other symptoms, such as difficulty thinking clearly, changes in mood, or difficulty sleeping. Parkinson's disease mostly affects older adults, but it can happen to younger people too. There is no cure, but treatments can help manage the symptoms and improve quality of life.\n\nWhile doctors and researchers know that Parkinson's disease affects people in different ways and can worsen over time, there are still many things they don't fully understand-especially for people who experience movement symptoms despite taking their usual Parkinson's medicines. Earlier studies did not follow these patients long enough or collect all the important information needed. This study is being done to fill those gaps.\n\nThe main purpose of this study is to better understand how Parkinson's disease changes over time in patients who experience movement symptoms while taking standard oral Parkinson's medications, what challenges patients and their care partners face, and how their treatments are working in real life. To do this, researchers will collect data on:\n\n* Sociodemographics (e.g. age, gender, race\u002Fethnicity, insurance provider).\n* Medical history and vital signs (e.g. comorbidities, family history of Parkinson's, height, weight, blood pressure).\n* Medications and treatments (e.g. Parkinson's and non-Parkinson's medications and other treatments, rehabilitation therapy sessions, use of mobility assistance devices).\n* Movement symptoms (e.g. tremor, slow movement, balance).\n* Non-movement symptoms (e.g. cognition, mood, sleep, activities of daily living).\n* Molecular data (e.g. genetics, α-synuclein).\n* Burden of care (e.g. economic cost).\n\nData will come from questionnaires or rating scales conducted by the doctor with the patient during study visits, diaries and logs completed by the patient, medical records, health insurance claims records, blood samples and skin biopsies, a digital device that records movement\u002Fnon-movement symptoms, and questionnaires completed by the care partner.\n\nData will be collected from December 2025 to December 2032. Each participant may be followed for up to 5 years.",[137],"Parkinson's Disease",[139,140,141,142,143,144,145,146,147],"Natural history study","Parkinson's disease","PD Motor (Hauser) Diary","MDS-UPDRS","Electronic health\u002Fmedical records","Administrative claims data","Biological samples","Digital health technology","Care partner",{"date":34,"type":35},{"date":150,"type":48},"2026-07-22",{"date":152,"type":48},"2033-06-01",{"name":154,"class":38},"Bayer",26,{"id":157,"slug":4,"hasResults":11,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":4,"eligibilityCriteria":161,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":162,"targetDuration":4,"studyType":49,"phases":164,"briefSummary":165,"conditions":166,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":171,"startDateStruct":172,"completionDateStruct":174,"leadSponsor":176,"locationsCount":178},"100054268","NCT06126276","Testing the Use of Neratinib or the Combination of Neratinib and Palbociclib Targeted Treatment for HER2+ Solid Tumors (A ComboMATCH Treatment Trial)","A Randomized Trial of Neratinib, A Pan-ERBB Inhibitor, Alone or in Combination With Palbociclib, a CDK4\u002F6 Inhibitor, in Patients With HER2+ Gynecologic Cancers and Other Solid Tumors: A ComboMATCH Treatment Trial","Inclusion Criteria:\n\n* Patient must have enrolled onto EAY191 and must have been given a treatment assignment to ComboMATCH to EAY191-N5 based on the presence of an actionable mutation as defined in EAY191\n* Patients must have a HER2 amplified solid tumor except breast cancer.\n\n  * If IHC is 0 or 1+, patient (pt) is NOT ELIGIBLE regardless of in situ hybridization (ISH)\u002FFISH or next generation sequencing (NGS) status\n  * If IHC is 3+, pt IS ELIGIBLE regardless of ISH\u002FFISH or NGS status\n  * If IHC is 2+, ISH\u002FFISH OR NGS must be positive for the patient to be ELIGIBLE. Otherwise, pt is NOT ELIGIBLE\n  * If IHC is unknown and…\n\n    * ISH\u002FFISH is positive, independent of NGS results, the patient IS ELIGIBLE\n    * ISH\u002FFISH is negative and NGS positive with ≥ 7 copies, the patient IS ELIGIBLE\n* Patients must have recurrent or persistent disease\n* No known evidence of RB1 loss or deletion including copy number loss or deleterious mutation\n* Patients must have disease that can be safely biopsied and agree to a pre-treatment biopsy or, if disease cannot be safely biopsied, have archival tissue available from within 12 months prior to the date of registration on the ComboMATCH Registration Trial (EAY191)\n* Patients must have measurable disease based on RECIST 1.1. A second measurable lesion outside of the biopsiable lesion is required\n* Patients with treated brain metastases are eligible if follow up brain imaging after central nervous system (CNS) directed therapy shows no evidence of progression for 3 months or more and patient is not on steroids and is asymptomatic\n* No known leptomeningeal disease\n* Patients may have received up to 5 prior lines of systemic therapy\n* Prior therapy with trastuzumab or pertuzumab, either alone or in combination, antibody drug conjugates (ADC) such as DS8201a or T-DM1 is allowed\n* No prior therapy with HER2 targeting tyrosine kinase inhibitors (TKI) such as neratinib or tucatinib\n* No prior therapy with CDK4\u002F6 inhibition\n* No cancer directed therapy within 3 weeks prior to registration. For oral therapy, the washout can be reduced to greater than or equal to 5 half lives of the drug. No HER2 targeting ADCs within 30 days prior to registration\n* Age ≥ 18\n* Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤ 2\n* Not pregnant and not nursing\n* Absolute neutrophil count (ANC) ≥ 1,500 cells\u002Fmm\\^3\n* Platelets ≥ 100,000 cells\u002Fmm\\^3\n* Hemoglobin ≥ 9 g\u002Fdl (Note: The use of transfusion or other intervention to achieve hemoglobin (Hgb) ≥ 9 g\u002Fdl is acceptable)\n* Creatinine clearance (CrCL) of ≥ 30 mL\u002Fmin by the Cockcroft-Gault formula\n* Total bilirubin level ≤ 1.5 x institutional upper limit of normal (ULN) (patients with known Gilbert's disease who have bilirubin level ≤ 3 x institutional ULN may be enrolled)\n* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x institutional upper limit of normal (ULN)\n* Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class 2B or better\n* No active infection requiring parenteral antibiotics\n* No current evidence of intra-abdominal abscess, abdominal\u002Fpelvic fistula (not diverted), gastrointestinal perforation, gastrointestinal (GI) obstruction, and\u002For need for drainage nasogastric or gastrostomy tube\n* No current evidence of malabsorption or chronic diarrhea or any other significant gastro-intestinal disease (e.g gastrectomy, ileal bypass, Crohn's disease, gastroparesis), associated with moderate to severe diarrhea (grade 2 or more) or inability to tolerate oral therapy\n* No lung disease causing dyspnea at rest\n* No interstitial lung disease with ongoing signs and symptoms at the time of registration\n* No history of allergic reaction to the study agents, compound of similar chemical or biologic composition of the study agents or any of their excipients",{"count":163,"type":48},70,[52],"This phase II ComboMATCH treatment trial compares the effect of neratinib to the combination of neratinib and palbociclib in treating patients with HER2 positive solid tumors. Neratinib and palbociclib are in a class of medications called kinase inhibitors. They work by blocking the action of an abnormal protein that signals cancer cells to multiply. This helps slow or stop the spread of tumor cells. Giving neratinib and palbociclib in combination may shrink or stabilize cancers that over-express a specific biomarker called HER2.",[167,168,169,170],"Malignant Female Reproductive System Neoplasm","Malignant Solid Neoplasm","Recurrent Malignant Female Reproductive System Neoplasm","Recurrent Malignant Solid Neoplasm",{"date":34,"type":35},{"date":173,"type":35},"2024-05-07",{"date":175,"type":48},"2027-02-20",{"name":177,"class":123},"National Cancer Institute (NCI)",188,{"id":180,"slug":4,"hasResults":11,"nctId":181,"briefTitle":182,"officialTitle":183,"acronym":4,"eligibilityCriteria":184,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":185,"targetDuration":4,"studyType":49,"phases":187,"briefSummary":188,"conditions":189,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":191,"startDateStruct":192,"completionDateStruct":194,"leadSponsor":196,"locationsCount":198},"100053422","NCT07405801","A Phase II Study Evaluating the Efficacy and Safety of Inavolisib Plus Ribociclib Plus Fulvestrant Versus Placebo Plus Ribociclib Plus Fulvestrant in Participants With Advanced Breast Cancer","A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Inavolisib Plus Ribociclib Plus Fulvestrant Versus Placebo Plus Ribociclib Plus Fulvestrant in Patients With Endocrine- Resistant Hormone-Receptor-Positive, HER2-Negative Advanced Breast Cancer With Chromosome 8P Loss and Without a PIK3CA Mutation","Inclusion Criteria:\n\n* Women or men with histologically or cytologically confirmed carcinoma of the breast that is locally advanced or metastatic and is not amenable to surgical or radiation therapy with curative intent\n* Documented estrogen receptor (ER)-positive and\u002For progesterone receptor (PR)-positive tumor according to American Society of Clinical Oncology\u002FCollege of American Pathologists (ASCO\u002FCAP) guidelines, defined as \\>=1% of tumor cells stained positive based on the most recent tumor biopsy and assessed locally (Allison et al. 2020)\n* Participants must not have received any prior systemic therapy for locally advanced unresectable or metastatic breast cancer (mBC) and must have progressed during adjuvant endocrine-based treatment or within 12 months after completing adjuvant endocrine-based therapy with an aromatase inhibitor or tamoxifen\n* Confirmed biomarker eligibility as documented through central laboratory testing of a tumor tissue sample documenting both the lack of a phosphatidylinositol-4,5-biphosphate 3-kinase catalytic subunit alpha gene (PIK3CA) mutation and the presence of heterozygous loss of chromosome 8p (i.e., PIK3CAnmd and chr8p loss)\n* Measurable disease per Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1)\n\nExclusion Criteria:\n\n* Metaplastic breast cancer\n* Radiotherapy within 2 weeks before randomization\n* Appropriate for treatment with cytotoxic chemotherapy at time of entry into the study, as per national or local treatment guidelines (e.g., participants with visceral crisis)\n* Type 2 diabetes requiring ongoing systemic treatment at the time of study entry; or any history of Type 1 diabetes\n* Known and untreated, or active Central nervous system (CNS) metastases (progressing or requiring anticonvulsants or corticosteroids for symptomatic control). Participants with a history of treated CNS metastases are eligible\n* Any history of leptomeningeal disease or carcinomatous meningitis",{"count":186,"type":48},80,[52],"A study to evaluate the efficacy and safety of triplet combination of inavolisib plus ribociclib and fulvestrant versus placebo plus ribociclib and fulvestrant in the first-line setting in participants with endocrine-therapy-resistant hormone receptor (HR)-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC).",[190],"Breast Cancer",{"date":34,"type":35},{"date":193,"type":35},"2026-04-07",{"date":195,"type":48},"2030-02-26",{"name":197,"class":38},"Hoffmann-La Roche",49,{"id":200,"slug":4,"hasResults":11,"nctId":201,"briefTitle":202,"officialTitle":203,"acronym":4,"eligibilityCriteria":204,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":205,"targetDuration":4,"studyType":49,"phases":207,"briefSummary":208,"conditions":209,"keywords":214,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":218,"startDateStruct":219,"completionDateStruct":221,"leadSponsor":223,"locationsCount":225},"100054248","NCT05607095","A Study of LN-144 or LN-145 in People With Advanced Uveal Melanoma, Undifferentiated Pleomorphic Sarcoma, Dedifferentiated Liposarcoma, or Angiosarcoma","A Pilot Trial of Autologous Tumor Infiltrating Lymphocytes (LN-144 or LN-145) for Patients With Advanced Uveal Melanoma, Undifferentiated Pleomorphic Sarcoma, Dedifferentiated Liposarcoma, or Angiosarcoma","Inclusion Criteria:\n\n* Cohort 1: Must have a confirmed diagnosis of metastatic Uveal Melanoma.\n\n  o Patients will be eligible regardless of the number of prior systemic therapies received.\n* Cohort 2: Must have a confirmed diagnosis of unresectable or metastatic undifferentiated pleomorphic sarcoma (UPS) or dedifferentiated liposarcoma (DDLPS) that is refractory to at least 1 prior line of systemic therapy\n\n  * Unresectable disease will be defined by an expert sarcoma surgical oncologist as either (a) low likelihood of obtaining an R0 resection or (b) unacceptable morbidity from a surgical procedure\n  * Prior systemic therapy in the neoadjuvant or adjuvant setting will count has prior systemic therapy\n  * Patients who refuse standard of care chemotherapy will be eligible\n* Cohort 3: Must have a confirmed diagnosis of unresectable or metastatic angiosarcoma that is refractory to at least 1 prior line of systemic therapy\n\n  * Unresectable disease will be defined by an expert sarcoma surgical oncologist as either (a) low likelihood of obtaining an R0 resection or (b) unacceptable morbidity from a surgical procedure\n  * Prior systemic therapy in the neoadjuvant or adjuvant setting will count has prior systemic therapy\n  * Patients who refuse standard of care chemotherapy will be eligible\n* One (1) lesion at least 1.5cm in size (solitary or aggregate) available for TIL harvesting that has not undergone prior embolization or RT in prior 3 months unless subsequent growth is demonstrated (at least 0.5cm).\n* Patients must be ≥ 18 years of age at the time of consent.\n* Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1\n* Patients must have an estimated life expectancy of ≥ 6 months in the opinion of the Investigator.\n* Patients must have the following hematologic parameters:\n\n  * Absolute neutrophil count (ANC) ≥ 1000\u002Fmm3\n  * Hemoglobin (Hb) ≥ 9.0 g\u002FdL\n  * Platelet ≥ 100,000\u002Fmm\\^3 Note: Transfusions or growth factors are not allowed 28 days prior to signing the ICF and continuing through the Screening Period\n* Patients must have adequate organ function:\n\n  * Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal (≤ 3 × ULN); patients with liver metastasis ≤ 5 × ULN\n  * Estimated creatinine clearance (eCrCl) ≥ 40 mL\u002Fmin using the Cockcroft-Gault formula at Screening\n  * Total bilirubin ≤ 2 mg\u002FdL\n  * Patients with Gilbert's syndrome must have a total bilirubin ≤ 3 mg\u002FdL\n* Patients must be seronegative for the following:\n\n  * Human immunodeficiency virus (HIV)-1 or HIV-2 antibodies\n  * Hepatitis B antigen (HBsAg), hepatitis B core antibody (anti- HBc), or hepatitis C antibody (HCV Ab). Patients with acute or chronic hepatitis infections may be enrolled if the viral load by polymerase chain reaction (PCR) is undetectable with\u002Fwithout active treatment.\n  * Syphilis (Rapid Plasma Reagin \\[RPR\\] test or venereal disease research laboratory \\[VDRL\\] test)\n  * Cytomegalovirus (CMV) IgM antibody titer or PCR assay; and Epstein-Barr virus (EBV) IgM or PCR assay indicating active infection\n  * Herpes simplex virus (HSV)-1 and HSV-2 IgM serology or PCR assay\n* Patients who are HSV immunoglobulin M (IgM) or PCR assay positive will need to receive appropriate treatment and become IgM or PCR assay negative prior to starting the NMA-LD pre-conditioning regimen\n* Anyone with prior COVID-19 infection must be asymptomatic for \\>30 days prior to NMA-LD.\n* Patients must have a washout period from prior anticancer therapy(ies) of a minimum duration, as detailed below prior to the first study treatment (ie, start of NMA-LD):\n\n  * Targeted therapy: prior targeted therapy with an EGFR, MEK, BRAF, ALK, ROS1, or other-targeted agents (eg, erlotinib, afatinib, dacomitinib, osimertinib, crizotinib, ceritinib, lorlatinib) is allowed provided the washout is a minimum of 14 days or 5 half-lives (whichever is longer) prior to the start of treatment\n  * Chemotherapy: minimum of 21 days prior to the start of treatment\n  * Immunotherapy: checkpoint-targeted therapy with an anti PD-1\u002Fanti PD-L1, other monoclonal antibodies, or vaccines are allowed, provided the washout is a minimum of 21 days prior to the start of study treatment\n* Palliative radiation therapy is permitted so long as it does not involve lesions being selected for TIL, or as target or non-target lesions. Washout is not required if all related toxicities have resolved to ≤ Grade 1 as per CTCAE v 5.0.\n* Patients must have recovered from all prior anti-cancer therapy-related adverse events (AEs) to ≤ Grade 1 (per Common Terminology Criteria for Adverse Events \\[CTCAE\\] v 5.0), except for alopecia or vitiligo, prior to enrollment.\n\n  * Patients with documented ≥ Grade 2 diarrhea or colitis as a result of previous treatment with immune checkpoint inhibitor(s) must have been asymptomatic for at least 6 months and\u002For had a normal colonoscopy post-immune checkpoint inhibitortreatment, by visual assessment, prior to tumor resection.\n  * Patients with immunotherapy-related endocrinopathies (e.g. hypothyroidism) stable for at least 6 weeks and controlled with hormonal replacement are allowed.\n* Previous surgical procedure(s) is\u002Fare permitted provided that wound healing has occurred, all complications have resolved, and at least 14 days have elapsed (for major operative procedures) prior to the tumor resection.\n* Patients of childbearing potential (or female partners of male participants) must be willing to take the appropriate precaution to avoid pregnancy or fathering a child for the duration of the study and practice an approved, highly effective method of birth control during treatment and for 12 months after their last dose of IL-2. Approved methods of birth control are as follows:\n\n  * Combined (estrogen and progesterone containing) hormonal birth control associated with inhibition of ovulation: oral, intravaginal, transdermal\n  * Progesterone-only hormonal birth control associated with inhibition of ovulation: oral, injectable, implantable\n  * Intrauterine device (IUD)\n  * Intrauterine hormone-releasing system (IUS)\n  * Bilateral tubal occlusion\n  * Vasectomized partner\n  * True sexual abstinence when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (eg, calendar ovulation, symptothermal, post-ovulation methods) is not acceptable\n* Patients (or legally authorized representative) must have the ability to understand the requirements of the study, have provided written informed consent as evidenced by signature on an ICF approved by an Institutional Review Board\u002FIndependent Ethics Committee (IRB\u002FIEC), and agree to abide by the study restrictions and return to the site for the required assessments, including the OS Follow-up Period.\n\nExclusion Criteria:\n\n* Patients who have received an organ allograft or prior cell transfer therapy that included a non-myeloablative or myeloablative chemotherapy regimen.\n* Patients who have a history of hypersensitivity to any component or excipient of LN-144\u002FLN-145 or other study drugs:\n\n  * NMA-LD preconditioning regimen (cyclophosphamide, mesna, and fludarabine)\n  * Proleukin®, aldesleukin, IL-2\n  * Antibiotics (ABX) of the aminoglycoside group (i.e., streptomycin, gentamicin); except those who are skin-test negative for gentamicin hypersensitivity\n  * Any component of the LN-144\u002FLN-145 infusion product formulation including dimethyl sulfoxide (DMSO), human serum albumin (HSA), IL-2, and dextran-40.\n* Patients with symptomatic brain metastases (of any size and any number).\n\n  o Patients with definitively treated brain metastases may be considered for enrollment, if, prior to tumor resection for TIL, the patient is clinically stable for ≥ 14 days, there are no symptomatic brain lesions, and that the patient does not require ongoing corticosteroid treatment.\n* Patients who are on chronic systemic immunosuppressive therapy except for those requiring steroid therapy for management of adrenal insufficiency; these patients may receive no more than 10 mg of prednisone or its equivalent daily. Transient use of steroids, e.g. in the perioperative period, is not an exclusion.\n* Patients who are pregnant or breastfeeding.\n* Patients who have active medical illness(es) that would pose increased risk for study participation, including: active systemic infections requiring systemic ABX, coagulation disorders, or other active major medical illnesses of the cardiovascular, respiratory, or immune systems.\n* Patients who have received a live or attenuated vaccination within 28 days prior to the start of NMA-LD pre-conditioning regimen.\n* Patients who have any form of primary immunodeficiency (such as severe combined immunodeficiency disease \\[SCID\\] and acquired immunodeficiency syndrome \\[AIDS\\]).\n* Patients who have a left ventricular ejection fraction (LVEF) \\\u003C45% or New York Heart Association (NYHA) functional classification \\> Class 1.\n\n  * Patients ≥ 60 years of age and who have a history of ischemic heart disease, chest pain, or clinically significant atrial and\u002For ventricular arrhythmias must have a cardiac stress test.\n  * Patients with any irreversible wall movement abnormalities are excluded.\n* Patients who have a smoking history or signs or symptoms of obstructive or restrictive pulmonary disease and have a documented forced expiratory volume in 1 second (FEV1) of ≤ 60% of predicted normal:\n\n  * If a patient is not able to perform reliable spirometry due to abnormal upper airway anatomy (i.e., tracheostomy), a 6-minute walk test may be used to assess pulmonary function.\n  * Patients who are unable to walk a distance of at least 80% predicted for age and sex or demonstrates evidence of hypoxia at any point during the test (SpO2 \\\u003C 90%) are excluded.\n* Active, uncontrolled systemic infections, including COVID-19, within 30 days of surgery or NMA-LD. An uncomplicated bacterial UTI treated successfully with symptom resolution is not an exclusion.\n* Participation in another clinical study with an investigational product within 21 days of the initiation of NMA-LD.\n* No other active, concurrent malignancy that requires ongoing systemic treatment (e.g. indolent prostate) or interferes with radiographic assessment of response as determined by the investigator. Exceptions may allow for adjuvant NED cancers undergoing hormone-based therapy assuming the other eligibility criteria are met and the PI affirms the hormonal agent would not change the response.\n\nEligibility Designation for Lymphodepletion\n\nPatients meeting eligibility criteria above between Day -21 and Day -8 prior to the planned initiation of lifileucel will be enrolled to the therapeutic portion of the protocol.\n\nAll patients' eligibility criteria, including repeating cardiopulmonary function tests as necessary, will be reassessed within several days prior to the scheduled lymphodepletion in all cases.\n\nPrior to beginning the NMA-LD preparative regimen the following requirements must be met:\n\n* Patients must meet all eligibility criteria at the time of NMA-LD.\n* Full resolution of any active infection should be documented\n* Critical evaluation of changes in cardiovascular, respiratory, renal, coagulopathy, or immune problems and other major illnesses that may have developed or worsened must be documented\n* Patients with symptomatic, recurrent, pleural effusions that require drainage should not proceed to lymphodepletion without prior placement of a temporary in-dwelling pleural drain\n* All active medical issues must be addressed by Investigator or designee. Re-evaluation with additional imaging or testing may be required\n\nBenefit over risk should be assessed and reassessed throughout the treatment course. Successive lifileucel components (preparative lymphodepleting chemotherapy, LN-144\u002FLN-145 and IL-2 ) should be withheld or discontinued if at any time during the treatment course, at the discretion of the treating physician, benefit may not be justified by risks to the patient\n\nSubsequent delays of lymphodepletion up to 14 days due to logistical issues such as production of lifileucel and\u002For major weather events will not constitute protocol violations and out of window assessments will not need to be repeated unless there is a change in clinical status.",{"count":206,"type":48},40,[51],"This is an open label study evaluating lifileucel (LN-144) in patients with metastatic uveal melanoma.",[210,211,212,213],"Uveal Melanoma","Melanoma","Metastatic Uveal Melanoma","Metastatic Melanoma",[210,211,212,213,215,216,217],"LN-144","22-109","Memorial Sloan Kettering Cancer Center",{"date":34,"type":35},{"date":220,"type":35},"2022-11-01",{"date":222,"type":48},"2027-05-01",{"name":217,"class":224},"OTHER",2,{"id":227,"slug":4,"hasResults":11,"nctId":228,"briefTitle":229,"officialTitle":230,"acronym":4,"eligibilityCriteria":231,"healthyVolunteers":11,"sex":232,"minAge":17,"maxAge":4,"enrollmentInfo":233,"targetDuration":4,"studyType":49,"phases":235,"briefSummary":237,"conditions":238,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":241,"startDateStruct":242,"completionDateStruct":244,"leadSponsor":246,"locationsCount":247},"100054227","NCT07198074","Testing the Addition of an Antiangiogenic Drug (Bevacizumab) to Chemotherapy (Carboplatin and Paclitaxel) Combined With Immunotherapy (Pembrolizumab) for pMMR, TP53 Mutated Endometrial Cancer","A Randomized Phase III Trial of Carboplatin, Paclitaxel, Pembrolizumab Versus Carboplatin, Paclitaxel, Bevacizumab Versus Carboplatin, Paclitaxel, Pembrolizumab, Bevacizumab in the Treatment of pMMR, TP53 Mutated Advanced or Recurrent Endometrial Cancer","Inclusion Criteria:\n\n* Documentation of disease:\n\n  * Stage III and stage IVA endometrial cancers (with measurable disease),\n  * Stage IVB endometrial cancer (with or without measurable disease), or\n  * Recurrent endometrial cancer (with or without measurable disease)\n* In patients with measurable disease, lesions will be defined and monitored by RECIST 1.1. Measurable disease (RECIST 1.1) is defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded). Each lesion must be ≥ 10 mm when measured by CT or MRI. Lymph nodes must be ≥ 15 mm in short axis when measured by CT or MRI\n* Histologic confirmation of the original primary tumor is required (submission of pathology report\\[s\\] is required). Patients with the following histologic types are eligible: endometrioid, serous, dedifferentiated\u002Fundifferentiated, clear cell, mixed epithelial, carcinosarcoma, adenocarcinoma not otherwise specified (N.O.S.)\n* Patients must have:\n\n  * Tumoral mismatch repair proficient (pMMR) disease as assessed by immunohistochemistry (IHC) AND\n  * P53 IHC with aberrant staining pattern (aberrant p53 expression is consistent with mutant TP53). TP53 mutation by next-generation sequencing will also be accepted\n* A pathology report demonstrating results of institutional MMR IHC and p53 IHC and\u002For TP53 by next-generation sequencing\n* Patients may have received:\n\n  * NO prior chemotherapy for treatment of endometrial cancer OR\n  * Prior adjuvant chemotherapy (e.g., paclitaxel\u002Fcarboplatin alone or as a component of concurrent chemotherapy and radiation therapy \\[with or without cisplatin\\]) provided adjuvant chemotherapy was completed ≥ 12 months prior to registration\n* Patients may have received prior radiation therapy for treatment of endometrial cancer. Prior radiation therapy may have included pelvic radiation therapy, extended field pelvic\u002Fpara-aortic radiation therapy, intravaginal brachytherapy, and\u002For palliative radiation therapy. All radiation therapy must be completed at least 4 weeks prior to registration. For patients with recent radiation, they must have RECIST-evaluable disease outside of the radiation field and have recovered their marrow function\n* Patients may have received prior hormonal (endocrine) therapy. All hormonal (endocrine) therapy must have been completed at least 1 week prior to registration\n* NO prior pembrolizumab (or other anti-PD1, anti-PDL1 or anti-CTLA4 therapy) or bevacizumab (or other antiangiogenic therapy)\n* Interval or cytoreductive surgery, after start of treatment on this trial, and prior to documentation of disease progression, is NOT permitted\n* Patients with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of disease progression. Patients with brain metastases must have follow up imaging demonstrating no evidence of disease progression and that the disease is stable off of steroids\n* Age ≥ 18\n* Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2\n* Not pregnant and not nursing\n* Absolute neutrophil count (ANC) ≥ 1,500 cells\u002Fmm\\^3\n* Platelets ≥ 100,000 cells\u002Fmm\\^3\n* Hemoglobin ≥ 8 g\u002Fdl\n* Creatinine clearance (CrCl) of ≥ 30 mL\u002Fmin by the Cockcroft-Gault formula\n* Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN) (patients with known Gilbert's disease who have bilirubin level ≤ 3 x institutional ULN may be enrolled)\n* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x institutional ULN\n* Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class II or better\n* No active infection requiring parenteral antibiotics\n* No current evidence of intra-abdominal abscess, abdominal\u002Fpelvic fistula (not diverted), gastrointestinal perforation, gastrointestinal (GI) obstruction, and\u002For need for drainage nasogastric or gastrostomy tube\n* No clinically significant bleeding within 28 days prior to registration\n* No uncontrolled hypertension, defined as systolic ≥ 160 mm Hg or diastolic ≥ 100 mm Hg\n* No major surgery within 28 days of initiation of bevacizumab\n* No active autoimmune disease or history of autoimmune disease that might recur, which may affect vital organ function or require immune suppressive treatment including corticosteroids. This includes, but is not limited to, patients with a history of immune related neurologic disease, multiple sclerosis, autoimmune (demyelinating) neuropathy, Guillain-Barre syndrome, myasthenia gravis; systemic autoimmune disease such as systemic lupus erythematosus (SLE), connective tissue diseases, scleroderma, inflammatory bowel disease (IBD), Crohn's, ulcerative colitis, hepatitis; and patients with a history of toxic epidermal necrolysis (TEN), Stevens-Johnson syndrome, or phospholipid syndrome because of the risk of recurrence or exacerbation of disease\n\n  * Patients with vitiligo, endocrine deficiencies including type I diabetes mellitus, thyroiditis managed with replacement hormones including physiologic corticosteroids are eligible\n  * Topical or inhaled steroids are allowed\n  * Patients with rheumatoid arthritis and other arthropathies, Sjogren's syndrome and psoriasis controlled with topical medication and patients with positive serology, such as antinuclear antibodies (ANA), and anti-thyroid antibodies should be evaluated with the presence of target organ involvement and potential need for systemic treatment but should otherwise be eligible\n* No history of (non-infectious) pneumonitis that required steroids, or current pneumonitis\n* No history of stem cell or solid organ transplant\n* No history of allergic reaction to the study agent(s) or compounds of similar chemical or biologic composition to the study agent(s) (or any of its excipients)","FEMALE",{"count":234,"type":48},255,[236],"PHASE3","This phase III trial compares the effect of bevacizumab in combination with carboplatin, paclitaxel and pembrolizumab to the usual treatments of carboplatin and paclitaxel with or without pembrolizumab in treating patients with stage III, IVA or IVB mismatch repair protein proficient (pMMR) and TP53 mutated endometrial cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that has come back after a period of improvement (recurrent). Bevacizumab is in a class of medications called antiangiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to tumor. This may slow the growth and spread of tumor. Carboplatin is in a class of medications known as platinum-containing compounds. Carboplatin works by killing, stopping or slowing the growth of tumor cells. Paclitaxel is in a class of medications called antimicrotubule agents. It stops tumor cells from growing and dividing and may kill them. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread. Adding bevacizumab to the combination of carboplatin, paclitaxel and pembrolizumab may be more effective than the usual treatment combinations of carboplatin and paclitaxel with or without pembrolizumab in treating patients with advanced or recurrent pMMR and TP53 mutated endometrial cancer.",[239,240],"Advanced Endometrial Carcinoma","Recurrent Endometrial Carcinoma",{"date":34,"type":35},{"date":243,"type":35},"2026-01-27",{"date":245,"type":48},"2028-07-01",{"name":177,"class":123},232,{"id":249,"slug":4,"hasResults":11,"nctId":250,"briefTitle":251,"officialTitle":252,"acronym":253,"eligibilityCriteria":254,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":255,"targetDuration":4,"studyType":49,"phases":257,"briefSummary":258,"conditions":259,"keywords":261,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":274,"startDateStruct":275,"completionDateStruct":277,"leadSponsor":279,"locationsCount":281},"100054209","NCT07428616","A Study of Zanzalintinib in Participants With Recurrent or Progressive Meningioma","A Phase 2, Single-Arm, Multicenter, Open-Label Study of Zanzalintinib in Participants With Recurrent or Progressive Meningioma","STELLAR-201","Key Inclusion Criteria:\n\n* Histologically confirmed World Health Organization (WHO) grade 1, 2, or 3 meningioma.\n* Developed recurrent disease or progressive disease (PD) after receiving standard therapy (for example, surgery and\u002For radiation) or have been deemed ineligible to receive these therapies. At least 1 prior course of meningioma-directed radiotherapy is required, if not contraindicated.\n* Radiologically documented progression of any existing tumor (growth \\> 15% of the bidimensional enhancing tumor within the prior 6 months or appearance of new lesions (including intra and extracranial manifestations).\n* For participants treated with external beam radiation, interstitial brachytherapy, or radiosurgery, an interval ≥ 24 weeks must have elapsed from completion of therapy to initiation of treatment.\n* Measurable disease by RANO meningioma criteria as determined by the investigator, obtained ≤ 14 days prior to initiation of treatment.\n* Karnofsky performance status (KPS) ≥ 60%.\n* Demonstrate adequate organ and marrow function within 14 days of treatment initiation\n\nKey Exclusion Criteria:\n\n* Prior history of hypertensive encephalopathy at any time.\n* Extracranial lesions invading major blood vessels including, but not limited to, inferior vena cava, pulmonary artery, or aorta.\n* Contraindication to magnetic resonance imaging (MRI).\n* Local therapy (surgery and\u002For radiation therapy) is indicated per investigator\n* Receipt of any type of cytotoxic, biologic or other systemic anticancer therapy (including investigational) within 4 weeks or 5 half-lives, whichever is shorter, before initiation of treatment. There is no limit on prior systemic therapies\n* Prior Surgery - completed wound healing must occur prior to initiation of treatment; ≥ 8 weeks for major surgery, ≥ 7 days for minor surgery, including stereotactic biopsies.\n* The participant has uncontrolled, significant intercurrent or recent illness including, but not limited to, the following conditions:\n\n  * Cardiovascular disorders, including uncontrolled hypertension,\n  * Gastrointestinal (GI) disorders including those associated with a high risk of perforation or fistula formation,\n  * Clinically significant hematuria, hematemesis, or hemoptysis of \\> 0.5 teaspoon (2.5 milliliters \\[mL\\]) of red blood within 12 weeks before initiation of treatment or other history of significant bleeding (eg, intracranial hemorrhage\u002Fbleeding), or\n  * Other clinically significant disorders.\n* Requirement for hemodialysis or peritoneal dialysis.\n* History of solid organ or allogeneic stem cell transplant.\n\nNote: Other protocol defined Inclusion\u002FExclusion criteria may apply.",{"count":256,"type":48},100,[52],"The objective of the study is to evaluate efficacy and safety of zanzalintinib in participants with recurrent or progressive meningioma refractory to standard therapies.",[260],"Meningioma",[260,262,263,264,265,266,267,268,269,270,271,272,273],"Recurrent Meningioma","Progressive Meningioma","Atypical Meningioma","Anaplastic Meningioma","Brain","Zanzalintinib","XL092","Grade 1 Meningioma","Grade 2 Meningioma","Grade 3 Meningioma","Intracranial Neoplasms","Extracranial Meningioma",{"date":34,"type":35},{"date":276,"type":35},"2026-05-21",{"date":278,"type":48},"2029-09-30",{"name":280,"class":38},"Exelixis",12,{"id":283,"slug":4,"hasResults":11,"nctId":284,"briefTitle":285,"officialTitle":286,"acronym":4,"eligibilityCriteria":287,"healthyVolunteers":11,"sex":16,"minAge":288,"maxAge":289,"enrollmentInfo":290,"targetDuration":4,"studyType":49,"phases":292,"briefSummary":293,"conditions":294,"keywords":296,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":306,"startDateStruct":307,"completionDateStruct":309,"leadSponsor":311,"locationsCount":124},"100054185","NCT06303193","Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic\u002FMyeloproliferative Neoplasms","Phase I\u002FII Trial of Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic\u002FMyeloproliferative Neoplasms","* INCLUSION CRITERIA:\n* Participants must have histologically or cytologically confirmed MDS or MDS\u002FMPN, including therapy-related MDS or MDS\u002FMPN, and MDS or MDS\u002FMPN with germline predisposition, by the Department of Laboratory Medicine Hematology Laboratory, CC or by the Laboratory of Pathology, NCI as defined according to the 2016 WHO criteria, 2022 WHO criteria, or 2022 International Consensus Classification\n* Age 12-17 years for phase I and age \\>= 18 years for phase II\n* Participants \\>= 18 years of age with HR-MDS must have resistance to hypomethylating agents as defined as failure to show improvement after at least 4 cycles of treatment (primary resistance) or relapse in participants with initial response to long-term treatment (secondary resistance) OR have intolerance to hypomethylating agents OR have a contraindication to hypomethylating agents\n* Participants \\>= 18 years of age with LR-MDS must be refractory to or ineligible to receive standard of care therapies, i.e. erythropoietin-stimulating agents, lenalidomide, luspatercept, and present with one of the following characteristics:\n\n  * Severe neutropenia defined by absolute neutrophils count \\\u003C=0.5(SqrRoot) 10\\^9\u002FL without the use of granulocyte colony-stimulating factors\n  * Symptomatic anemia defined by hemoglobin 16-week average \\\u003C10 g\u002FdL and symptoms that may include fatigue, weakness, reduced exercise tolerance, dyspnea on exertion, palpitations, (orthostatic) hypotension, near syncope and restless legs\n  * Thrombocytopenia defined as platelets \\\u003C20(SqrRoot) 10\\^9\u002FL or platelets \\\u003C50(SqrRoot) 10\\^9\u002FL and a history of clinically relevant non-major or major bleeding according to the ISTH classification\n* Participants 12-17 years of age with MDS must be relapsed\u002Frefractory OR ineligible to receive immunosuppressive therapy and hematopoietic stem cell transplantation\n\n  --Ineligibility to receive hematopoietic stem cell transplantation will include participants who are not anticipated to be candidates to receive transplantation within the next 3 months due to medical comorbidities, lack of appropriate donor, or logistical barriers to transplant\n* Participants with MDS\u002FMPN must be relapsed\u002Frefractory (failed a minimum of 1 standard of care therapy) OR ineligible to receive standard of care OR without known life-prolonging therapy options OR have a diagnosis for which no known standard of care exists\n* Participants 12-17 years of age must weigh \\>= 35 kg\n* If any of the prior therapies noted below were given to the participant, they must have been completed within the following timeframes:\n\n  * 7 days from last dose of short-acting myeloid growth factors (i.e., filgrastim) and \\>= 14 days for long-acting (i.e., pegfilgrastim)\n  * 14 days from last dose of short-acting thrombopoietic growth factors (i.e.,eltrombopag) and \\>= 28 days for long-acting (i.e., romiplostim)\n  * 14 days or 5 pharmacokinetic half-lives from biological therapy agent\n  * 21 days from myelosuppressive chemotherapy\n  * 28 days from last dose of immunosuppressive therapy (e.g., ATG, cyclosporine, steroids greater than physiologic replacement)\n  * 28 days from last dose of lenalidomide\n  * 28 days from last dose of venetoclax\n  * 28 days from any other investigational agent\n  * 42 days from last dose of erythropoiesis stimulating agents\n  * 56 days from last dose of luspatercept\n  * 100 days from stem cell transplant with no evidence of active graft vs. host disease in participants who relapsed following transplant\n  * 150 days from total body irradiation\n* Performance status:\n\n  * For participants \\>= 16 years of age, ECOG performance status \\\u003C 2 (Karnofsky \\>= 60%)\n  * For participants \\\u003C 16 years of age, Lansky \\>= 60%\n* Participants must have adequate organ function as defined below:\n\n  * Total bilirubin: \\\u003C= 1.5 X institutional upper limit of normal OR \\\u003C= 3 x institutional upper limit of normal in participants with Gilbert s syndrome\n  * AST(SGOT)\u002FALT(SGPT): \\\u003C= 2.5 X institutional upper limit of normal\n  * Creatinine clearance: \\>= 50 mL\u002Fmin\u002F1.73 m\\^2 for participants with creatinine levels above institutional normal\n  * PT and PTT: \\\u003C= 1.5 X institutional upper limit of normal, except in the setting of PTT elevation due to lupus anticoagulant, in which case these participants would be exempt from meeting this inclusion criterion\n* Individuals of child-bearing potential (IOCBP) and individuals able to father a child with a partner able to become pregnant must agree to use one (1) highly effective form of contraception (e.g., intrauterine device \\[IUD\\], surgical) or two (2) effective forms of contraception (e.g., barrier method) while on study drug and for 30 days after the last dose of study drug\n* Nursing participants must discontinue breastfeeding and\u002For not begin breastfeeding until 2 weeks after the last dose of study drug\n* Ability of participant or parent\u002Fguardian (for participants 12-17 only) to understand and the willingness to sign a written informed consent document\n\nEXCLUSION CRITERIA:\n\n* Participants with platelet transfusion-refractory thrombocytopenia, with inability to keep platelet threshold above 10 K\u002FmcL with transfusions\n* Participants with evidence of ongoing hemorrhage, active signs\u002Fsymptoms of bleeding, or history of severe (grade \\>= 3) unprovoked bleeding complications in the one year prior to enrollment, or any unprovoked grade 2 bleeding complications in the 3 months prior\n\nto enrollment\n\n* Use of anti-platelet or anticoagulant medication other than low-dose aspirin (100 mg daily or less) in the 14 days prior to enrollment, or any ongoing requirement for these medications\n* Participants who are unwilling to accept blood transfusions\n* Participants with ANC \\\u003C 500 cells\u002FmcL AND hospitalization for a fungal infection in the 12 months prior to enrollment\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib\n* Concomitant administration with sensitive substrates\u002Fnarrow therapeutic index drugs of CYP3A4, CYP1A2, P-gp BCRP, and OCT1 should be avoided. Concurrent use of strong inhibitors and inducers of CYP3A4 are not allowed. Prior use is allowed as long as medication is stopped two weeks prior to study drug initiation. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment\u002Finformed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.\n* Concomitant administration of medications with significant potential to cause QTc prolongation.\n* Participants with the following cardiac conditions at screening:\n\n  * Symptomatic congestive heart failure\n  * Unstable angina pectoris\n  * Uncontrolled cardiac dysrhythmias\n  * QTc(F) prolongation \\>450 ms, or other factors that increase the risk for QT prolongation (i.e., heart failure, or a history of long QT interval syndrome)\n* Grade \\>= 3 cardiac complication in the 6 months prior to enrollment\n* Left ventricular ejection fraction \\\u003C= 50% by transthoracic echocardiogram (TTE) at screening\n* Participants with any active, uncontrolled viral, bacterial, or fungal infection, including active HIV-1, Hepatitis B (HBV) and\u002For Hepatitis C (HCV) infection (positive HBV or HCV viral load in the setting of positive HBV core antibody or surface antibody or HCV antibody); history of HIV, HBV, or HCV is allowed if there is no uncontrolled viral infection\n* Pregnancy\n* Presence of another known cause of cytopenia or dysplastic marrow that is untreated and may limit interpretation of results\n* Uncontrolled intercurrent illness or any significant disease, evaluated by history, physical exam and chemistries or social situations that may limit interpretation of results, limit compliance with study requirements, or that could increase risk to the participant","12 Years","120 Years",{"count":291,"type":48},160,[51,52],"Background:\n\nMyelodysplastic syndrome (MDS) and myelodysplastic\u002Fmyeloproliferative neoplasm (MDS\u002FMPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS\u002FMPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed.\n\nObjective:\n\nTo test a study drug (pacritinib) in adults and children with MDS or MDS\u002FMPN.\n\nEligibility:\n\nChildren (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS\u002FMPN.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone.\n\nPacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants.\n\nParticipants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years.\n\nBone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.",[295],"Myelodysplastic Syndromes",[297,298,299,300,301,302,303,304,305],"Myelodysplastic Syndrome","bone marrow disorder","Myelodysplasia","myeloproliferative disorders","pediatric bone marrow failure","5q deletion","multi-lineage dysplasia","Clonal Hematopoiesis","Aplastic Anemia",{"date":34,"type":35},{"date":308,"type":48},"2026-07-16",{"date":310,"type":48},"2035-01-01",{"name":177,"class":123},{"id":313,"slug":4,"hasResults":11,"nctId":314,"briefTitle":315,"officialTitle":316,"acronym":4,"eligibilityCriteria":317,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":318,"enrollmentInfo":319,"targetDuration":4,"studyType":49,"phases":320,"briefSummary":321,"conditions":322,"keywords":325,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":331,"startDateStruct":332,"completionDateStruct":333,"leadSponsor":335,"locationsCount":124},"100054161","NCT07111065","FAST for DM - Fatty Acid Supplementation Trial (FAST) for Dermatomyositis (DM)","Phase II, Randomized, Double-Blind, Placebo-Controlled Trial of Omega-3 Fatty Acid (O3FA) Supplementation for Adult and Juvenile Dermatomyositis (DM\u002FJDM)","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n* Adults 18-60 years of age with probable or definite EULAR-ACR criteria for adult or juvenile dermatomyositis (DM, JDM).\n* Willingness to adhere to the general healthy diet pattern regimen, undergo dietary coaching on weekly to biweekly basis (10 sessions), and to complete online random reporting of dietary intake over a 6-month period.\n* Ability and willingness to comply with taking 4 study pills twice a day for 6 months.\n* Ability and willingness to wear ActiGraph device at home for 7 continuous days, twice in the study.\n* Willingness and ability to complete and consent to study testing, including blood, stool, and urine samples, and imaging studies.\n* Ability and willingness to complete a total of 5 study visits (screening, weeks -6, 0, 12, 24) onsite at NIH Clinical Center in Bethesda, Maryland.\n* Has the ability\u002Ftransportation methods to attend on-site visits. Willing to pay for travel and out-of-pocket expenses.\n* Own or have reliable access to a computer, laptop or smart phone device (iPhone or Android) with internet access, and an active email address, to complete study consent form, online questionnaires, telehealth visits, and review online dietary education materials and videos.\n* Ambulatory\n* Must live within the United States.\n* Must be proficient in the English language and complete questionnaires in English (forms validated in English). Ability and willingness to complete forms online.\n* Moderately active DM\u002FJDM defined by:\n\n  * MD global VAS with a \\>= 2.0 cm on a 10 cm scale and maximum value of 7.0 cm, and\n  * At least 2 of the following criteria:\n\n    * Patient global activity \\>= 2 cm out of 10 cm visual analog scale (VAS).\n    * MMT-8 score of \\\u003C=138 out of 150.\n    * Health Assessment Questionnaire disability index with a minimum value of \\>= 0.50 out of 3.0\n    * Elevation of at least one of the muscle enzymes (which includes creatine kinase (CK), aldolase, lactate dehydrogenase (LDH), ALT and AST) at a minimum level of 1.3X the upper limit of normal.\n    * Global Extramuscular disease activity score with a minimum value of \\>= 1.0 cm on a 10 cm VAS scale (this measure is the physician s composite evaluation and is based on assessments of activity scores on the constitutional, cutaneous, skeletal, gastrointestinal, pulmonary and cardiac scales of the Myositis Disease Activity Assessment Tool (MDAAT).\n* Physician global damage and muscle damage both \\\u003C= 5.0 cm\u002F10 cm VAS\n* If receiving prednisone and methotrexate, the dose must be stable for at least 4 weeks prior to the Week 6 visit, and daily prednisone \\\u003C= 20 mg\u002Fday.\n* Background therapy with other non-corticosteroid immunosuppressive agent, if required, must be at a stable dose for at least 6 weeks prior to the Week 6 visit, except with IVIG regimen should be stable 90 days prior to the Week 6 visit and for rituximab, stable regimen for 4 months prior to Week 6.\n* If an immunosuppressive agent was discontinued prior to the screening visit, then there must be a washout period before week -6 visit:\n\n  * 4-week washout for prednisone, methotrexate, and IV methylprednisolone (IV pulse therapy)\n  * 8-week washout for other immunosuppressive drugs, including azathioprine, mycophenolate mofetil, leflunomide, tacrolimus, cyclosporine, cyclophosphamide, colchicine, and hydroxychloroquine\n  * 8-week washout for IVIG\n  * For discontinuation of biologic or targeted drug therapies or dietary supplements, a washout prior to visit 1 (week 6) is required of 4 terminal half-lives.\n\n    * Half-lives of most common biologics and targeted drug therapies used in the treatment of DM\u002FJDM:\n\n      * Etanercept Half-life 70 hours, Waiting period before enrollment (4 Half-lives) 12 days\n      * Adalimumab, Half-life, 14 days, Waiting period before enrollment (4 Half-lives) 60 days\n      * Rituximab, Half-life 32 days, Waiting period before enrollment (4 Half-lives) 130 days\n      * Infliximab, Half-life 9 days, Waiting period before enrollment (4 Half-lives) 36 days\n      * Abatacept, Half-life 17 days, Waiting period before enrollment (4 Half-lives) 68 days\n      * Anakinra, Half-life 6 hours, Waiting period before enrollment (4 Half-lives) 1 day\n      * Tofacitinib, Half-life 3 hours, Waiting period before enrollment (4 Half-lives) 1 day\n      * Baricitinib, Half-life 12hours, Waiting period before enrollment (4 Half-lives) 2 days\n* Negative pregnancy test (urine or blood sample) if born female.\n* Body Mass Index (BMI) \\> 18 and \\\u003C= 35 kg\u002Fm\\^2\n* Fish intake of less than 2 servings per week on average for the past 3 months.\n* Intake of meat products (beef, lamb, pork, venison, rabbit, cow s milk or dairy products) within 2 months of screening visit and of week 0 and have no reaction (no shortness of breath, hives, rash, or diarrhea) within 6 hours of ingestion of these meat products.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n* Polymyositis; inclusion body myositis; cancer-associated myositis, defined as the diagnosis of myositis within 3 years of the diagnosis of cancer, except basal or squamous cell skin cancer or carcinoma in situ of the cervix if at least 5 years since excision.\n* Myositis in overlap with another autoimmune disease will be excluded under the following conditions:\n\n  i. Myositis overlapping with another autoimmune disease that precludes accurate assessment of treatment response (e.g., difficulty assessing muscle strength in a patient with scleroderma and associated myositis).\n\nii. Myositis overlapping with inflammatory bowel disease, including Crohn s disease, ulcerative colitis, or celiac disease.\n\niii. Myositis overlapping with autoimmune thyroid disease (e.g., Hashimoto s disease or Graves disease), unless the thyroid disease is\n\nstable and well controlled with no changes in thyroid-related medications for at least 3 months prior to enrollment, in which case the\n\npatient may be included.\n\n* Drug- or toxin-induced myositis, including known HMG-CoA reductase autoantibody-positive necrotizing myopathy following statin use.\n* Moderate to severely active myositis that would require initiation of another immunosuppressive treatment.\n* Joint disease, severe calcinosis, or other musculoskeletal condition, which precludes the ability to assess\u002Fquantitate muscle strength and function.\n* Concomitant illness that would prevent adequate patient assessment or in the investigators opinion pose an added risk for study participants. The investigator may consider further evaluation or consultation if clinically indicated prior to study enrollment:\n\n  * Recurrent or chronic infections, including HIV, hepatitis B and C, Epstein Barr virus, active coronavirus infection, active or recurrent gastrointestinal infection (including Helicobacter pylori), active or recurrent skin infections with calcinosis.\n  * Disorders that would preclude accurate assessment of neuromuscular function.\n  * Severe swallowing dysfunction with inability to swallow pills.\n  * Patients with generalized lipodystrophy.\n  * Severe cardiomyopathy or arrhythmias, including atrial fibrillation or atrial flutter, New York Heart Association Classification III or IV for congestive heart failure, severe interstitial lung requiring oxygen therapy, gastrointestinal vasculopathy\u002Fulceration or gastroparesis, renal failure requiring dialysis, that in the investigators opinion poses an additional risk for study participants.\n  * Subjects with any acute and life-threatening condition unrelated to myositis, such as prior sudden cardiac arrest, acute myocardial infarction, stroke, embolism in last 3 months.\n  * History of malignancy, except basal or squamous cell skin cancer or carcinoma in situ of the cervix if at least 5 years since excision.\n  * Uncontrolled hypertension with average blood pressure \\>= 140\u002F90, requiring a new anti-hypertensive medication in the past 8 weeks.\n  * Psychiatric illness that precludes compliance or neuromuscular assessment, including major psychiatric illness requiring hospitalization within the past year and\u002For has had a change in depression or anxiety prescription medications within the past 3 months (by discretion of study physician).\n  * Subjects with chronic diarrhea, gastric bypass or lap-band procedures, ostomies, bowel motility problems, irritable bowel syndrome, symptomatic gallstones, or other conditions that could affect intestinal fat absorption.\n  * Subjects with clinically diagnosed hepatic disease, including but not limited to hepatitis, steatosis, cirrhosis.\n  * Osteoporotic fracture under therapy for pain control or impacting ambulation.\n  * Serum creatinine \\> 2.0mg\u002Fdl or eGFR less than 50 mL\u002Fmin per 1.73 m\\^2.\n  * Subjects with coagulation or bleeding disorders (such as hemophilia) or receiving anti-platelet or anti-coagulant medications, including daily aspirin, warfarin, or Plavix.\n  * Life-threatening non-myositis illness that would interfere with the patient s ability to complete the study.\n* Known contraindications to O3FAs, excipients or placebo contents (e.g., allergy or known hypersensitivity to that drug or its excipients, including porcine gelatin, allergies to fish or shellfish, tocopherols, glycerin, or corn). Religious or ethical reasons to not consume fish, corn and\u002For porcine (pork) products.\n* Participants with any of the following:\n\n  * Idiopathic anaphylaxis\n  * Alpha-gal reaction\n  * Known food allergies to beef, pork, lamb or other meat products, including cow s milk and dairy products, with a history of shortness of breath, rash, or hives within 6 hours of eating these foods.\n* Currently using O3FAs or consuming EPA\u002FDHA in any form for the past 6 months.\n* Currently taking supplements or medications that affect lipoproteins for the past 6 months, including fish oil supplements, bile-acid sequestrants, plant sterol supplements, PCSK9 inhibitors, fibrates, statins, or niacin.\n* Use of medications or dietary supplements that interact with O3FA per pharmacy evaluation. A PharmD will evaluate the patient's current medication list for medications\u002Fsupplements with the potential for significant interactions with O3FA.\n* No antibiotic usage in past 3 months, as well as no usage of anti-virals, antifungals, anti-parasitics in past 3 months (except antimalarials and Paxlovid or other COVID-19 anti-viral therapy allowed).\n* Subjects being treated with tamoxifen, estrogens or progestins that have not been stable for \\> 4 weeks.\n* Uncontrolled diabetes with HgbA1C \\> 8 or hospitalization in past 6 months for diabetes.\n* Uncontrolled hyperlipidemia with TC \\> 400 mg\u002FdL, TG \\>150mg\u002FdL.\n* Currently on a weight-loss program\n* Has experienced a weight change (gain or loss) of greater than 15 pounds or greater than 20 percent in the past 3 months\n* Currently taking a GLP-1 receptor agonist medication\n* No restrictive dietary habits per discretion of the study team.\n* Current use of medications or dietary supplements for weight or appetite control, including laxatives or diarrheal inhibitors within the past 4 weeks.\n* History of eating disorder.\n* Initiation of an exercise program within 4 weeks of screening visit.\n* Known or suspected history of drug or alcohol abuse within the past 6 months as determined by the medical record or patient interview.\n* Blood donation in the last 6 weeks or planned blood donation during study or requiring regular blood transfusion.\n* Pregnant females or nursing mothers within past 3 months, or those planning to get pregnant during the next 9 months.\n* Low total WBC \\\u003C 2000, platelets \\\u003C 100,000\u002Fmm\\^3; hemoglobin \\\u003C 9.5 gm\u002Fdl.\n* Vitamin D level \\\u003C 20 ng\u002Fml (at screening visit - necessitates addition of supplement and re-screen after minimum of 8 weeks).\n* Subjects with TSH levels greater than 1.5X upper limit of normal or clinical evidence of hypothyroidism (at screening visit- necessitates addition of supplement and re-screen after minimum of 8 weeks).\n* Participants with severe claustrophobia.\n* History of or anticipated poor non-cooperation with study requirements.\n* Participation in another clinical experimental therapeutic study within 30 days of screening visit or during the study.\n* Hospitalization within past 30 days (other than for routine infusions).\n* Prisoners or subjects who are involuntarily incarcerated.\n* Resident of a nursing home, ward of the state, or institutionalized during any part of the study period.\n* Persons with decisional incapacity\u002Fcognitive impairment.\n* Any history or evidence of severe illness or any other condition that would make the patient, in the opinion of the investigator, unsuitable for the study.\n* Participants who do not complete the ASA24 within 4 calendar days of screening will be excluded from the protocol. Additionally, participants will be excluded if their energy intake from the ASA24 is above or below established cut-off values for age and gender based on the 5th and 95th percentile of energy intakes from National Health and Nutrition Examination Survey (NHANES) data. Cut-off values for exclusion are \\\u003C600 kcal or \\>4400 kcal for women and \\\u003C650 kcal and \\>5700 kcal for men.","60 Years",{"count":134,"type":48},[52],"Dermatomyositis (DM) is a rare autoimmune disease that causes muscle weakness, skin rashes, and other symptoms. Researchers think both genetic and environmental factors play a role in this disease. They want to find out more about how diet and lifestyle choices affect people with DM\u002FJDM.\n\nObjective:\n\nTo see if omega-3 fatty acid supplements from fish oil, combined with a healthy diet, can help people with DM\u002FJDM.\n\nEligibility:\n\nAdults 18-60 years old, who live in the United States, can read English, and access Internet to complete questionnaires can participate.\n\nDesign:\n\nParticipants will have 5 or 6 inpatient visits. For 5 visits they may need to stay in the Clinical Center for up to 5 days. Participants will be screened. They will have a physical exam with blood, urine and stool tests. They will have tests of their heart and lung function. Their muscle strength will be measured. They may have an imaging scan of their thighs and pelvis. They will complete online questionnaires about their health and lifestyle. They may complete two optional skin biopsies. Participants will take 4 small capsules by mouth twice a day for up to 6 months. The capsules will contain omega-3 fatty acids from fish oil or a placebo. The placebo looks just like the regular capsule but contains no active ingredients. Participants will not know which capsules they are taking. They will follow a healthy diet based on the General Healthy Eating Pattern.\n\nParticipants will receive dietary coaching and will have virtual check-ins throughout the study. For two 7-day periods, they will wear a watch-like device to track their daily activity and sleep patterns. Participants may opt to remain in the study for an additional 12 weeks. All will receive the fish oil supplements during this stage.",[323,324],"Dermatomyositis (DM)","Juvenile Dermatomyositis (JDM)",[326,327,328,329,330],"Fish oil supplements","Omega-3 Fatty Acid","Clinical Trial","Online questionnaires","Diet Study",{"date":34,"type":35},{"date":308,"type":48},{"date":334,"type":48},"2031-11-03",{"name":336,"class":123},"National Institute of Environmental Health Sciences (NIEHS)",{"id":338,"slug":4,"hasResults":11,"nctId":339,"briefTitle":340,"officialTitle":341,"acronym":4,"eligibilityCriteria":342,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":343,"targetDuration":4,"studyType":49,"phases":345,"briefSummary":346,"conditions":347,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":352,"startDateStruct":353,"completionDateStruct":355,"leadSponsor":357,"locationsCount":358},"100053397","NCT07281417","Testing the Addition of Cemiplimab (REGN2810) to Chemotherapy Treatment Given Prior to Surgery in Patients With Sinonasal Squamous Cell Carcinoma","Neoadjuvant Chemotherapy With or Without Cemiplimab (REGN2810) in Sinonasal Squamous Cell Carcinoma: A Randomized Phase 2 Study","Inclusion Criteria:\n\n* Patients must have histologically confirmed squamous cell carcinoma of sinonasal origin\n* Patients must have a T stage (T3, T4a, and select T4b) primary tumor according to American Joint Committee on Cancer (AJCC) 8th edition. Patients must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal lesions and short axis for nodal lesions) as ≥ 20 mm (≥ 2 cm) by chest x-ray or as ≥ 10 mm (≥ 1 cm) with CT scan, MRI, or calipers by clinical exam\n* No evidence of metastatic disease determined by pre-treatment imaging. Metastatic disease to neck nodes is considered locally advanced and therefore allowable. Patients with N0 and N1-3 disease will be eligible\n* Known HPV status (i.e., HPV negative, p16 immunohistochemistry \\[IHC\\] positive, high risk \\[HR\\]-HPV in situ hybridization \\[ISH\\] positive) from testing performed prior to referral. HPV status data (e.g., date of test, type of test \\[p16 IHC or HR-HPV ISH\\] and testing result) must be collected during enrollment. Patients who do not have this information available for collection will not be enrolled on this study\n* Age ≥ 18 years\n\n  * Because no dosing or adverse event data are currently available on the use of cemiplimab (REGN2810) in combination with carboplatin and paclitaxel in patients \\\u003C 18 years of age, children are excluded from this study\n* Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 60%)\n* Hemoglobin ≥ 8 g\u002FdL (acceptable to reach via transfusion)\n* Absolute neutrophil count ≥ 1,500\u002FmcL\n* Platelets ≥ 100,000\u002FmcL\n* Total bilirubin ≤ 1.5 × institutional upper limit of normal (ULN)\n* Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \\[SGOT\\])\u002Falanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \\[SGPT\\]) ≤ 3 × institutional ULN\n* Creatinine clearance ≥ 40 mL\u002Fmin\n* Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial\n* For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated\n* Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load\n* Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial\n* Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class II or better\n* Based on its mechanism of action, cemiplimab (REGN2810) can cause fetal harm when administered to a pregnant woman. Animal studies have demonstrated that inhibition of the PD-1\u002FPD-L1 pathway can lead to increased risk of immune-mediated rejection of the developing fetus resulting in fetal death. For this reason and because paclitaxel is a class D agent with the potential for teratogenic or abortifacient effects, women of childbearing potential (WCBP) and men should use highly effective contraception during treatment and for 6 months after the last dose of the study drugs. WCBP and men should avoid donating eggs\u002Fsperm during treatment and for 6 months after the last dose of the study drugs. Women should discontinue nursing during treatment and for 6 months after the last dose of the study drugs\n* Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants\n* Prior to enrollment, verification of payment coverage by insurance (or other payment) for neoadjuvant paclitaxel and carboplatin chemotherapy must be obtained\n\nExclusion Criteria:\n\n* Patients with unresectable disease\n* Patients presenting with T3 disease without the need for maxillectomy and\u002For orbital invasion requiring orbital dissection\u002Fresection\n* Patients who have had any previous systemic therapy to the index lesion in the past 12 months. This includes cemiplimab (REGN2810) and\u002For other immune modulating agents. Previous systemic therapy may alter or affect response\n* Patients who had palliative RT (\\\u003C 20 Gy) within 1 week prior to entering the study\n* Ongoing or recent (within 5 years) evidence of significant autoimmune disease that required treatment with systemic immunosuppressive treatments, which may suggest risk for immune-mediated adverse events (imAEs)\n* History of pneumonitis within the last 5 years\n* Patients who have not recovered from adverse events (AEs) due to prior anti-cancer therapy (i.e., have residual toxicities \\> grade 1) with the exception of alopecia\n* Patients who are receiving any other investigational agents\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to cemiplimab (REGN2810) or carboplatin and paclitaxel\n* Patients with uncontrolled intercurrent illness or any other significant condition(s) that would make participation in this protocol unreasonably hazardous\n* Pregnant women are excluded from this study because of the increased risk of immune-mediated rejection of the developing fetus with cemiplimab (REGN2810). Men and WCBP who are not prepared to use highly effective contraception during and for 6 months after completion of treatment are excluded from this study",{"count":344,"type":48},108,[52],"This phase II trial compares the effect of chemotherapy (carboplatin and paclitaxel) with versus without cemiplimab given before surgery (neoadjuvant) in patients with sinonasal squamous cell cancer. Carboplatin is in a class of medications known as platinum-containing compounds. Carboplatin works by killing, stopping or slowing the growth of cancer cells. Paclitaxel is in a class of medications called antimicrotubule agents. It stops cancer cells from growing and dividing and may kill them. Immunotherapy with monoclonal antibodies, such as cemiplimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. The usual approach for patients with sinonasal squamous cell cancer is surgery followed by radiation therapy, with or without chemotherapy. Recently, some patients have also been treated with neoadjuvant chemotherapy before surgery. Adding cemiplimab to chemotherapy before surgery may be more effective at stopping the cancer from growing or spreading, compared to chemotherapy alone.",[348,349,350,351],"Sinonasal Squamous Cell Carcinoma","Stage III Sinonasal Cancer AJCC v8","Stage IVA Sinonasal Cancer AJCC v8","Stage IVB Sinonasal Cancer AJCC v8",{"date":34,"type":35},{"date":354,"type":48},"2026-11-24",{"date":356,"type":48},"2030-12-16",{"name":177,"class":123},5,{"id":360,"slug":4,"hasResults":11,"nctId":361,"briefTitle":362,"officialTitle":363,"acronym":4,"eligibilityCriteria":364,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":365,"enrollmentInfo":366,"targetDuration":4,"studyType":49,"phases":368,"briefSummary":369,"conditions":370,"keywords":372,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":385,"startDateStruct":386,"completionDateStruct":388,"leadSponsor":390,"locationsCount":392},"100053387","NCT06318169","A Study Evaluating the Efficacy and Safety of Pegozafermin in Participants With MASH and Fibrosis (ENLIGHTEN-Fibrosis)","A Phase 3 Study to Evaluate the Efficacy and Safety of Pegozafermin in Subjects With Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis","Key Inclusion Criteria:\n\n* Males or non-pregnant females aged between 18 and 80 years (inclusive) at time of signing the informed consent form (ICF)\n* Biopsy-confirmed MASH with fibrosis stage F2 or F3\n* Body mass index (BMI) at screening ≥25.0 kilograms (kg)\u002Fmeters squared (m\\^2) (≥23 kg\u002Fm\\^2 for Asian participants).\n\nKey Exclusion Criteria:\n\n* Chronic liver diseases other than MASH\n* Evidence of cirrhosis on screening liver biopsy\n* Have type 1 diabetes or poorly controlled type 2 diabetes\n* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥250 units per liter (U\u002FL)\n* Participants taking vitamin E (\\>400 international units \\[IU\\]\u002Fday) or pioglitazone must be on stable dose for at least 6 months prior to Screening\n\nOther inclusion and exclusion criteria may apply.","80 Years",{"count":367,"type":48},1350,[236],"The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.",[371],"Metabolic Dysfunction-Associated Steatotic Liver Disease (MASH) \u002F Nonalcoholic Steatohepatitis (NASH) With Fibrosis",[373,374,375,376,377,378,379,380,381,382,383,384],"Liver Diseases","Fatty Liver","Digestive System Diseases","Metabolic diseases","Metabolic Dysfunction-Associated Steatohepatitis","MASH","Steatohepatitis","Metabolic dysfunction-associated steatotic liver disease","MASLD","Nonalcoholic fatty liver disease","NAFLD","Fibrosis",{"date":34,"type":35},{"date":387,"type":35},"2024-03-13",{"date":389,"type":48},"2029-02",{"name":391,"class":38},"89bio, Inc.",361,{"id":394,"slug":4,"hasResults":11,"nctId":395,"briefTitle":396,"officialTitle":397,"acronym":398,"eligibilityCriteria":399,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":400,"targetDuration":4,"studyType":49,"phases":402,"briefSummary":404,"conditions":405,"keywords":408,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":415,"startDateStruct":416,"completionDateStruct":418,"leadSponsor":420,"locationsCount":124},"100053354","NCT07000045","Intravascular Lithotripsy With or Without Rotational Atherectomy for Coronary Calcified Nodule Treatment","Coronary Calcified Nodule Management With Rotational Atherectomy Plus Shockwave Intravascular Lithotripsy vs. Intravascular Lithotripsy Alone: A Prospective, Randomized Trial (NODULE-SHOCK)","NODULE-SHOCK","Inclusion Criteria\n\n* Age ≥18 years who signed written informed consent\n* Presence of a clinical indication for coronary intervention\n* Patients undergoing PCI for a de novo calcified lesion with planned drug-eluting stent (DES) implantation\n* Native coronary artery with significant stenosis defined as:\n* ≥70% and \\\u003C100% stenosis on angiography, or\n* 50-70% stenosis with evidence of ischemia (positive stress test, FFR ≤ 0.8, or OCT minimal lumen area (MLA) ≤ 4.0 mm2)\n* Reference vessel diameter: ≥2.5 mm to ≤ 4.0 mm\n* Lesion length: ≥5mm\n* Moderate to severe calcification of the target lesion confirmed by angiography\n* Thrombolysis in Myocardial Infarction (TIMI) flow grade 3 at baseline\n\nExclusion Criteria\n\n* Cardiogenic shock at the time of procedure\n* Primary PCI for ST-segment elevation myocardial infarction (STEMI)\n* Pregnant, nursing, or childbearing potential without adequate contraception\n* Known allergy or contraindication to DAPT (aspirin, clopidogrel, prasugrel, ticagrelor)\n* Planned surgery within 6 months unless DAPT can be maintained\n* Life expectancy \\\u003C12 months due to a serious medical illness (e.g., advanced cancer, end-stage heart failure)\n* Severe kidney dysfunction (CrCl \\\u003C30 mL\u002Fmin) without dialysis\n* Concurrent participation in another investigational study\n* Referral for coronary artery bypass grafting (CABG) after a heart team discussion\n* Angiographic evidence of thrombus at the target lesion\n* Angiographic evidence of significant dissection at the treatment site prior to intervention\n* Lesion with a previously placed stent within 10mm (visual estimate)\n* Last remaining vessel with severely compromised left ventricular function (LVEF \\\u003C30%)\n* Target lesion within a saphenous vein graft (SVG)",{"count":401,"type":48},120,[403],"NA","The NODULE-SHOCK trial is a prospective, investigator-initiated, single-center, randomized controlled trial designed to compare the efficacy of intravascular lithotripsy (IVL) with or without rotational atherectomy (RA) in patients with coronary calcified nodules (Cohort A), and operator-determined vs maximum IVL pulses in patients with non-nodular severe coronary calcium (Cohort B).",[406,407],"Coronary Artery Disease","Coronary Calcification",[409,410,411,412,413,414],"Coronary calcification","Calcified nodule","Intravascular lithotripsy","Rotational atherectomy","Optical coherence tomography","Percutaneous coronary intervention",{"date":34,"type":35},{"date":417,"type":35},"2025-10-06",{"date":419,"type":48},"2027-12-31",{"name":421,"class":224},"Annapoorna Kini",{"id":423,"slug":4,"hasResults":11,"nctId":424,"briefTitle":425,"officialTitle":425,"acronym":4,"eligibilityCriteria":426,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":427,"enrollmentInfo":428,"targetDuration":4,"studyType":49,"phases":430,"briefSummary":431,"conditions":432,"keywords":434,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":440,"startDateStruct":441,"completionDateStruct":442,"leadSponsor":444,"locationsCount":124},"100054106","NCT06664814","An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis","* INCLUSION CRITERIA:\n\nIndividuals must meet all the following inclusion criteria to be eligible to participate in this study:\n\n* Prior kidney biopsy demonstrating FSGS obtained within 10 years prior to the screening visit.\n* Age \\>=18 years weighing more than 50 kg.\n* If the patient is on immunosuppressive therapy (e.g., prednisone, cyclosporine, tacrolimus or mycophenolate mofetil) he\u002Fshe should be on these medications for at least 3 months prior to study evaluation and should be on a stable dose of the medication for at least 4 weeks before start of trial, with no plans to alter the regimen during 12 weeks of study period, except to stabilize levels and\u002For for any safety concerns.\n* Subjects will be allowed to continue with standard of care (SOC) non-immunosuppressant antiproteinuric agents to include RAAS inhibitors, mineralocorticoid antagonists (MRA), sodium-glucose co-transporter-2 inhibitors (SGLT2i), non-dihydropyridine calcium channel blockers (NDHP-CCBs), and glucagon-like peptide-1 (GLP-1) receptor agonists, in addition to other SOC adjuvant therapies such as diuretics, if they are able to maintain a stable dose throughout the trial. Subjects and their primary nephrologists will be encouraged to optimize their SOC treatments as much as possible prior to trial commencement. Subjects must be on a stable dose for at least 4 weeks before start of trial. Subjects should attempt to keep stable doses of both immunosuppressants and anti-proteinuric drugs throughout trial duration, to avoid confounding effects. Patients not receiving any of these SOC agents either due to allergy or intolerance will still be eligible.\n* Subjects must have a spot random urine PCR of \\>= 2g\u002Fg on each of 3 measurements collected on at least 2 separate days during screening period plus a 24-hr urine protein collection of \\>= 2g\u002Fday. The rationale for using this degree of proteinuria is that proteinuria beyond this threshold value significantly increases the risk of progressive decline of renal functions in the absence of effective therapies to mitigate this risk. Conversely, this threshold value could also allow for the selection of a cohort of patients who are most likely to benefit from ManNAc therapy.\n* Subjects with an estimated glomerular filtration rate (eGFR) \\>=45 mL\u002Fmin\u002F1.73\u002Fm\\^2 using the race-free CKD-EPI 2021 equation based on creatinine and cystatin-C \\[Inker et al, 2021\\]. The rationale is that below this eGFR threshold, sialic acid, the key metabolite of ManNAc markedly accumulates and may potentially result in systemic toxicity. Prior pharmacokinetic studies have shown that renal elimination of sialic acid is primarily through glomerular filtration, and it is neither reabsorbed nor secreted in the renal tubules. Hence decline in eGFR below 45 mL\u002Fmin\u002F1.73m\\^2 directly correlates with rising blood sialic acid levels \\[Fuentes et al, 2020\\], in addition, these subjects may not benefit as much from ManNAc therapy as they have advanced disease pathology, which may be irreversible. Future studies with personalized precision ManNAc dosing may benefit the patient population with eGFR \\\u003C45 mL\u002Fmin\u002F1.73m\\^2.\n* Subjects of reproductive potential must be willing to use at least one effective form of birth control throughout the trial period, unless they have had a permanent birth control procedure\u002Fintervention including but not limited to hysterectomy, tubal ligation and\u002For vasectomy. These may include the following: barrier methods (such as condoms), oral or depot injection (for example, Norplant or Depo-Provera) contraception medication, and\u002For intrauterine devices.\n* Evidence of a personally signed and dated informed consent indicating that the patient has been informed of all pertinent aspects of the study and their willingness to comply with all aspects of the study and their willingness to comply with all aspects of the study protocol, including treatment plan, laboratory tests, baseline and follow-up visits and procedures that include completion of daily diaries to record symptoms and medication intake.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n* Individuals who are unwilling or unable to provide informed consent.\n* Individual presenting for initial therapy of uncontrolled nephrotic syndrome which we define as proteinuria as measured by spot urine protein:creatinine ratio \\>3g\u002Fg or 24-hour urine protein excretion of \\>3.5g\u002Fday with symptomatic peripheral edema or pulmonary edema, hypoalbuminemia (serum albumin \\\u003C3.0g\u002Fdl), electrolyte disturbances (related or unrelated to medical therapy), and\u002For active thromboembolism (initiated on systemic anticoagulation in the last 3 months). The rationale for this criterion is that patient presenting for initial therapy for uncontrolled nephrotic syndrome may need to be initiated on various medications including immunosuppressives as well as non-immunosuppressive drugs which can significantly change the level of proteinuria. Additionally, fluid shifts due to diuretic use for treating edema can alter GFR which could confound the pharmacokinetics and pharmacodymanics of the investigational drug.\n* Individuals who acutely require optimization of volume status with intravenous diuretics to control volume overload, as this may result in fluid shifts between the intravascular space and the remainder of extracellular fluid volume. This might alter drug pharmacokinetics and pharmacodynamics as mentioned above in # 2.\n* Individuals with a psychiatric illness or neurological disease that in the judgement of the investigators would interfere with the ability to adhere with the requirements of this protocol. This includes, but is not limited to, uncontrolled\u002Funtreated psychotic depression, bipolar disorder, schizophrenia, substance abuse or dependence, antisocial personality disorder, panic disorder, or behavioral problems, which might interfere with effective communication.\n* Vulnerable individuals, including those with impaired cognitive function or are incarcerated.\n* Individuals whose renal biopsy shows evidence of an additional pathology along with FSGS.\n* Renal biopsy showing interstitial fibrosis and tubular atrophy (IFTA) \\>50% per biopsy report.\n* Individuals with uncontrolled hypertension with blood pressures consistently \\>140\u002F90 mmHg on 3 or more community clinic blood pressure measurements.\n* Individuals with clinical evidence of any type of active infection including but not limited to HIV, Hepatitis B and\u002For Hepatitis C or patients who are positive on screening test for HIV including antibody or viral load, HBV surface antigen and\u002For HCV antibody. If a patient is positive for HCV antibody, then an HCV viral load will be measured to identify subjects with active infection. Additional tests may include those to screen for active CMV, Infectious Mononucleosis (Epstein-Barr Virus), parvovirus B19 and\u002For COVID-19 infection as per investigator judgement.\n* Individuals with evidence and\u002For documented history of progressive deterioration of liver functions, including the production of clotting factors, removal of toxic metabolic products, bile excretion for at least 6 months, routine hepatic laboratory indices including but not limited to (AST, ALT, or GGT) greater than 3 times the upper limit of normal, and\u002For individuals with a documented history and\u002For diagnosis of chronic liver disease.\n* Individuals with hypertriglyceridemia \\>500 mg\u002FdL.\n* Individuals with a documented history of malignancy (identified in last 5 years or on active therapy at time of screening).\n* Individuals with a documented history of Type I or Type II Diabetes Mellitus\n* Individuals with documented history of hematological conditions commonly associated with FSGS such as sickle cell disease and myeloproliferative disorders.\n* Individuals with a documented history of cardiac conditions commonly associated with FSGS such as congenital cyanotic heart disease. Individuals with a documented history of connective tissue disorders commonly associated with FSGS such as SLE and multiple sclerosis.\n* Individuals taking medications (either currently or within the last 6 months) associated with FSGS such as interferons, lithium, androgenic steroids, sirolimus, heroin, anthracyclines and bisphosphonates.\n* Individuals who are pregnant, will be breastfeeding or refuse birth control anytime during the study.\n* Individuals who have received treatment with another investigational drug, investigational device, or approved therapy for investigational use less than 60 days prior to planned ManNAc dosing.\n* Individuals with hypersensitivity to ManNAc.\n* Individuals who have been treated with ManNAc, sialic acid, IVIG, and\u002For other supplements containing sialic acid (e.g., sialyllactose) less than 60 days prior to planned ManNAc dosing.\n* Individuals who received a renal or any other solid organ and\u002For bone marrow\u002Fstem cell transplantation.\n* Individuals who in the judgment of the investigator, have a condition that places the subject at increased risk for AEs or has any other illness or clinical condition that might confound the results of the study or pose an additional risk in administering study drug to the subject.\n* Patients who need systemic immunosuppressive or glucocorticoid therapy for non-renal indication at any time throughout the trial.\n* Any patient receiving rituximab, cyclosphosphamide and\u002For plasmapheresis within 6 months of screening.\n* A documented history of active alcohol and\u002For substance abuse within the past 2 years.\n* Any patient with collapsing FSGS variant on renal biopsy.\n* Individuals with clinical history suggesting a post-adaptive FSGS such as those with neonatal history of low birth weight, congenital anomaly of the kidney or urinary tract (CAKUT) based on renal ultrasound findings or history of nephrectomy, vesicoureteral reflux or obesity as defined by Body Mass Index (BMI) \\> 40kg\u002Fm\\^2.","115 Years",{"count":429,"type":48},30,[52],"Background:\n\nFocal segmental glomerulosclerosis (FSGS) is a disease that causes scarring in parts of the kidneys that filter waste. This can lead to protein loss in the urine, which can worsen kidney function. The kidneys may fail over time, and dialysis or a kidney transplant may be needed. Other treatments for this disease do not always work and often have adverse effects. Better treatments for FSGS are needed.\n\nObjective:\n\nTo test a study drug (ManNAc) in people with FSGS.\n\nEligibility:\n\nPeople aged 18 years and older with FSGS.\n\nDesign:\n\nParticipants will have 5 to 6 clinic visits over 14 weeks. Two of the visits will require overnight stays for 2 or 3 nights.\n\nManNAc is a white powder that comes in a sachet. It is dissolved in water and taken twice a day by mouth. Participants will take their first dose at the clinic. They will learn how to store ManNAc and prepare each dose. They will record their doses in a diary. They will also write down any adverse effects or troubles they have using the drug at home.\n\nDuring clinic visits, participants will have physical exams with blood and urine tests. They will complete questionnaires about their health, sleep habits, and fatigue symptoms.\n\nDuring overnight visits, participants will also have 24-hour urine collection.\n\nA study team member will call participants 1 week after the first dose to check on their health. Follow-up phone calls will then be every 2 weeks after each clinic visit.\n\nParticipants may meet with a dietitian to discuss nutrition while taking the ManNAc.\n\nParticipants may choose to have genetic tests.",[433],"Focal Segmental Glomerulosclerosis",[435,436,437,438,439],"FSGS","ManNAc","Sialic Acid","Proteinuria","Kidney Disease",{"date":34,"type":35},{"date":308,"type":48},{"date":443,"type":48},"2027-12-01",{"name":445,"class":123},"National Human Genome Research Institute (NHGRI)",{"id":447,"slug":4,"hasResults":11,"nctId":448,"briefTitle":449,"officialTitle":450,"acronym":4,"eligibilityCriteria":451,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":452,"enrollmentInfo":453,"targetDuration":4,"studyType":49,"phases":455,"briefSummary":456,"conditions":457,"keywords":464,"overallStatus":467,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":468,"startDateStruct":469,"completionDateStruct":470,"leadSponsor":472,"locationsCount":124},"100053350","NCT07613099","Fibrotic Disease Activity in Cardiopulmonary Disorders Using 18F-Fibroblast Activation Protein Inhibitor (18F-FAPI-74) PET\u002FCT Imaging","Evaluation of Fibrotic Disease Activity in Cardiopulmonary Disorders Using 18F-Fibroblast Activation Protein Inhibitor (18F-FAPI-74 PET\u002FCT Imaging)","* INCLUSION CRITERIA\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Provision of signed and dated informed consent form\n2. Stated willingness to comply with all study procedures and availability for the duration of the study\n3. Participant \\>=18 years old\n4. Has a specific diagnosis of a cardiopulmonary and\u002For vascular disease that puts them at risk of developing or having developed tissue fibrosis.\n5. Has undergone prior imaging of lungs, heart, and\u002For vasculature with chest CT\n6. Able to lie on the PET\u002FCT scanner for imaging up to 45 minutes.\n7. For females of reproductive potential: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 4 weeks after the end of FAPI-PET\u002FCT or the FDG PET\u002FCT scan.\n8. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner during study participation and for an additional 4 weeks after the end of FAPI-PET\u002FCT or the FDG PET\u002FCT scan.\n9. Ability of subject to understand and the willingness to sign a written informed consent document.\n\nEXCLUSION CRITERIA\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n1. History of allergic reactions attributed to compounds of similar chemical or biologic composition to 18F-FAPI-74 or other agents used in the study.\n2. Uncontrolled intercurrent illness, factors, or social situations that would limit compliance with study requirements\n3. Pregnancy or lactation\n4. Women able to become pregnant or men actively trying to father a child and are unwilling to use an effective form of birth control during the study and 4 weeks after the last 18F-FAPI-74 PET\u002FCT scan or the FDG PET\u002FCT scan.\n5. Subjects with severe claustrophobia unresponsive to oral anxiolytics.\n6. Subjects weighing \\> 350 lbs (weight limit for PET scanner table), or unable to fit within the imaging gantry","100 Years",{"count":454,"type":48},210,[236],"Background:\n\nInjury or diseases of the heart and lung can sometimes cause scar tissue (fibrosis) to build up in those organs. Current imaging scans can see this scar tissue once it has formed, but researchers want to find a way to detect the fibrosis in its earliest stages, while there might still be time to prevent serious damage. A new tracer (a radioactive substance injected during imaging scans) may be able to help.\n\nObjective:\n\nTo test a new tracer (18F-FAPI-74) during imaging scans in people with heart or lung disease.\n\nEligibility:\n\nPeople aged 18 years and older with lung or heart disease that may cause scarring in those organs.\n\nDesign:\n\nParticipants will have 6 clinic visits over 2 years.\n\nParticipants will be screened: They will have blood tests and tests of their heart and lung function. Those with heart disease will have a magnetic resonance imaging (MRI) scan of the heart.\n\nThe study tracer will be used with positron emission tomography (PET)\u002Fcomputed tomography (CT) scans. The study tracer will be injected into a vein in the arm. Participants will lie on a padded bed that slides through a donut-shaped machine.\n\nParticipants will have scans with the study tracer 2 times, 8 to 12 months apart. They will also have standard CT scans and blood tests during these visits. They will also have blood tests at 3 and 6 months between these visits.\n\nParticipants will have a follow-up visit after 18 to 24 months. The study scans, MRI and standard CT scans, and lung function tests may be repeated....",[458,459,460,461,462,463],"Allogeneic Stem Cell Transplantation","Lung Allograft Transplantation","Interstitial Lung Disease","Acute Lung Injury","Pulmonary Arterial Hypertension","Cardiovascular Diseases",[465,466],"Fibroblast activation protein (FAP) FAPI\u002FPET","Fibrotic Disease Activity","NOT_YET_RECRUITING",{"date":34,"type":35},{"date":308,"type":48},{"date":471,"type":48},"2033-05-26",{"name":473,"class":123},"National Heart, Lung, and Blood Institute (NHLBI)",{"id":475,"slug":4,"hasResults":11,"nctId":476,"briefTitle":477,"officialTitle":478,"acronym":479,"eligibilityCriteria":480,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":481,"enrollmentInfo":482,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":484,"conditions":485,"keywords":487,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":491,"startDateStruct":492,"completionDateStruct":494,"leadSponsor":496,"locationsCount":124},"100053346","NCT05991440","Examining Racial and Socioeconomic Differences in Chronic Low Back Pain","Racial and Socioeconomic Differences in Chronic Low Back Pain","ERASED","Inclusion:\n\n* Non-specific cLBP that has persisted for at least 3 months and has resulted in pain on at least half the days in the past 6 months.\n* Age 18 - 85; the lower end of this age range was chosen to capture the growing prevalence of young adults with cLBP, and participants over 85 years are increasingly likely to meet one or more exclusion criteria.\n* Participants report ethnic group as non-Hispanic and racial group as either Black\u002FAfrican American or White\u002FCaucasian.\n\nExclusion:\n\n* Low back pain that is attributable to other factors such as ankylosing spondylitis, infection, malignancy, compression fracture of other trauma.\n* Systemic rheumatic disease\u002Fcondition (e.g. rheumatoid arthritis, systemic lupus erythematosus, fibromyalgia).\n* Any other chronic pain condition that the participant believes to be more prominent or severe than the low back pain.\n* A history of clinically significant surgery to the low back within the past year.\n* Uncontrolled hypertension (i.e. SBP\u002FDBP of \\> 150\u002F95), cardiovascular or peripheral arterial disease.\n* Poorly controlled diabetes (HbA1c \\> 8%).\n* Neurological disease (e.g. Parkinson's, multiple sclerosis, epilepsy).\n* Serious psychiatric disorder requiring hospitalization within the past 12 months or characterized by active suicidal ideation.\n* Diminished cognitive function that would interfere with understanding of study procedures.","85 Years",{"count":483,"type":48},240,"The purpose of this research study is to gain a better understanding of how people's racial background and socioeconomic status (e.g., income, education, and employment) impact their experiences of chronic low back pain (cLBP).",[486],"Chronic Pain",[488,489,490],"lower back pain","Race","Socioeconomic",{"date":34,"type":35},{"date":493,"type":35},"2024-04-22",{"date":495,"type":48},"2028-12-31",{"name":497,"class":224},"Washington University School of Medicine",{"id":499,"slug":4,"hasResults":11,"nctId":500,"briefTitle":501,"officialTitle":502,"acronym":4,"eligibilityCriteria":503,"healthyVolunteers":11,"sex":16,"minAge":504,"maxAge":4,"enrollmentInfo":505,"targetDuration":4,"studyType":49,"phases":507,"briefSummary":508,"conditions":509,"keywords":512,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":514,"startDateStruct":515,"completionDateStruct":517,"leadSponsor":519,"locationsCount":358},"100053345","NCT07160179","Study to Assess the Adverse Events and How Intravitreal ABBV-6628 Moves Through the Body of Adult Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration","Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of ABBV-6628 in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration","Inclusion Criteria:\n\nStage 1 and Stage 2\n\n-Diagnosed with Geographic atrophy (GA) secondary to age-related macular degeneration (AMD) in the study eye.\n\nStage 1\n\n* Foveal or non-foveal GA with total GA lesion area ≥ 0.5 DA (1.25 mm2) in the study eye, as assessed by the investigator at Screening and confirmed by the central reading center prior to Baseline\u002FDay 1\n* Absence of choroidal neovascularization (CNV) in the study eye as assessed by the investigator at Screening and confirmed by the central reading center prior to Baseline\u002FDay 1. In addition, investigators should confirm eligibility prior to treatment administration on Baseline\u002FDay 1.\n\nStage 2\n\n* Non-foveal GA with total lesion area of 1 to 7 DA (2.5 to 17.5 mm2); within 0.5 to 1.5 mm from fovea center in the study eye, as assessed by the investigator at Screening and confirmed by the central reading center prior to Baseline\u002FDay 1.\n* Absence of CNV in both eyes as assessed by the investigator at Screening and confirmed by the central reading center prior to Baseline\u002FDay 1. In addition, investigators should confirm eligibility prior to treatment administration on Baseline\u002FDay 1.\n\nExclusion Criteria:\n\nStage 1 and Stage 2\n\n* History of recurrent or currently active ocular or intraocular inflammation (e.g., uveitis, endophthalmitis) in at least one eye at Screening and Baseline\u002FDay 1.\n* Active periocular, ocular, or intraocular infection in at least one eye at Baseline\u002FDay 1.\n* History or clinical signs of diabetic retinopathy, diabetic macular edema (DME), or any retinal vascular disease other than AMD in at least one eye at Screening and Baseline\u002FDay 1.","50 Years",{"count":506,"type":48},66,[51,52],"Age-related macular degeneration (AMD) is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. Geographic Atrophy (GA) is an advanced form of dry AMD. The purpose of this study is to assess the adverse events and how intravitreal ABBV-6628 moves through the body of adult participants with secondary to age-related macular degeneration\n\nABBV-6628 is an investigational monoclonal antibody fragment being developed for the treatment of geographic atrophy (GA) secondary to (AMD) age-related macular degeneration. Participants in the Stage 1 part will be placed in 1 of 4 groups, called treatment arms. Participants in Stage 2 will be placed into 1 of 2 groups. Each group receives different treatment. Adult participants aged 50 and older years with a diagnosis GA secondary to age-related macular degeneration will be enrolled. Around 66 participants will be enrolled in the study at approximately 27 sites across the US.\n\nParticipants in Stage 1 will be given ABBV-6628 as an intravitreal injection (injection into the jelly-like tissue that fills the eyeball injection) with dose escalation. Participants in Stage 2 will receive ABBV-6628 or SYFOVRE, an approved treatment for geographic atrophy, administered as per the FDA-approved label. The treatment duration is approximately 22 months and 3 months of follow-up.\n\nThere may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular weekly visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.",[510,511],"Geographic Atrophy","Age-Related Macular Degeneration",[510,511,513],"ABBV-6628",{"date":34,"type":35},{"date":516,"type":35},"2025-08-13",{"date":518,"type":48},"2029-10",{"name":520,"class":38},"AbbVie",{"id":522,"slug":4,"hasResults":11,"nctId":523,"briefTitle":524,"officialTitle":524,"acronym":4,"eligibilityCriteria":525,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":526,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":528,"conditions":529,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":533,"startDateStruct":534,"completionDateStruct":536,"leadSponsor":538,"locationsCount":540},"100054277","NCT03664609","Deep Brain Stimulation (DBS) Retrospective Outcomes Study","Inclusion Criteria:\n\n* Must be previously treated with or eligible for implantation with a deep brain stimulation system\n\nExclusion Criteria:\n\n* No Exclusion Criteria",{"count":527,"type":48},5000,"The primary objective of this study is to characterize real-world clinical outcomes of Deep Brain Stimulation (DBS) using retrospective review of de-identified patient records.",[530,531,532],"Parkinson Disease","Essential Tremor","Dystonia",{"date":34,"type":35},{"date":535,"type":35},"2019-03-12",{"date":537,"type":48},"2028-12",{"name":539,"class":38},"Boston Scientific Corporation",19,{"id":542,"slug":4,"hasResults":11,"nctId":543,"briefTitle":544,"officialTitle":545,"acronym":4,"eligibilityCriteria":546,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":547,"targetDuration":4,"studyType":49,"phases":549,"briefSummary":550,"conditions":551,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":554,"startDateStruct":555,"completionDateStruct":557,"leadSponsor":559,"locationsCount":560},"100054084","NCT07003295","Testing the Anti-cancer Drug, Glofitamab, in Patients With Mantle Cell Lymphoma (A Type of Blood Cancer) Whose Disease Returned After CAR-T Cell Therapy","A Phase II Study of Glofitamab for Relapsed\u002FRefractory Mantle Cell Lymphoma in Patients Previously Treated With CD19-Directed CAR T-Cell Therapy","Inclusion Criteria:\n\n* Patients must have histologically or cytologically confirmed diagnosis of mantle cell lymphoma that is relapsed (disease progression after the last treatment regimen) or refractory (failure to achieve a partial or complete response from the last treatment regimen)\n* Patients must have been previously treated with an anti-CD19 CAR T-cell therapy and have failed or been intolerant to Bruton's tyrosine kinase (BTK) inhibition. Both commercial and investigational CAR-T products which target CD19 will be allowed, including dual-targeting products\n* Patients must have at least one bi-dimensionally measurable (defined as at least 1.5 cm) nodal lesion or one bi-dimensionally measurable (at least 1 cm) extranodal lesion, as measured on CT scan\n* Age ≥ 18 years. Because no dosing or adverse event (AE) data are currently available on the use of glofitamab and obinutuzumab in patients \\\u003C 18 years of age, children are excluded from this study\n* Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 60%)\n* Absolute neutrophil count ≥ 1,000\u002FmcL\n* Platelets ≥ 50,000\u002FmcL\n* Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN) or ≤ 3 x institutional ULN if the patient has Gilbert syndrome\n* Aspartate aminotransferase (AST)(serum glutamic oxaloacetic transaminase \\[SGOT\\])\u002Falanine aminotransferase (ALT)(serum glutamic pyruvic transaminase \\[SGPT\\]) ≤ 3 x institutional ULN\n* Creatinine ≤ 1.5 x institutional ULN OR glomerular filtration rate (GFR) ≥ 60 mL\u002Fmin\u002F1.73 m\\^2\n* Patients with human immunodeficiency virus (HIV) infection are eligible if on effective anti-retroviral therapy with undetectable viral load within 6 months\n* Patients with a history of hepatitis B virus (HBV) infection or positive total hepatitis B core antibody (HBcAb) are eligible if the hepatitis B surface antigen (HBsAg) is negative and HBV DNA viral load is undetectable by polymerase chain reaction (PCR) at the time of screening. Such patients must be managed with appropriate anti-viral therapy, if indicated, and must be willing to undergo HBV DNA testing on day 1 of each cycle and every 3 months for at least 12 months after the final cycle of study treatment\n* Patients with a history of hepatitis C virus (HCV) infection or positive HCV antibody are eligible if HCV ribonucleic acid (RNA) viral load is undetectable by PCR\n* Patients with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of progression and patients are asymptomatic from CNS involvement\n* Patients with new, progressive brain metastases (active brain metastases) or leptomeningeal disease are eligible if the treating physician determines that patients are asymptomatic and immediate CNS specific treatment is not required and is unlikely to be required during the first cycle of therapy\n* Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial, such as patients with prostate cancer or breast cancer receiving hormonal therapy\n* The effects of glofitamab and obinutuzumab on the developing human fetus are unknown. For this reason and because glofitamab and obinutuzumab are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 2 months after completion of glofitamab and 6 months after completion of obinutuzumab administration. Women of childbearing potential must use effective contraceptive precautions 2 months after completion of glofitamab treatment and 18 months after the last dose of obinutuzumab treatment\n* Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants\n\nExclusion Criteria:\n\n* Patients who have not recovered from AEs due to prior anti-cancer therapy (i.e., have residual toxicities \\> grade 1) with the exception of alopecia\n* Patients who are receiving any other investigational agents\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to glofitamab and obinutuzumab\n* Pregnant women are excluded from this study because glofitamab and obinutuzumab are agents with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for AEs in nursing infants secondary to treatment of the mother with glofitamab and obinutuzumab, breastfeeding should be discontinued if the mother is treated with glofitamab and obinutuzumab\n* Patients with active CRS or requiring intervention for CRS within 14 days prior to study enrollment\n* Patients with active neurotoxicity or requiring intervention for neurotoxicity within 14 days prior to study enrollment\n* Patients requiring antimicrobial treatment or hospitalization for active infection within 14 days prior to study enrollment for known active bacterial, viral (including severe acute respiratory syndrome coronavirus 2 \\[SARS-CoV-2\\]), fungal, mycobacterial, parasitic, or other infection (excluding fungal infections of nail beds). Infection must be completely resolved prior to initiation of study treatment\n* Patients receiving systemic immunosuppressive medications within 14 days prior to study enrollment, including, but not limited to: prednisone ≥ 20 mg per day, azathioprine, methotrexate, anti-tumor necrosis factor agents, anti-interleukin-6\u002F6R agents, or anti-interleukin 1 agents, are not eligible. The use of inhaled corticosteroids is permitted\n* Patients with known or suspected chronic active Epstein Barr virus (EBV) or cytomegalovirus (CMV) infection\n* Patients with known or suspected history of hemophagocytic lymphohistiocytosis (HLH)\n* Patients with prior treatment with glofitamab or other bispecific antibodies targeting both CD20 and CD3\n* Patients with known history of progressive multifocal leukoencephalopathy\n* Patients with current or past history of CNS disease, such as stroke, epilepsy, CNS vasculitis, or neurodegenerative disease. Patients with a history of stroke who have not experienced a stroke or transient ischemic attack in the past 2 years and have no residual neurologic deficits as judged by the investigator are allowed\n* Patients with the significant cardiovascular disease, including New York Heart Association class III or IV or otherwise symptomatic heart failure (stage C or D), myocardial infarction within the last 3 months, unstable arrhythmias, or unstable angina\n* Patients who have undergone major surgery for non-diagnostic purposes within 4 weeks before first study treatment\n* Patients who have received a live attenuated vaccine within 4 weeks before first study treatment administration or anticipation that such a live, attenuated vaccine will be required during the study\n* Patients with a positive SARS-CoV-2 test 7 days prior to enrollment\n* Patients with current or past history of Waldenström macroglobulinemia\n* Patients with an active autoimmune disease that could be exacerbated by immunotherapy, excluding those with a remote history or well-controlled autoimmune disease",{"count":548,"type":48},20,[52],"This phase II trial tests the safety and side effects of glofitamab and obinutuzumab and how well they work in treating patients with mantle cell lymphoma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory) after receiving CD19-directed chimeric antigen receptor (CAR) T-cell therapy. CAR T-cell therapy is a form of immunotherapy where the immune system cell, T-cell, is changed to attack cancer cells. Glofitamab is a bispecific antibody that can bind to two different antigens at the same time. Glofitamab binds to CD3, a protein found on T cells (a type of white blood cell), and CD20 a protein found on B cells (another type of white blood cell) and some lymphoma cells. This may help the immune system kill cancer cells. Obinutuzumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Giving glofitamab and obinutuzumab may be safe, tolerable, and\u002For effective in treating patients with relapsed or refractory mantle cell lymphoma after receiving CD19-directed CAR T-cell therapy.",[552,553],"Recurrent Mantle Cell Lymphoma","Refractory Mantle Cell Lymphoma",{"date":34,"type":35},{"date":556,"type":35},"2026-07-08",{"date":558,"type":48},"2027-08-31",{"name":177,"class":123},9,{"id":562,"slug":4,"hasResults":11,"nctId":563,"briefTitle":564,"officialTitle":565,"acronym":566,"eligibilityCriteria":567,"healthyVolunteers":11,"sex":16,"minAge":568,"maxAge":4,"enrollmentInfo":569,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":571,"conditions":572,"keywords":574,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":580,"startDateStruct":581,"completionDateStruct":583,"leadSponsor":585,"locationsCount":587},"100053340","NCT07144137","A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA)","A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)","Pre-GAIN","Inclusion:\n\n1. Participants must be aged ≥55 years, at the time of signing the informed consent at the screening visit.\n2. Participants with bilateral GA secondary to AMD as confirmed by the Central Reading Center using FAF and\u002For OCT images with at least 1 eye having a total GA lesion area must be between 1.25 mm2 and 17.5 mm2 inclusive, determined by FAF images taken at the screening visit.\n3. Best-corrected visual acuity (BCVA) in both eyes should be sufficient to ensure navigational vision (defined as 20\u002F400 or better for purposes of this study).\n4. BCVA between 20 and 75 letters and LLVA\\>0 letters using an ETDRS chart.\n5. Mean retinal sensitivity as measured by microperimetry using the study grid must be equal to or greater than 5 dB.\n6. Able and willing to provide signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol, and in the opinion of the investigator, is able to perform all study assessments.\n\nExclusion criteria:\n\n1. Macular atrophy secondary to any condition other than AMD in the study eye.\n2. Any pathology of the macula other than GA secondary to AMD and other changes consistent with early, intermediate or atrophic AMD in the study eye.\n3. Evidence of prior or current Choroidal Neovascularization (CNV), also known as wet-AMD, in either eye.\n4. Atrophic retinal disease of causality other than AMD including monogenetic macular dystrophies (incorporating pattern dystrophy), myopia-related maculopathy and Stargardt disease in the study eye.\n5. A history of vitrectomy in the study eye.\n6. Any prior treatment for AMD or any prior intravitreal treatment for any indication in the study eye, except oral supplements of vitamins and minerals such as the age-related eye disease study (AREDS) formula.\n7. Any intraocular surgery (except cataract surgery within 6 months of enrollment) or thermal laser within 3 months of screening, or any ophthalmic condition that may require surgery during the study.\n8. Any macular laser, macular surgery or retinal surgery at any time point in the study eye.\n9. Any ocular or periocular infection in the 12 weeks prior to screening.\n10. History of uveitis or endophthalmitis in either eye.\n11. Any sign of diabetic retinopathy in either eye, or HbA1c \\>8, in the 12 months prior to enrollment.\n12. High myopia or hyperopia (≥6 diopter) in the study eye.\n13. Uncontrolled IOP measurement of ≥25 mmHg for \\>1 month despite being on 2 or more ocular hypotensive agents, or IOP \\>21 mm Hg in the presence of C\u002FD asymmetry of \\>0.3 per the reading center; or glaucomatous damage to the optic nerve or visual field.\n14. Retinal disease other than AMD or other ocular disorders which may cause safety concerns per the judgment of the investigator; however, benign conditions of the vitreous or peripheral retina are not exclusionary (i.e., paving stone degeneration).\n15. Any ophthalmologic condition that reduces the clarity of the media and that, in the opinion of the investigator interferes with ophthalmologic examination (e.g., cataract or corneal abnormalities) either at the time of enrollment or during the 2-year post dosing follow-up period; or prevents adequate imaging of the retina judged by the site or CRC.\n16. Aphakia or absence of the posterior capsule. Previous violation of the posterior capsule is also excluded unless it occurred as a result of yttrium aluminum garnet (YAG) laser posterior capsulotomy in association with prior posterior chamber intraocular lens implantation and at least 60 days prior to baseline.\n17. Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the treatment period unlikely, or in general a poor medical risk because of other systemic diseases or active uncontrolled infections, including but not limited to immunodeficiency state, autoimmune diseases, malignancy other than prostate or basal cell carcinoma, connective tissue disorders and collagen vascular disorders.\n18. Participation in an interventional clinical study, or use of any experimental treatment for AMD or any investigational product within 6 months or 5 half-lives of the active ingredient (whichever is longer) prior to screening.\n19. History of abnormal laboratory tests (e.g. hematology, serum chemistry, renal or liver function tests, or infectious disease screen) that in the opinion of the investigator is clinically significant at the time of enrollment and not suitable for study participation.\n20. Hypersensitivity to medications used in (standard of care) study procedures.\n21. Situations where, in the opinion of the investigator, the risk of harm to a participant outweighs any potential benefits from study participation (these could include participants with conditions such as brittle diabetes or advanced osteoporosis as examples).\n22. History or current use of medications associated with macular changes, retinal dysfunction or optic nerve damage. These include but are not limited to the following prohibited concomitant medications:\n23. Any previous gene or cell therapy (ophthalmic or systemic),\n24. Chloroquine (where taken for \\>30 days cumulative across participant's lifetime), hydroxychloroquine, platinum containing medications, ethambutol, tamoxifen, pentosan, chronic use of phosphodiesterase inhibitors, and GLP-1 antagonists in the setting of a crowded optic disc.","55 Years",{"count":570,"type":48},75,"This is a non-interventional, observational study to provide insights into the short-term progression of GA secondary to AMD in participants aged ≥55 years.\n\nThis is a multi-center, non-interventional, observational study which aims to identify participants who have progressive GA to allow quantification of structural and functional parameters that characterize the progression of GA, and to investigate whether these correlate with genetic or lifestyle factors.",[573],"Geographic Atrophy Secondary to Age-related Macular Degeneration",[575,576,577,578,579],"GA","AMD","Geographic atrophy","Dry AMD","Age-related macular degeneration",{"date":34,"type":35},{"date":582,"type":35},"2025-07-30",{"date":584,"type":48},"2027-06-30",{"name":586,"class":38},"Complement Therapeutics",13,{"id":589,"slug":4,"hasResults":11,"nctId":590,"briefTitle":591,"officialTitle":592,"acronym":4,"eligibilityCriteria":593,"healthyVolunteers":594,"sex":16,"minAge":595,"maxAge":105,"enrollmentInfo":596,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":598,"conditions":599,"keywords":604,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":611,"startDateStruct":612,"completionDateStruct":4,"leadSponsor":614,"locationsCount":124},"100053339","NCT01386437","Natural History of Individuals With Immune System Problems That Lead to Fungal Infections","The Natural History and Pathogenesis of Human Fungal Infections","* INCLUSION CRITERIA:\n\nPatients:\n\nPatients with or without inherited or acquired abnormalities of immune function manifesting mucocutaneous and\u002For invasive fungal infections are eligible for screening and assessment under this protocol. Specifically, patients must meet all the following inclusion criteria in order to participate in this study:\n\nAdults or children (regardless of age, sex, or ethnicity\u002Frace) with a known or yet uncharacterized inherited immunodeficiency and a definitively diagnosed mucocutaneous or invasive fungal infection.\n\nOR\n\nAdults or children (regardless of age, sex, or ethnicity\u002Frace) with acquired immunodeficiency and a severe, unusual, persistent or treatment-refractory chronic mucocutaneous fungal infection.\n\nOR\n\nAdults or children (regardless of age, sex, or ethnicity\u002Frace) with acquired immunodeficiency and a possible, probable or proven invasive fungal infection (European Organization for Research and Treatment of Cancer \u002F Mycoses Study Group criteria).\n\nOR\n\nAdults or children (regardless of age, sex, or ethnicity\u002Frace) with a well-documented prior, unusual, severe, persistent, or treatment-refractory mucocutaneous or invasive fungal infection(s), who have clinically recovered from the fungal infection.\n\nOR\n\nAdults or children (regardless of age, sex, or ethnicity\u002Frace) with confirmed or suspected autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED) who have not yet developed CMC.\n\nOngoing care by a referring\u002Fprimary care physician (inside or outside NIH).\n\nWilling to allow storage of blood and tissue samples for future analyses.\n\nWilling to allow genetic testing from blood, body fluids or tissue specimens.\n\nWilling to have HIV testing\n\nAble to provide informed consent or be accompanied by a parent(s)\u002Flegal guardian(s) or legally authorized representative (LAR) who is able to provide informed consent.\n\nNo children under the age of 2 years will be seen at the Clinical Center, however they will be able to participate via mail-in specimens\n\nPatient Relatives:\n\nIndividuals (regardless of age, sex, or ethnicity\u002Frace) who are genetically related to the patient (e.g., mother, father, siblings, children) may be recruited to establish the genetic origin of immune defects that may be identified in the study patients at the discretion of the PI. Relatives must meet all the following inclusion criteria in order to participate in this study:\n\nWilling to allow storage of blood and tissue samples for future analyses.\n\nWilling to allow genetic testing from blood, body fluids or tissue specimens.\n\nWilling to have HIV testing\n\nAble to provide informed consent or, if younger than 18, be accompanied by a parent(s)\u002Flegal guardian(s) who is able to provide informed consent.\n\nHealthy Volunteers:\n\nHealthy adults regardless of sex, and ethnicity\u002Frace between the ages of 18 and 85 years old may be eligible to participate in this study. Healthy volunteers must meet all the following inclusion criteria in order to participate in this study:\n\nWilling to allow storage of blood and tissue samples for future analyses.\n\nWilling to allow genetic testing from blood, body fluids or tissue specimens.\n\nWilling to have HIV testing\n\nAble to provide informed consent\n\nNIH employees are eligible\n\nEXCLUSION CRITERIA:\n\nPatients:\n\nA patient will not be eligible if he\u002Fshe has any of the following:\n\nAny condition which, in the investigator's opinion, may interfere with the evaluation of a co-existing abnormality of immunity that is the subject of study under this protocol.\n\nAny condition which, in the investigator's opinion, places the patient at undue risk by participating in the study.\n\nUnwillingness to undergo testing or procedures associated with this protocol.\n\nHemoglobin of \\\u003C 7 gm\u002FdL. If a patient is enrolled solely to obtain left-over pathology specimens, saliva, a buccal sample, skin swab, vaginal swab, or a stool sample, this exclusion criteria will not apply as there will be no blood withdrawal.\n\nPatient Relatives:\n\nA genetically related relative will not be eligible for this study if he\u002Fshe has any condition which, in the investigator's opinion, may interfere with the evaluation of an immune system abnormality that is the subject of study under this protocol.\n\nHealthy Volunteers:\n\nA healthy volunteer will not be eligible if he\u002Fshe has any of the following:\n\nHIV infection.\n\nHistory of recurrent or severe infections.\n\nHistory of an underlying malignancy or receipt of cancer chemotherapy within the past 5 years\n\nReceipt of systemic corticosteroids or other systemic immunosuppressants or immunomodulators within the past 30 days\n\nPregnancy or lactating\n\nHistory of heart, lung, kidney disease, or bleeding disorders.\n\nAny condition which, in the investigator's opinion, may interfere with the comparison of clinical specimens against those obtained from affected subjects.",true,"1 Day",{"count":597,"type":48},1200,"Background:\n\n\\- The immune system is made up of special cells, tissues, and organs that fight infections. Problems with this system may lead to frequent, severe, or unusual fungal infections. These infections are often difficult to treat. Researchers want to collect blood and tissue samples from people who have unusual, persistent or severe fungal infections or immune problems that increase the risk of these infections.\n\nObjectives:\n\n\\- To collect medical information and samples for a long-term study of people with immune system problems that lead to fungal infections.\n\nEligibility:\n\n* People with a history of fungal infections caused by immune system problems.\n* Parents, children, and siblings of this group.\n* Healthy volunteers not related to the first two groups.\n\nDesign:\n\n* This long-term study may last for up to 25 years. Those in the study may need to provide new information about every 6 months. The procedures for each person may vary with the particular diagnosis and the extent of fungal infection. Healthy volunteers may have only one or two visits.\n* At the first visit, those in the study will have a full medical history and physical exam. They will also provide blood.\n* Research procedures may include the following:\n* Saliva, urine or stool testing\n* Mouthwash collection for DNA testing\n* Collection of cheek cells, nail clippings, or vaginal fluid\n* Tests of leftover tissue or body fluid from previous medical procedures\n* Skin or oral mucous membrane biopsy\n* Collection of white blood cells\n* Followup visits will involve a physical exam and updated medical history. Blood, saliva, urine, or nail clipping samples may be taken for ongoing studies. Any additional tests or exams required by the study doctors may also be done.\n* Participants may withdraw from the study pool at any time.",[600,601,602,603],"Chronic Mucocutaneous Candidiasis","Invasive Aspergillosis","CARD9","APECED",[605,606,607,608,116,609,610],"Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)","Fungal Infections","Molds","Autoimmune Polyendocrinpathy Syndrome (APS) Type 1","Mucocutaneous Fungal Infection","Invasive Fungal Infection",{"date":34,"type":35},{"date":613,"type":35},"2012-11-05",{"name":122,"class":123},{"id":616,"slug":4,"hasResults":11,"nctId":617,"briefTitle":618,"officialTitle":619,"acronym":4,"eligibilityCriteria":620,"healthyVolunteers":11,"sex":16,"minAge":621,"maxAge":481,"enrollmentInfo":622,"targetDuration":4,"studyType":49,"phases":624,"briefSummary":625,"conditions":626,"keywords":628,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":631,"startDateStruct":632,"completionDateStruct":634,"leadSponsor":635,"locationsCount":637},"100053325","NCT06404268","Facilitating Neuroplastic Changes of Acute Stroke Survivors","Facilitating Neuroplastic Changes of Acute Stroke Survivors With Severe Hemiplegia","Inclusion Criteria:\n\n* Acute first time unilateral hemispheric stroke (hemorrhagic or ischemic stroke, 24 hours after admission to 1 month post-stroke at the start of the proposed treatment)\n* Hemiplegia or hemiparesis\n* 0≤Manual Muscle Testing (MMT)\\\u003C=2\n* Age 30-85\n* Ankle impairments including stiff calf muscles and\u002For inadequate dorsiflexion\n\nExclusion Criteria:\n\n* Medically not stable\n* Associated acute medical illness that interferes with ability to training and exercise\n* No impairment or very mild ankle impairment of ankle\n* Severe cardiovascular problems that interfere with ability to perform moderate movement exercises\n* Cognitive impairment or aphasia with inability to follow instructions\n* Severe pain in legs\n* Severe ankle contracture greater than 15° plantar flexion (when pushing ankle to dorsiflexion)\n* Pressure ulcer, recent surgical incision or active skin disease with open wounds present below knee","30 Years",{"count":623,"type":48},68,[51,52],"This project will develop a wearable rehabilitation robot suitable for in-bed acute stage rehabilitation. It involves robot-guided motor relearning, passive and active motor-sensory rehabilitation early in the acute stage post-stroke including patients who are paralyzed with no motor output. The early acute stroke rehabilitation device will be evaluated in this clinical trial.",[627],"Stroke",[627,629,630],"Paraplegia","Acute",{"date":34,"type":35},{"date":633,"type":35},"2025-07-01",{"date":495,"type":48},{"name":636,"class":224},"University of Maryland, Baltimore",3,{"id":639,"slug":4,"hasResults":11,"nctId":640,"briefTitle":641,"officialTitle":641,"acronym":4,"eligibilityCriteria":642,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":643,"targetDuration":4,"studyType":108,"phases":4,"briefSummary":645,"conditions":646,"keywords":649,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":656,"startDateStruct":657,"completionDateStruct":658,"leadSponsor":660,"locationsCount":124},"100053322","NCT07314736","Stakeholders of Rare Diseases Informing Values In Neuroethics","Inclusion Criteria:\n\nGroup 1 (Parental Caregiver and Patient Participants)\n\n* Parental\u002Fprimary caregiver with a child who has a genetic diagnosis of an ultrarare disorder with pediatric onset, or a clinical diagnosis with a suspected genetic etiology.\n* Child is under 21 years of age at the time of enrollment.\n* Child has an expected survival of at least one year following study enrollment.\n* Patients (age ≤ 25 years) with a genetic diagnosis of an ultrarare disorder with pediatric onset, or clinical diagnosis with suspected genetic etiology.\n* Willingness to provide verbal informed consent (or assent, as appropriate) to participate\n\nGroup 2 (Other Family)\n\n* Family member of a Group 1 participant who plays an active role in the child's life or care.\n* Includes siblings (≥ 13 years of age), grandparents, or other non-primary caregivers directly affected by the child's diagnosis.\n* Demonstrated familiarity with the child's medical and family experience.\n* Willingness to provide verbal informed consent (or assent, as appropriate) to participate.\n\nGroup 3 (Non-Family Stakeholders)\n\n* Individuals currently engaged, or recently active, in clinical care, research, advocacy or policy work related to pediatric-onset rare genetic disorders.\n* May include clinicians (e.g., neurologists, genetic counselors, nurses, child-life specialists, home-health staff), members of patient-advocacy organizations, institutional-review-board (IRB) members, payers, sponsors, funders, or representatives of hospital systems or regulatory agencies.\n* Willingness to provide verbal informed consent to participate in semi-structured interviews or focus groups\n\nExclusion Criteria:\n\n* Limited English proficiency\n* Unable to complete the survey materials or complete the interviews in English.\n* Inability or unwillingness of research participant to give verbal informed consent (in English)\n* Condition or chronic illness, which in the opinion of the PI\u002FCo-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).",{"count":644,"type":48},385,"The purpose of this research study is to learn more about the perspectives of key stakeholders-patients, families, healthcare providers, and researchers-on the ethical challenges of small-scale, personalized treatment trials for rare neurological diseases (RND).",[647,648],"Rare Disorder","Disorder, Neurologic",[650,651,652,653,654,655],"Rare Neurological Disorders (RND)","Stakeholders","Patient Participant","Caregiver","Other Family","Non-Family Stakeholder",{"date":34,"type":35},{"date":556,"type":35},{"date":659,"type":48},"2031-01",{"name":661,"class":224},"St. Jude Children's Research Hospital",""]