[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"National Heart, Lung, and Blood Institute (NHLBI)\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":542},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,68,0,25,[9,49,74,92,113,139,164,190,204,215,224,234,251,278,288,311,337,360,384,407,428,451,479,500,520],{"id":10,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":33,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100053350",false,"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","ALL","18 Years","100 Years",{"count":20,"type":21},210,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","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....",[27,28,29,30,31,32],"Allogeneic Stem Cell Transplantation","Lung Allograft Transplantation","Interstitial Lung Disease","Acute Lung Injury","Pulmonary Arterial Hypertension","Cardiovascular Diseases",[34,35],"Fibroblast activation protein (FAP) FAPI\u002FPET","Fibrotic Disease Activity","NOT_YET_RECRUITING","2026-07-10",{"date":39,"type":40},"2026-07-13","ACTUAL",{"date":42,"type":21},"2026-07-16",{"date":44,"type":21},"2033-05-26",{"name":46,"class":47},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":50,"slug":4,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":58,"briefSummary":60,"conditions":61,"keywords":63,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":68,"startDateStruct":69,"completionDateStruct":70,"leadSponsor":72,"locationsCount":73},"100053930","NCT06948097","Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Trial to Evaluate the Safety and Tolerability of Fostamatinib in Lung Transplant Patients With Donor-Specific Antibodies","Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Phase 1, Double-Blind, Randomized, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of Fostamatinib in Lung Transplant Patients With Donor-Specific Antibodies.","* INCLUSION CRITERIA:\n\nSubjects who do not meet any of the following criteria during screening will not be randomized but will be counted toward study accrual. Screen failures may be rescreened at a later time if the reason for screening failure is revised. In order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n* First time LT recipients\n* Have provided signed written informed consent, prior to performing any study procedure, including screening procedures.\n* Age greater than or equal to 18 years\n* Patients who are positive for de novo DSA that is first reported on or after day 21 post-transplantation in a recipient with no prior history of the same DSA specificity, and who sign informed consent within 30 days of positive test results.\n* No prior demonstration of DSA specificity at any time point preceding the qualifying positive test (including pre-transplant and post-transplant testing).\n* Demonstrate no clinical or spirometry signs of allograft dysfunction at the time of enrollment.\n* Have adequate liver function, as defined by:\n\n  * Serum aspartate aminotransferase (AST) \\\u003C=1.5 x Upper Limit of Normal (ULN) (unless the increased AST is assessed by the Investigator as due to hemolysis) and alanine aminotransferase (ALT) \\\u003C=1.5 x ULN.\n  * Absolute neutrophil count \\>=1.0 x 10\\^9\u002FL.\n  * Hemoglobin \\>= 9 g\u002FdL\n* For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception).\n* For women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use 2 effective forms of contraception from the time of giving informed consent, during the study, and for 28 days following the last dose of study treatment. An effective form of contraception is defined as hormonal oral contraceptives, injectables, patches, intrauterine or subdermal contraceptive implants, and barrier methods.\n* Be willing to comply with all study procedures for the duration of the study.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n* Have a significant medical condition that confers an unacceptable risk to participating in the study, and\u002For that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:\n\n  * History of neutropenia (benign ethnic neutropenia and\u002For acquired neutropenia) within 90 days of screening.\n  * History of posterior reversible encephalopathy syndrome (PRES)\n  * History of poorly controlled hypertension or hypertensive crises (defined as systolic blood pressure \\>=180 mmHg or average diastolic blood pressure \\>=120 mmHg based on an average of 3 blood pressure readings despite adequate antihypertensive therapy) unless controlled for \\>90 days prior to enrollment\n  * History of positive post-transplant active hepatitis C and\u002For hepatitis B viral infection.\n  * History of drug-induced cholestatic hepatitis within 90 days of screening.\n  * History of any primary malignancy within the last 5 years, with the exception of: curatively treated non-melanomatous skin cancer; curatively treated cervical or breast carcinoma in situ; or other primary tumor treated with curative intent, no known active disease present, and no treatment administered during the last 5 years.\n  * Testing positive for human immunodeficiency virus 1 or 2 Ab with evidence for ongoing active infection (i.e., CD 4 count \\\u003C400\u002Fmicroliter and viral load \\>100,000 copies\u002Fml) on antiretroviral therapy.\n  * Current or recent history of psychiatric disorder within the last 90 days that, in the opinion of the Investigator or Medical Monitor, could compromise the ability of the subject to cooperate with study visits and procedures.\n  * Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo.\n  * Having had a prior lung or any organ transplant.\n  * Currently pregnant or lactating.\n  * Estimated glomerular filtration (eGFR) rate less than 30 mL\u002Fmin.\n  * Any grade 3 diarrhea within 90 days of screening.\n* Subjects on strong CYP3A4 inducers. Glucocorticoids are standard transplant therapies and are not excluded. Relevant strong inducers include apalutamide, carbamazepine, encorafenib, enzalutamide, fosphenytoin, lumacaftor, lumacaftor-ivacaftor, mitotane,\n\nphenytoin, rifampin (rifampicin) - (https:\u002F\u002Fwww.uptodate.com\u002Fcontents\u002Fimage?imageKey=CARD%2F76992)\n\n-Subjects who have received prior treatment for DSA within 6 months of screening. Patients on an on-going therapy for first-time DSA are eligible, if screening is performed within 30 days of positive DSA results.","99 Years",{"count":57,"type":21},30,[59],"PHASE1","Background:\n\nPeople who have lung transplants often survive 6 or 7 years. But some people develop donor-specific antibodies (DSA) after their transplants; antibodies are proteins that attack foreign invaders in the body. Antibodies typically kill viruses and other agents that can cause disease. But when the antibodies attack a transplanted organ, they can cause the body to reject the new tissues. People who develop DSA after a transplant have a higher risk of death within 1 year.\n\nObjective:\n\nTo test a drug called fostamatinib in people who develop DSA after a lung transplant.\n\nEligibility:\n\nAdults aged 18 and older who developed DSA after a lung transplant.\n\nDesign:\n\nParticipants will continue with their standard care after a transplant.\n\nFostamatinib is a pill taken by mouth. Some participants will take the study drug along with their standard care; others will take a placebo. A placebo is a pill that looks just like the real drug but contains no medicine. All participants will take 1 pill per day for 2 weeks. Then they will take 2 pills per day for the next 6 weeks.\n\nParticipants will have clinic visits every 2 weeks while taking their pills. They will have a physical exam, with blood and urine tests, during each visit.\n\nIf participants have fluid samples collected from their airways during their standard treatment, some extra fluid may be collected for this study.\n\nParticipants will have a follow-up visit 4 weeks after they stop taking their pills.",[62],"Lung Transplantation",[64,65,66,67],"Fostamatinib","Lung Transplant","Donor-specific antibodies","Antibody-mediated rejection",{"date":39,"type":40},{"date":42,"type":21},{"date":71,"type":21},"2028-07-14",{"name":46,"class":47},5,{"id":75,"slug":4,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":4,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":80,"enrollmentInfo":81,"targetDuration":4,"studyType":22,"phases":82,"briefSummary":83,"conditions":84,"keywords":4,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":87,"startDateStruct":88,"completionDateStruct":89,"leadSponsor":91,"locationsCount":48},"100053404","NCT07566494","Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease","A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease","* INCLUSION CRITERIA:\n\nSubjects will enroll onto the study and undergo screening. Subjects who do not meet any of the following criteria during screening will not receive the study intervention but will be counted toward study accrual. Screen failures may be rescreened at a later time. In order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n* Have provided signed written informed consent prior to performing any study procedure, including screening procedures.\n* Age between 18-70 years. Adults over 70 years of age will be excluded from this study due to an increased risk of more severe disease and higher prevalence of multiple comobodities.\n* Unequivocal diagnosis of SCD (HbSS, HbSC, HbSbeta+ or HbSbeta\\^0) confirmed by hemoglobin electrophoresis performed on patients at least 60 days after a blood transfusion if previously transfused.\n* No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC)\n* Serum aspartate aminotransferase (AST) \\\u003C= 1.5 x Upper Limit of Normal (ULN) (unless the increased AST is assessed by the Investigator as due to hemolysis) and alanine aminotransferase (ALT) \\\u003C=1.5 x ULN.\n* Hemoglobin \\>= 7 g\u002FdL\n* Serum creatinine \\\u003C=1.25 x ULN. If serum creatinine is \\>1.25 x ULN, then glomerular filtration rate (based on creatinine) must be \\>=60 mL\u002Fmin.\n* If on hydroxyurea, participant must have been on stable dose of hydroxyurea (defined as a stable dose for at least 90 days and inclusive of dose modifications for hematological toxicity per PI discretion) prior to signing consent.\n* If on L-glutamine and\u002For crizanlizumab, participant must have been on a stable dose for ast least 90 days prior to signing consent.\n* Agree to abstain from taking any other products\u002Fsupplements containing turmeric or curcumin until all study visits have been completed (oral, topical, etc.)\n* For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception).\n* Women of reproductive potential be abstinent as part of their usual lifestyle, or agree to use 2 effective forms of contraception from the time of giving informed consent, during the study, and for 28 days following the last dose of study treatment. An effective form of contraception is defined as hormonal oral contraceptives, injectables, patches, intrauterine or subdermal contraceptive implants, and barrier methods.\n* Be willing to comply with all study procedures for the duration of the study.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n* Vaso-occlusive crisis requiring parenteral treatment within 14 days of signing consent.\n* Three or more vaso-occlusive crises in the 12 months prior to screening that resulted in receiving treatment in an urgent care, outpatient infusion center\u002Fday-clinic, emergency department or inpatient setting.\n* Have a significant medical condition that confers an unacceptable risk to participating in the study, and\u002For that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:\n\n  * Poorly controlled hypertension (defined as systolic blood pressure \\[BP\\] \\>150 mmHg or diastolic BP \\>90 mmHg) refractory to medical management.\n  * History of recent (within 24 weeks prior to signing consent) decompensated congestive heart failure; myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism.\n  * Clinically symptomatic cholelithiasis or cholecystitis. Prior cholecystectomy is not exclusionary. Subjects with symptomatic cholelithiasis or cholecystitis may be rescreened once the disorder has been treated and clinical symptoms have resolved.\n  * History of drug-induced cholestatic hepatitis.\n  * Iron overload sufficiently severe to result in a clinical diagnosis by the Investigator of cardiac (e.g., clinically significant impaired left ventricular ejection fraction), hepatic (e.g., fibrosis, cirrhosis), or pancreatic (e.g., diabetes) dysfunction.\n  * Positive test for hepatitis B surface antigen or hepatitis C virus (HCV) antibody (Ab) with signs of active hepatitis B or C virus infection. If the subject is positive for HCVAb, a reverse transcriptase-polymerase chain reaction test will be conducted. Subjects with hepatitis C may be rescreened after receiving appropriate hepatitis C treatment.\n  * Active infection requiring any use of systemic antimicrobial agents (parenteral or oral) or Grade \\>=3 in severity (per National Cancer Institute Common Terminology Criteria for Adverse Events) within 4 weeks prior to signing consent.\n  * Diabetes mellitus judged to be under poor control by the Investigator or requiring \\>3 antidiabetic agents, including insulin (all insulins are considered 1 agent); use of insulin per se is not exclusionary.\n  * History of any primary malignancy, with the exception of: curatively treated nonmelanomatous skin cancer; curatively treated cervical or breast carcinoma in situ; or other primary tumor treated with curative intent, no known active disease present, and no treatment administered during the last 3 years.\n  * Current or recent history of psychiatric disorder that, in the opinion of the Investigator or Medical Monitor, could compromise the ability of the subject to cooperate with study visits and procedures.\n  * Have had a prior bone marrow or stem cell transplant.\n* Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo.\n* Taking nitroglycerin, or any other nitrate-enhancing drug.\n* Have exposure to any investigational drug, device, or invasive procedure within 12 weeks prior to signing consent. All non-investigational invasive procedures within 12 weeks of signing consent may be considered as a potential exclusion criteria per the PI's discretion.\n* Currently pregnant or breastfeeding.\n* Currently receiving hematopoietic stimulating agents (e.g., erythropoietins, granulocyte colony stimulating factors, thrombopoietins) that have not been stopped for a duration of at least 90 days prior to signing consent.\n* Have a history of allergy to turmeric, curcuminoids, or formulations thereof.","90 Years",{"count":7,"type":21},[59],"Background:\n\nSickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen. Research has shown that curcumin, a natural compound found in turmeric, can improve the health of red blood cells in people with SCD. But the body cannot absorb curcumin well when it is taken by mouth. Researchers want to know if a skin gel (VAS-101) can help the body better absorb curcumin. VAS-101 contains curcumin, which comes from turmeric.\n\nObjective:\n\nTo test VAS-101 in people with stable SCD.\n\nEligibility:\n\nPeople aged 18 to 70 years with stable SCD.\n\nDesign:\n\nPeople who want to join the study will be screened with physical exam with blood tests to see if they are eligible. If they qualify, they can enroll in the study.\n\nParticipants will have up to 15 clinic visits over about 14 weeks. Some may need to stay overnight in the hospital for up to 2 days to make it easier to collect blood samples after the gel is applied.\n\nFor 6 weeks, a special gel called VAS-101 will be put on the forearms in the clinic two times a week. Staff will rub the gel into the skin for at least 30 seconds using a soft toothbrush. The area stays uncovered for at least 10 minutes, then is covered with a bandage or sleeve. After 24 hours, the dressing can be removed and the skin can be washed.\n\nSome visits will include blood tests and other exams. On three visits, a test called near infrared spectroscopy (NIRS) will be done. For this test, probes are placed on the skin to measure blood flow, oxygen levels, and the makeup of skin and muscle. A blood pressure cuff is used to squeeze the arm for up to 5 minutes.\n\nThe last clinic visit will happen about 4 weeks after the final gel application....",[85],"Sickle Cell Disease, Hemolytic Anemia","RECRUITING",{"date":39,"type":40},{"date":42,"type":21},{"date":90,"type":21},"2027-04-27",{"name":46,"class":47},{"id":93,"slug":4,"hasResults":11,"nctId":94,"briefTitle":95,"officialTitle":95,"acronym":4,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":97,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":100,"conditions":101,"keywords":103,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":107,"startDateStruct":108,"completionDateStruct":109,"leadSponsor":111,"locationsCount":112},"100053457","NCT05372627","NHLBI-Emory Advanced Cardiac CT Reconstruction","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Male or female, aged \\>= 18 years\n2. Undergoing clinically-indicated contrast-enhanced time-resolved cardiac CT for structural heart disease.\n3. 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. Unable to complete contrast-enhanced cardiac CT acquisition for any reason\n2. Unwilling to authorize future use of their imaging data for research",{"count":98,"type":21},1000,"OBSERVATIONAL","Background:\n\nDoctors use computed tomography (CT) to get detailed pictures of the heart. CT uses x-rays to gather raw data. Computers assemble this data to make the images doctors look at. A new computer technique can make higher resolution images from the same CT scans. In this natural history study, researchers will take normal CT images of the heart. They will compare those images to super high-resolution (super high-res) images made with a super-computer.\n\nObjective:\n\nTo improve the quality of heart CT scans by using new methods to create the images.\n\nEligibility:\n\nPeople aged 18 years or older who need a CT scan for heart disease.\n\nDesign:\n\nParticipants will have a normal CT scan. A substance will be injected through a tube in their arm. They will lie on a table in a large, donut-shaped machine. An X-ray tube will move around their body, taking many pictures.\n\nResearchers will use the normal CT scans to create super high-res images. They may do this at the NIH. They may also send the images to the company that made the CT scanner. Participants personal information will be removed before images are sent to the company. The personal information will be replaced by a code.\n\nThe super high-res images will be returned to the NIH.\n\nSome information will be collected from participants medical records. Researchers will compare the normal scans to the super high-res images.\n\nParticipants' own doctors will also have a chance to see the super high-res images.\n\nParticipants' CT pictures will be stored and used for future NIH research.",[102],"Structural Heart Disease",[104,105,106],"Heart Disease","Image","Natural History",{"date":39,"type":40},{"date":42,"type":21},{"date":110,"type":21},"2031-01-30",{"name":46,"class":47},2,{"id":114,"slug":4,"hasResults":11,"nctId":115,"briefTitle":116,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":118,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":120,"conditions":121,"keywords":127,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":133,"startDateStruct":134,"completionDateStruct":136,"leadSponsor":138,"locationsCount":48},"100053610","NCT05213572","Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Stated willingness to comply with all study procedures and availability for the duration of the study\n2. Male or female, aged \\>=18 years\n3. Patients with current or previous diagnosis of any type of SCD (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal) who:\n\n   1. plan to receive an allogeneic HCT or gene therapy,\n\n      Or\n   2. are receiving non-curative treatment (standard of care or investigational) and who do not plan to receive an allogeneic HCT or gene therapy\n4. Ability to travel to the NIH Clinical Center\n5. Ability of subject to understand a written informed consent document.\n6. At least one of the following eligibility criteria:\n\n   * History of stroke or abnormal transcranial doppler examination (\\>= 200 m\u002Fs)\n   * History of SCD-related renal insufficiency defined as a creatinine level \\>=1.3 mg\u002FdL and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \\\u003C 50mL\u002Fmin\n   * Tricuspid regurgitant velocity \\>= 2.5 m\u002Fs\n   * Recurrent tricorporal priapism defined as at least 2 episodes of an erection lasting \\> 4 hours involving the corpora cavernosa and corpus spongiosa\n   * SCD-associated liver disease defined as EITHER ferritin \\> 1000 mcg\u002FL OR direct bilirubin \\> 0.4 mg\u002FdL\n   * \\> 1 hospitalization per year for vaso-occlusive crises while on a therapeutic dose of hydroxyurea\n   * Any acute chest syndrome while on a therapeutic dose of hydroxyurea\n   * Osteonecrosis of 2 or more joints\n   * Red cell alloimmunization\n\nEXCLUSION CRITERIA:\n\nAll individuals meeting any of the exclusion criteria at baseline will be excluded from study participation.\n\n1. Prior transplantation (including but not limited to HSCT and kidney transplant)\n2. Pregnant or breastfeeding\n3. Patients with allergy to iodine or iodinated contrast solutions will not undergo Iothalamate or Iohexal GFR clearance testing but can undergo the other deep phenotype testing\n4. Implanted metal object that is not compatible with MRI (e.g.: cerebral aneurysm clip, cochlear implant, or pacemaker)\n5. Patients with a pacemaker or automated implantable cardioverter defibrillator will not undergo VCTE but can enroll and undergo the other deep phenotype testing as long as the device is compatible with MRI and MRI testing can be performed\n6. Patients requiring peritoneal or hemodialysis\n7. Uncontrolled infection or acute illness",{"count":119,"type":21},200,"Background:\n\nPeople with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens.\n\nObjective:\n\nTo study the benefits of treatments that are intended to cure SCD.\n\nEligibility:\n\nPeople aged 18 and older with SCD who are either receiving curative therapy in the next 3 months or don t have any plans to receive a curative therapy in the next 2 years.\n\nDesign:\n\nAt their first visit, participants will be screened with their medical history and a physical exam.\n\nParticipants will then have a baseline visit. This will take about a week to complete and will include:\n\nBlood and heart tests\n\nMRI of the brain, heart, and lungs. Participants will lie on a bed that will move into the MRI scanner. Special padding may be placed around their head to keep it still.\n\nInteractive games. Participants will complete computer games that test memory, attention, problem solving, language, spatial orientation, processing speed, and emotion.\n\nQuestionnaire rating quality of life\n\nIothalamate test. An IV catheter will be placed into a vein. A contrast agent will be injected through the IV. Blood will then be collected at different time points.\n\nLung function tests and a 6-minute walk test\n\nVibration controlled transient elastography. A probe placed on the abdomen will measure liver scarring.\n\nDOS test. A light attached to the finger or toe will measure blood oxygen.\n\nParticipants will have an end-of-study visit about 2 years after their baseline visit. This will include repeats of the baseline visit tests.",[122,123,124,125,126],"Mortality in Sickle Cell","Sickle Cell Cardiopulmonary Complications","Sickle Cell Organ Damage","Sickle Cell Life Expectancy and Risk Factors for Early Death","Sickle Cell Lung Disease and Sudden Death",[128,129,130,131,132,106],"Hydroxyurea","Hydroxyurea-Increased Fetal Hemoglobin","sickle cell anemi","Patterns of mortality in sickle cell disease","Mortality rates and age at death from sickle cell",{"date":39,"type":40},{"date":135,"type":40},"2022-03-24",{"date":137,"type":21},"2035-06-01",{"name":46,"class":47},{"id":140,"slug":4,"hasResults":11,"nctId":141,"briefTitle":142,"officialTitle":142,"acronym":4,"eligibilityCriteria":143,"healthyVolunteers":144,"sex":16,"minAge":145,"maxAge":18,"enrollmentInfo":146,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":148,"conditions":149,"keywords":154,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":160,"startDateStruct":161,"completionDateStruct":4,"leadSponsor":163,"locationsCount":48},"100053333","NCT00943514","Natural History of Bronchiectasis","* PARTICIPANT INCLUSION CRITERIA:\n\n  1. Children age 5 years and above and adults referred to the NIH with chronic or recurring respiratory infections will be eligible for participation.\n  2. Males and females will be considered without regard to race or ethnicity or upper age limit.\n  3. Emphasis will be primarily on non CF bronchiectasis for elucidation of mechanisms of infection susceptibility. However, select patients with cystic fibrosis or acquired immune defects (such as HIV) may be studied if relevant host defects are suspected, if needed for comparison purposes, or to evaluate and study the management of chronic pathogen associated conditions such as nontuberculous mycobacterial infections or allergic bronchopulmonary aspergillosis.\n  4. Subjects must have a primary or referring physician\n  5. Subjects must be willing to have samples stored\n\nINCLUSION CRITERIA FOR RELATIVES:\n\nAs a part of this protocol we may obtain blood, sputum, urine, or buccal swabs from some blood relatives of patients on the study, with the hope of isolating and characterizing the primary host defense defect(s) or genetic links responsible for airway infection susceptibility and\u002For bronchiectasis seen within families. Male and female relatives will be accepted without limitation due to age. These relatives may have pertinent disease-related history obtained, but will neither receive treatment nor have any other protocol procedures done unless they are enrolled on the study.\n\nPARTICIPANT EXCLUSION CRITERIA:\n\n1. We wish to avoid enrolling subjects, especially children, who have common respiratory problems (aeroallergen sensitivity, asthma, gastric esophageal reflux) that are not associated with an underlying abnormality in host defenses. Evidence of significance of chronic or recurring infections suggestive of an underlying airway surface or systemic host defense defect should be documented by one or more of the following: a) sinus or lower airway cultures positive for bacterial, fungal, or mycobacterial pathogens characteristic of these defects; or b) radiographic evidence of sinusitis with mucosal thickening and\u002For air-fluid levels; or c) radiographic evidence of bronchiectasis; d) severity of otitis media requiring placement of tympanic membrane pressure equalization tubes; e) severity of sinusitis requiring surgical intervention.\n2. Patients who are unable or unwilling to provide informed consent either directly or via appropriately designated surrogate.\n3. Any patient who, in the opinion of the Investigator, is unable or unwilling to comply with regular follow-up or is unlikely to provide pertinent information regarding disease progression or response to treatment may be excluded from longitudinal follow-up.\n\nPARTICIPATION OF CHILDREN:\n\nChildren under the age of 5 will be excluded from this protocol due to the difficulty of performing pertinent assays in infants and younger children, difficulty distinguishing significance of respiratory infections which occur commonly in younger children, and the lack of adequate facilities and equipment for management of children younger than 2 years.",true,"5 Years",{"count":147,"type":21},900,"Background:\n\n* Bronchiectasis is a disease characterized by airways that are inflamed, abnormally dilated, and chronically infected. Individuals with bronchiectasis have a history of chronic and recurring respiratory infections. Depending on the underlying cause, these infections may involve the entire respiratory tract, resulting in sinus, ear, and lung disease.\n* Bronchiectasis continues to be a significant problem in developing countries and in specific groups of individuals, particularly in people who have cystic fibrosis. Although treatments are available or under development for bronchiectasis related to cystic fibrosis, many of the disease-specific treatments may not be effective for bronchiectasis not related to cystic fibrosis.\n\nObjectives:\n\n\\- To study the natural history of bronchiectasis to identify inherited and immune factors that may explain why certain individuals have chronic recurring infections.\n\nEligibility:\n\n* Individuals 5 years of age and older who have an established diagnosis of bronchiectasis or a history of chronic\u002Frecurring respiratory infections.\n* Direct family members (e.g., parents, siblings, children) of patients in the study may also be asked to participate.\n\nDesign:\n\n* Potential participants will be screened with an initial clinic evaluation and full medical history, as well as a general quality of life and respiratory symptom questionnaire.\n* The following standard procedures may be done as part of the study: air sampling from the nose; imaging studies, which may include an x-ray or computed tomography (CT), lung function tests; and collection of samples of blood, urine, and sputum (phlegm or mucus). Other tests may be performed as required by the researchers, and will be explained to patients as needed.\n* Both patients and relatives (if asked to participate) will provide the following samples: blood or buccal (cheek swab) cells for genetic testing, sputum, and urine.\n* To prevent infections and potential disease progression, patients may receive standard medical care and treatment for bronchiectasis and related infections during this protocol.",[150,151,152,153],"Bronchiectasis","Cystic Fibrosis","Autoimmune Disease","Common Variable Immunodeficiency",[155,156,157,158,159,106],"Variant Cystic Fibrosis","Bronchopulmonary Mycosis","Airway Infection Susceptibility","Chronic Respiratory Infection","Primary Ciliary Dyskinesia",{"date":39,"type":40},{"date":162,"type":40},"2009-08-01",{"name":46,"class":47},{"id":165,"slug":4,"hasResults":11,"nctId":166,"briefTitle":167,"officialTitle":168,"acronym":4,"eligibilityCriteria":169,"healthyVolunteers":11,"sex":16,"minAge":170,"maxAge":171,"enrollmentInfo":172,"targetDuration":4,"studyType":22,"phases":174,"briefSummary":176,"conditions":177,"keywords":180,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":184,"startDateStruct":185,"completionDateStruct":187,"leadSponsor":189,"locationsCount":48},"100053445","NCT07599176","Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors","Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia","* INCLUSION CRITERIA\n\nRECIPIENT:\n\nParticipants must fulfill one disease category (1 or 2) and 3\n\n1. Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F):\n\n   A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb\n\n   B. Abnormal trans-cranial Doppler examination (\\>=200 cm\u002Fs); OR\n\n   C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR\n\n   D. Sickle cell related renal insufficiency defined by a creatinine level \\>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \\\u003C60mL\u002Fmin\u002F1.73m2 for patients \\\u003C16 years of age or \\\u003C50mL\u002Fmin for patients \\>16 years of age OR requiring peritoneal or hemodialysis; OR\n\n   Age (Years): \\\u003C= 5 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 0.8\n\n   Age (Years): 5 \\\u003C age \\\u003C= 10 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.0\n\n   Age (Years): 10 \\\u003C age \\\u003C= 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.2\n\n   Age (Years): \\> 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.3\n\n   E. Tricuspid regurgitant jet velocity (TRV) of \\>=2.5 m\u002Fs in patients at least 3 weeks after a vaso-occlusive crisis; OR\n\n   F. Recurrent severe priapism defined as at least two episodes of an erection lasting \\>=4 hours requiring medical intervention (e.g. aspiration, injection of vasoconstrictor, prior penile surgery.); OR\n\n   G. Sickle hepatopathy defined as EITHER ferritin \\>1000mcg\u002FL OR direct bilirubin \\>0.4 mg\u002FdL at baseline; OR\n\n   H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment \u002Fmedication; OR\n\n   I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment \u002Fmedication\n2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:\n\n   * Portal fibrosis by liver biopsy\n   * Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week)\n   * Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans\n3. Non disease specific\n\n   * Ages \\>=4 years and less than 65 years old\n   * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10)\n   * Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable\n   * Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration.\n\n     * Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant\u002Finjection from start of screening until immunosuppression is stopped.\n     * Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped.\n\nDONOR:\n\n* Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study.\n* Donors age 4 or older and \\>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study.\n* Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA\n\nRECIPIENT:\n\n* Karnofsky or Lanksy performance status of \\\u003C40\n* Diffusing capacity of carbon monoxide \\\u003C35% predicted: DLCO corrected for hemoglobin or KCO (corrected for lung volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing.\n* Baseline oxygen saturation of \\\u003C85% or PaO2 \\\u003C70\n* Left ventricular ejection fraction: \\\u003C35% estimated by ECHO\n* Transaminases \\>5x upper limit of normal for age\n* Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen\n* Major anticipated illness or organ failure incompatible with survival from HCT\n* Pregnant or breastfeeding\n\nDONOR:\n\n* Pregnant or breastfeeding\n* Cognitively impaired subjects","4 Years","65 Years",{"count":173,"type":21},90,[59,175],"PHASE2","This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.",[178,179],"Sickle Cell Disease","Beta-thalassemia",[178,181,182,183],"Beta-Thalassemia","Stem Cell Transplant","Chimerism",{"date":39,"type":40},{"date":186,"type":40},"2026-07-08",{"date":188,"type":21},"2035-06-30",{"name":46,"class":47},{"id":191,"slug":4,"hasResults":11,"nctId":166,"briefTitle":167,"officialTitle":168,"acronym":4,"eligibilityCriteria":192,"healthyVolunteers":11,"sex":16,"minAge":170,"maxAge":171,"enrollmentInfo":193,"targetDuration":4,"studyType":22,"phases":194,"briefSummary":176,"conditions":195,"keywords":196,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":198,"startDateStruct":200,"completionDateStruct":202,"leadSponsor":203,"locationsCount":48},"100641763","* INCLUSION CRITERIA\n\nRECIPIENT:\n\nParticipants must fulfill one disease category (1 or 2) and 3\n\n1. Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F):\n\n   A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb\n\n   B. Abnormal trans-cranial Doppler examination (\\>=200 cm\u002Fs); OR\n\n   C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR\n\n   D. Sickle cell related renal insufficiency defined by a creatinine level \\>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \\\u003C60mL\u002Fmin\u002F1.73m2 for patients \\\u003C16 years of age or \\\u003C50mL\u002Fmin for patients \\>16 years of age OR requiring peritoneal or hemodialysis; OR\n\n   Age (Years): \\\u003C= 5 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 0.8\n\n   Age (Years): 5 \\\u003C age \\\u003C= 10 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.0\n\n   Age (Years): 10 \\\u003C age \\\u003C= 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.2\n\n   Age (Years): \\> 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.3\n\n   E. Tricuspid regurgitant jet velocity (TRV) of \\>=2.5 m\u002Fs in patients at least 3 weeks after a vaso-occlusive crisis; OR\n\n   F. Recurrent severe priapism defined as at least two episodes of an erection lasting \\>=4 hours requiring medical intervention (e.g. aspiration, injection of vasoconstrictor, prior penile surgery.); OR\n\n   G. Sickle hepatopathy defined as EITHER ferritin \\>1000mcg\u002FL OR direct bilirubin \\>0.4 mg\u002FdL at baseline; OR\n\n   H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment \u002Fmedication; OR\n\n   I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment \u002Fmedication\n2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:\n\n   * Portal fibrosis by liver biopsy\n   * Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week)\n   * Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans\n3. Non disease specific\n\n   * Ages \\>=4 years and less than 65 years old\n   * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10\n   * Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable\n   * Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration.\n\n     * Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant\u002Finjection from start of screening until immunosuppression is stopped.\n     * Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped.\n\nDONOR:\n\n* Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study.\n* Donors age 4 or older and \\>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study.\n* Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA\n\nRECIPIENT:\n\n* Karnofsky or Lanksy performance status of \\\u003C40\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C35% predicted (corrected for hemoglobin and alveolar volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing.\n* Baseline oxygen saturation of \\\u003C85% or PaO2 \\\u003C70\n* Left ventricular ejection fraction: \\\u003C35% estimated by ECHO\n* Transaminases \\>5x upper limit of normal for age\n* Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen\n* Major anticipated illness or organ failure incompatible with survival from HCT\n* Pregnant or breastfeeding\n\nDONOR:\n\n* Pregnant or breastfeeding\n* Cognitively impaired subjects",{"count":173,"type":21},[59,175],[178,179],[178,181,182,183],"2026-07-01",{"date":199,"type":40},"2026-07-02",{"date":201,"type":21},"2026-07-07",{"date":188,"type":21},{"name":46,"class":47},{"id":205,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":206,"targetDuration":4,"studyType":22,"phases":207,"briefSummary":208,"conditions":209,"keywords":210,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":211,"startDateStruct":212,"completionDateStruct":213,"leadSponsor":214,"locationsCount":48},"100641631",{"count":20,"type":21},[24],"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.",[27,28,29,30,31,32],[34,35],{"date":199,"type":40},{"date":201,"type":21},{"date":44,"type":21},{"name":46,"class":47},{"id":216,"slug":4,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":4,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":80,"enrollmentInfo":217,"targetDuration":4,"studyType":22,"phases":218,"briefSummary":83,"conditions":219,"keywords":4,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":220,"startDateStruct":221,"completionDateStruct":222,"leadSponsor":223,"locationsCount":48},"100636500",{"count":7,"type":21},[59],[85],{"date":199,"type":40},{"date":201,"type":21},{"date":90,"type":21},{"name":46,"class":47},{"id":225,"slug":4,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":55,"enrollmentInfo":226,"targetDuration":4,"studyType":22,"phases":227,"briefSummary":60,"conditions":228,"keywords":229,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":230,"startDateStruct":231,"completionDateStruct":232,"leadSponsor":233,"locationsCount":73},"100588952",{"count":57,"type":21},[59],[62],[64,65,66,67],{"date":199,"type":40},{"date":201,"type":21},{"date":71,"type":21},{"name":46,"class":47},{"id":235,"slug":4,"hasResults":11,"nctId":236,"briefTitle":237,"officialTitle":237,"acronym":4,"eligibilityCriteria":238,"healthyVolunteers":144,"sex":16,"minAge":145,"maxAge":18,"enrollmentInfo":239,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":240,"conditions":241,"keywords":243,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":245,"startDateStruct":246,"completionDateStruct":248,"leadSponsor":250,"locationsCount":48},"100558565","NCT06552767","Thrombosis and Inflammation in Vessels Initiative (TIVI)","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n* Age \\>= 5 years at the time of consent\n* Ability of subject or Legally Authorized Representative (LAR) as applicable to understand and the willingness to sign a written informed consent document\n\nIn addition, the following cohort-specific inclusion criteria apply:\n\nAffected Subjects:\n\n-Known or possible thrombotic, immune, or vascular disorder after review of the subject s medical records and\u002For discussion of medical history\n\nRelatives of Affected Subjects:\n\n-Being a relative of an affected subject\n\nUnrelated Healthy Controls:\n\n-In good general health as evidenced by medical history\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n-Any condition that in the opinion of the Investigator would warrant exclusion\n\nUnrelated Healthy Controls:\n\n* Unrelated healthy volunteers who decline to have blood drawn and\u002For tissue studies or who do not consent to have samples stored for future research\n* Cognitively impaired individuals",{"count":98,"type":21},"Background:\n\nDiseases related to the immune system, blood clots, and blood vessels can affect every part of the body. These diseases are now known to be interrelated: People who have strokes, blood clots in their legs, or autoimmune disease, for example, are at greater risk of complications in the heart, brain, and other organs. Researchers want to learn more about how these diseases start, how they change over time, and how they affect different organs.\n\nObjective:\n\nTo learn more about how inflammation and diseases of the blood vessels start and how they change over time.\n\nEligibility:\n\nPeople aged 5 years and older with a disease related to blood clots, the immune system, or blood vessels. Healthy relatives of people with these diseases and unrelated healthy volunteers are also needed.\n\nDesign:\n\nParticipants will have a baseline visit: They will provide a medical history, physical exam and blood test. All other tests and procedures are optional; these may be spread over more than 1 day:\n\nTests of heart and lung function.\n\nFill in a family tree form.\n\nImaging scans\n\nTreadmill or bike stress tests and a 6-minute walk test.\n\nTests of blood pressure and the flow of blood through vessels.\n\nPhotos of the face and body.\n\nEye exams, with photos taken of the retina.\n\nSaliva and urine samples.\n\nBiopsies (tissues samples) of the skin and fat.\n\nTests of thinking and mental function.\n\nEvaluations by other medical specialists.\n\nParticipants may opt to return for repeat testing for up to 90 months (7.5 years).\n\nSome visits may be done by telehealth.",[32,242],"Vascular Diseases",[244],"Vascular",{"date":199,"type":40},{"date":247,"type":40},"2024-11-22",{"date":249,"type":21},"2034-01-30",{"name":46,"class":47},{"id":252,"slug":4,"hasResults":11,"nctId":253,"briefTitle":254,"officialTitle":254,"acronym":4,"eligibilityCriteria":255,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":256,"enrollmentInfo":257,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":259,"conditions":260,"keywords":267,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":272,"startDateStruct":273,"completionDateStruct":275,"leadSponsor":277,"locationsCount":48},"100494895","NCT05724121","Observational Study of Cardiac Arrhythmias During Treatment With BTK Inhibitors or Venetoclax","* INCLUSION CRITERIA:\n\nTo be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Currently receiving or planning to receive a BTKi or venetoclax.\n2. Male or female, aged 18 or older\n3. Stated willingness to comply with all study procedures and availability for the duration of the study\n4. 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. Any acute cardiac condition including myocardial infarction or decompensated heart failure within the past 3 months\n2. Pregnancy or lactation- use of BTK inhibitors is contraindicated in pregnant or nursing individuals.","110 Years",{"count":258,"type":21},135,"Background:\n\nBruton s tyrosine kinase inhibitors (BTKi) are used to treat a form of leukemia. But taking BTKi can also increase a person s risk of developing an abnormal heart rhythm. This can cause sudden death. In this natural history study, researchers want to learn how BTKi affects the heart.\n\nObjective:\n\nTo identify and monitor the effects of BTKi on the heart.\n\nEligibility:\n\nPeople aged 18 and older currently receiving or planning to receive BTKi or venetoclax.\n\nDesign:\n\nParticipants who have not yet started BTKi will have 2 required clinic visits: 1 before they start taking BTKi, and 1 about 6 months later. Participants who are already taking BTKi will have 1 required visit.\n\nParticipants will undergo multiple tests:\n\nA physical exam, including collection of blood and saliva.\n\nA test that measures heart activity via stickers placed on the chest.\n\nA test that uses sound waves to capture images of the heart.\n\nAn exercise stress test that monitors heart activity and blood pressure while the participant works on a treadmill or stationary bike. Sound wave images of the heart may also be taken while the participant exercises.\n\nStress magnetic resonance imaging (MRI) may be done in place of an exercise test. Participants will lie on a table that slides into a tube. They will be given drugs to stress the heart while images are taken.\n\nParticipants may wear a device to monitor their heart at home.\n\nParticipants may have repeat visits if they develop heart symptoms or if they need to stop taking BTKi. They will have follow-up phone calls each year for up to 3 years.",[261,262,263,264,265,266],"Chronic Lymphocytic Leukemia (CLL)","Waldenstr(SqrRoot)(Delta)m s Macroglobulinemia","Mantle Cell Lymphoma","Sudden Cardiac Death","Cardiac Arrhythmias","Hematologic Malignancies",[268,261,269,270,271,106],"SUDDEN CARDIAC DEATH","Ibrutinib Treatment","Atrial Fibrillation\u002FFlutter","Ventricular Arrhythmias",{"date":199,"type":40},{"date":274,"type":40},"2023-03-01",{"date":276,"type":21},"2027-04-08",{"name":46,"class":47},{"id":279,"slug":4,"hasResults":11,"nctId":94,"briefTitle":95,"officialTitle":95,"acronym":4,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":280,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":281,"conditions":282,"keywords":283,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":284,"startDateStruct":285,"completionDateStruct":286,"leadSponsor":287,"locationsCount":112},"100467888",{"count":98,"type":21},"Background:\n\nDoctors use computed tomography (CT) to get detailed pictures of the heart. CT uses x-rays to gather raw data. Computers assemble this data to make the images doctors look at. A new computer technique can make higher resolution images from the same CT scans. In this natural history study, researchers will take normal CT images of the heart. They will compare those images to super high-resolution (super high-res) images made with a super-computer.\n\nObjective:\n\nTo improve the quality of heart CT scans by using new methods to create the images.\n\nEligibility:\n\nPeople aged 18 years or older who need a CT scan for heart disease.\n\nDesign:\n\nParticipants will have a normal CT scan. A substance will be injected through a tube in their arm. They will lie on a table in a large, donut-shaped machine. An X-ray tube will move around their body, taking many pictures.\n\nResearchers will use the normal CT scans to create super high-res images. They may do this at the NIH. They may also send the images to the company that made the CT scanner. Participants personal information will be removed before images are sent to the company. The personal information will be replaced by a code.\n\nThe super high-res images will be returned to the NIH.\n\nSome information will be collected from participants medical records. Researchers will compare the normal scans to the super high-res images.\n\nParticipants' own doctors will also have a chance to see the super high-res images.\n\nParticipants' CT pictures will be stored and used for future NIH research....",[102],[104,105,106],{"date":199,"type":40},{"date":201,"type":21},{"date":110,"type":21},{"name":46,"class":47},{"id":289,"slug":4,"hasResults":11,"nctId":290,"briefTitle":291,"officialTitle":291,"acronym":4,"eligibilityCriteria":292,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":80,"enrollmentInfo":293,"targetDuration":4,"studyType":22,"phases":295,"briefSummary":297,"conditions":298,"keywords":300,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":305,"startDateStruct":306,"completionDateStruct":308,"leadSponsor":310,"locationsCount":48},"100452353","NCT05170399","Vaccine Responses in Patients With B Cell Malignancies","* INCLUSION CRITERIA:\n* Diagnosis of CLL, FL, MCL, MZL, NHL NOS or WM\n* Must fulfil one of the following criteria to be enrolled in one study arm per vaccine received:\n\n  1. Patients with CLL AND one of the following:\n\n     i. Arm 1: Must be treatment naive (no prior cancer directed therapy)\n\n     ii. Arm 2: Patients that have received prior cancer directed therapy and are currently not receiving active treatment\n\n     iii. Arm 3: Must be receiving treatment with a BTKi. This arm is not available to patients receiving the HEPLISAV-B vaccine\n\n     iv. Arm 4: Must be receiving treatment with a BTKi for \\>= 6 months prior to vaccination and be willing to hold their treatment for up to 7 weeks around the time of each vaccination. This arm is not available to patients who have had a prior episode of disease flare during periods of drug hold, or for patients with CLL that is actively progressing.\n\n     v. Arm 5: Must be receiving treatment with a BCL-2 inhibitor\n\n     Or\n  2. Patients with FL, MCL, MZL, NHL NOS or WM AND one of the following:\n\n     i. Arm 1: Must currently not be receiving active treatment (treatment na(SqrRoot) ve or previously treated)\n\n     ii. Arm 2: Must be receiving treatment with targeted therapies (e.g. BTKi, BCL-2 inhibitors, PI3K inhibitors, immunomodulatory agents, proteasome inhibitors)\n* If prior exposure to Hepatitis-B vaccination, must have documentation of negative serologic response\n* Age \\>= 18 years\n* Able to comprehend the investigational nature of the protocol and provide informed consent\n\nEXCLUSION CRITERIA:\n\n1. Female patients who are currently pregnant\n2. History of severe allergic reaction to vaccines\n3. Concomitant inherited immunodeficiency\n4. Any life-threatening illness, medical condition, or organ system dysfunction that, in the investigator s opinion, could compromise the subject s safety or put the study outcomes at undue risk.\n5. Receive cytotoxic chemotherapy within 2 weeks prior to vaccination\n6. Receive intravenous immunoglobulin (IVIG) within 2 months prior to vaccination\n7. Receive anti-CD20 and\u002For anti-CD19 monoclonal antibody therapy within 6 months prior to vaccination\n8. Receive cellular therapy (e.g. CAR-T cells) within 12 months prior to vaccination\n9. History of allogeneic stem cell transplantation",{"count":294,"type":21},350,[296],"PHASE4","Background:\n\nPeople with B cell malignancies (blood cancers) often cannot mount a full immune response to infections or certain vaccines. Bruton tyrosine kinase inhibitors (BTKis), which are used to treat blood cancers, may also negatively affect a person s response to certain vaccines. Researchers want to learn more about vaccine responses in people with certain types of blood cancers. The findings may help develop better vaccine strategies for people with these cancers.\n\nObjective:\n\nTo learn how well vaccines work in people who have certain types of blood cancers.\n\nEligibility:\n\nAdults aged 18 years or older who have chronic lymphocytic leukemia (CLL), Waldenstrom macroglobulinemia, or certain non-Hodgkin lymphomas.\n\nDesign:\n\nParticipants will get one or more vaccines for illnesses such as COVID-19, hepatitis B, and shingles. They can choose which vaccines they receive. They will give a blood sample before they get each vaccine. Some vaccines require a second dose 3-6 weeks later. Participants may give an optional blood sample with the second vaccine dose. About 4 weeks after they finish each vaccine series, they will give another blood sample. They will have 2-3 study visits per vaccine.\n\nParticipants may receive a booster dose for some vaccines. The booster dose is optional. They will give another blood sample with the booster dose.\n\nParticipants will have pregnancy tests, if needed.\n\nParticipants with CLL who receive BTKis may be asked to pause treatment for up to 7 weeks.\n\nParticipants may give follow-up blood samples up to 2 times a year for 5 years. These blood samples are optional.\n\nParticipation will last for up to 5 years after each vaccine series is received.",[299],"Lymphoma",[301,302,303,299,304],"CLL","SLL","Booster","Vaccines",{"date":199,"type":40},{"date":307,"type":40},"2022-09-14",{"date":309,"type":21},"2026-08-16",{"name":46,"class":47},{"id":312,"slug":4,"hasResults":11,"nctId":313,"briefTitle":314,"officialTitle":314,"acronym":4,"eligibilityCriteria":315,"healthyVolunteers":144,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":316,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":318,"conditions":319,"keywords":325,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":331,"startDateStruct":332,"completionDateStruct":334,"leadSponsor":336,"locationsCount":48},"100444826","NCT05072483","Natural History Study of CADASIL","* INCLUSION CRITERIA:\n\nEligibility for this study may be determined based on information collected under other NHLBI-approved protocols, outside records and patient report.\n\nIn order to be eligible to participate in this study, an individual must meet criteria 1 \\& 2 and either criteria 3 or 4:\n\n1. Stated willingness to comply with all study procedures and availability for the duration of the study.\n2. Male or female, aged 18 to 100 years (inclusive).\n3. Established diagnosis of CADASIL or NOTCH3 mutations, as determined by genetic testing.\n4. Healthy controls.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n1. Pregnancy at time of consent.\n2. Subjects who lack capacity to consent and don't have a legally authorized representative.\n3. Subjects who decline to provide samples for blood and\u002For tissue studies.\n4. Subjects who do not speak English.\n5. Subjects whose scans or examinations show unexpected brain conditions (outside of CADASIL) which would interfere with interpretation of testing.\n6. Subjects unable to undergo an MRI scan or subjects meeting the following criteria:\n\n   * Subjects who have internal non-MRI compatible metals (i.e., cardiac pacemaker, brain stimulator, shrapnel, surgical metal, clips in the brain or on blood vessels, cochlear implants, artificial heart valves or metal fragments in the eye) as these rendering an MRI unsafe\n   * Subjects with ferromagnetic dental bridges or crowns (exclusion only for 7.0T)\n   * Subjects unable to remain supine for the expected length of the MRI (i.e., up to 1 hour)\n   * Subjects with uncontrolled head movements\n   * Subjects who are claustrophobic for the expected length of the MRI (i.e., up to 1 hour) and claustrophobia cannot be controlled with anti-anxiety medication.",{"count":317,"type":21},155,"Background:\n\nCADASIL (cerebral autosomal dominant arteriopathy with subcortical infarct and leukoencephalopathy) is a genetic disorder. It causes narrowing of the small blood vessels and can lead to strokes and dementia. Researchers want to monitor people with CADASIL over time.\n\nObjective:\n\nTo learn more about how CADASIL affects a person s blood vessels over time.\n\nEligibility:\n\nAdults ages 18 and older who have CADASIL, and healthy volunteers.\n\nDesign:\n\nParticipants will be screened with a medical record review.\n\nParticipants will have 4 study visits over 9 years. Visits will last 6 8 hours per day, for 2 4 days.\n\nParticipants will give blood and urine samples. They will have an electrocardiogram to record their heart s electrical activity. They will fill out a family tree. They will have tests that measure mental abilities like memory and attention. They may have a skin biopsy. They may have a lumbar puncture.\n\nParticipants will have an eye exam. Their pupils will be dilated. They will receive a dye via intravenous (IV) line. Pictures will be taken of their eyes.\n\nParticipants will have an imaging scan of their brain. They may receive a contrast agent via IV.\n\nParticipants blood flow and blood vessel flexibility will be measured. In one test, a probe will be pressed against the skin of the their wrist, neck, and groin. In another test, they will hold one arm still while a microscope makes videos of the blood flow through a fingernail. In another test, they will perform light exercise or other activities while wearing an elastic band around their head or probes placed on their arm or leg.\n\nHealthy volunteers will complete some of the above tests.",[320,321,322,323,324],"Cardiovascular Disease","Arterial Stiffness","Germline Mutation in the NOTCH 3 Gene","Pathogenesis of CADASIL","Clinical Phenotype of CADASIL",[326,327,328,329,330],"Biospecimen Procurement","Laboratory Research Specimens","progressive chronic hypoperfusion","Stroke","progressive white matter degeneration, and debilitating dementia.",{"date":199,"type":40},{"date":333,"type":40},"2022-04-18",{"date":335,"type":21},"2041-06-01",{"name":46,"class":47},{"id":338,"slug":4,"hasResults":11,"nctId":339,"briefTitle":340,"officialTitle":340,"acronym":4,"eligibilityCriteria":341,"healthyVolunteers":11,"sex":16,"minAge":170,"maxAge":342,"enrollmentInfo":343,"targetDuration":4,"studyType":22,"phases":345,"briefSummary":346,"conditions":347,"keywords":351,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":354,"startDateStruct":355,"completionDateStruct":357,"leadSponsor":359,"locationsCount":48},"100325717","NCT03520647","Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count less than or equal to 1000 cells\u002F microL or for patients receiving granulocyte transfusions, absolute neutrophil count less than or equal to 1000 cells\u002FmicroL before beginning granulocyte transfusions).\n\nOR\n\n--History of severe aplastic anemia transformed to MDS. that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C5% myeloblasts and \\\u003C30% of cellularity in the bone marrow on screening morphologic analysis.\n\nOR\n\n* PNH that is either refractory to treatment with eculizumab\u002Fravulizumab or occurs in patients who don t have access to treatment with eculizumab associated with either a) life- threatening thrombosis and\u002For b) cytopenia associated with transfusion dependence and\u002For c) recurrent and debilitating hemolytic crisis.\n\n  * Subjects with severe aplastic anemia, hypoplastic MDS or PNH with associated bone marrow failure syndromes who have intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n  * Availability of at least one HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) to serve as a stem cell donor for the allogeneic transplant.\n  * Availability of a backup stem cell source in the event of graft rejection:\n* at least one additional haploidentical related alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>= 9\u002F10 HLA matched unrelated donor who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the haplo-transplant has been rejected.\n* umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the haplo-transplant has been rejected\n\n  * The patient does not have any HLA antibodies detectable against any of the mismatched HLA alleles expressed by the haplo-donor.\n  * Ages 4-60 years inclusive.\n  * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* The patient is deemed to be a candidate for a 12\u002F12 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C40% (evaluated by ECHO)\n* Transaminases \\> 5x upper limit of normal.\n* Direct bilirubin \\>3 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm\\^2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n\nINCLUSION CRITERIA - DONOR:\n\nDonor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA - RELATED DONOR:\n\nNone","75 Years",{"count":344,"type":21},56,[175],"Background:\n\nSevere aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria\n\n(PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants.\n\nObjectives:\n\nTo see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy.\n\nEligibility:\n\nRecipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75\n\nDesign:\n\nRecipients will have:\n\n* Blood, urine, heart, and lung tests\n* Scans\n* Bone marrow sample\n\nRecipients will need a caregiver for several months. They may make fertility plans and a power of attorney.\n\nDonors will have blood and tissue tests, then injections to boost stem cells for 5-7 days.\n\nDonors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg.\n\nIn the hospital for about 1 month, recipients will have:\n\n* Central line inserted in the neck or chest\n* Medicines for side effects\n* Chemotherapy over 8 days and radiation 1 time\n* Stem cell transplant over 4 hours\n\nUp to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests.\n\nAt day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.",[348,349,350],"Severe Aplastic Anemia (SAA)","Hypo-Plastic Myelodysplastic Syndrome (MDS)","Paroxysmal Nocturnal Hemoglobinuria (PNH)",[352,353],"Haploidentical CD34+cells","Non-Myeloablative",{"date":199,"type":40},{"date":356,"type":40},"2019-02-19",{"date":358,"type":21},"2028-06-01",{"name":46,"class":47},{"id":361,"slug":4,"hasResults":11,"nctId":362,"briefTitle":363,"officialTitle":363,"acronym":4,"eligibilityCriteria":364,"healthyVolunteers":144,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":365,"targetDuration":4,"studyType":22,"phases":367,"briefSummary":369,"conditions":370,"keywords":372,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":378,"startDateStruct":379,"completionDateStruct":381,"leadSponsor":383,"locationsCount":48},"100311207","NCT03331380","Technical Development of Cardiovascular Magnetic Resonance Imaging (CMR) Using a Low Specific Absorption Rate (SAR) Scanner System","* INCLUSION CRITERIA:\n\nInclusion Criteria for All Participants (Objectives 1, 2, 3, and 4)\n\n* Men and women age greater than or equal to 18 years\n* Able to provide informed consent in writing\n* Willingness to cooperate with all study procedures (including food restriction) and available for scheduled study events\n\nInclusion Criteria for Healthy Volunteers (Objectives 1 and 2)\n\n-Currently healthy, self-reported\n\nInclusion Criteria for Subjects with Heart Disease (Objective 3)\n\n* Subjects having known heart disease including but not limited to\n* Stable angina pectoris due to epicardial coronary artery obstruction\n* Past myocardial infarction\n* Heart failure with reduced ejection fraction\n* Valvular heart disease\n* Pulmonary artery hypertension\n* Congenital heart disease with or without prior repair\n* Myocarditis\n* Infiltrative cardiomyopathy\n* Hypertrophic cardiomyopathy\n\nInclusion Criteria for Subjects with Non-Cardiac Disease (Objective 4)\n\n* Having known brain disease including but not limited to:\n\n  * Transient ischemic attack or stroke after 24 hours of onset\n  * Infection, inflammation meningitis\n  * Cognitive decline, neurodegenerative disorders\n  * Demyelinating disease, multiple sclerosis\n  * Loss of consciousness, seizures, epilepsy\n  * Brain tumor, metastases, abscess, lesion\n  * Vascular pathology\n  * Headache\n  * Hemorrhage\n  * Trauma\n* Have known musculoskeletal disease including but not limited to:\n\n  * Persistent neck pain or radiculopathy\n  * Cancer or tumors of the spine\n  * Congenital abnormalities of the spinal cord or knee\n  * Multiple sclerosis\n  * Injury or trauma\n  * Fracture evaluation\n  * Infectious or inflammatory processes\n  * Soft tissue damage\n  * Muscle or tendon disorders\n  * Knee meniscal disorders\n  * Marrow abnormalities\n  * Mechanical knee symptoms\n  * Vascular conditions\n* Have known abdominal diseases including but not limited to:\n\n  * Bowel obstruction\n  * Masses and tumors\n  * Crohns disease\n  * Diffuse liver disease such as hemochromatosis, hemosiderosis, fatty infiltration\n  * Focal hepatic lesions\n  * Cirrhotic liver\n  * Iron content determination\n  * Cystic kidney disease\n  * Vascular abnormalities\n* Have known lung disease including but not limited to:\n\n  * Cancer, tumors and masses\n  * Vascular and lymphatic abnormalities\n  * Pulmonary thromboembolic disease\n  * Trauma\n  * Suspected bronchiolitis\n  * Bronchiectasis or pneumonitis\n  * Asthma and other obstructive lung diseases\n  * Pulmonary lymphangioleiomyomatosis\n  * Cystic and interstitial lung diseases such as pulmonary lymphangioleiomyomatosis and cystic fibrosis\n* Have other known non-cardiovascular disease\n\nInclusion criteria for bronchodilators:\n\n\\- Only subjects who use bronchodilators routinely or those have undergone previous pulmonary function testing with bronchodilators will be invited to undergo bronchodilator testing during MRI.\n\nEXCLUSION CRITERIA:\n\nExclusion Criteria for All Participants:\n\n* Conditions that are thought to make MRI unsafe (that will be determined by filling out a separate form) including:\n\n  * Cerebral aneurysm clip unless it is labeled safe for MRI\n  * Neural stimulator (e.g. TENS-Unit) unless it is labeled safe for MRI\n  * Any type of ear implant unless it is labeled safe for MRI\n  * Ocular foreign body (e.g. metal shavings)\n  * Metal shrapnel or bullet\n  * Any implanted device (e.g. insulin pump, drug infusion device), unless it is labeled safe for MRI\n* Pregnancy. When uncertain, subjects will undergo either urine or serum pregnancy testing. Results from up to 3 days prior to the examination will be used. Post-menopausal and surgically sterilized subjects are exempt from this testing.\n\n  * If a urine or serum pregnancy test was administered as part of a referral protocol, and the test was administered up to 3 days prior to MRI examination for this protocol, a new pregnancy test will not be required for this protocol.\n  * If a pregnancy test has not been administered within 3 days of MRI examination for this protocol, a serum or urine pregnancy test will be administered. Medical personnel will determine which pregnancy test is appropriate based on subject s medical history, screening, and individual scenario. In addition, the subject will be asked if she may be pregnant prior to the performance of the MRI, even if the pregnancy test was negative within the past week. The pregnancy test will be repeated if she answers in the affirmative.\n\nExclusion Criteria for Gadolinium:\n\n-When gadolinium based contrast agent (GBCA) exposure is planned\n\n* Objective 1 and 2, Healthy Volunteers: No gadolinium based contrast agent exposure is permitted if eGFR \\\u003C 60 mL\u002Fmin\u002F1.73m\\^(2) using the CKD-EPI equation or equivalent and a serum creatinine measured within 2 weeks without intercurrent change in medical condition or medications.\n* Objective 3 and 4, Volunteers with Cardiac and Non-Cardiac Disease: No gadolinium based contrast agent exposure is permitted if eGFR \\\u003C 30 mL\u002Fmin\u002F1.73m\\^(2) using the CKDEPI equation \\[25\\] or equivalent and a serum creatinine measured within 2 weeks without\n\nintercurrent change in medical condition or medications.\n\n--For all objectives not more than two research MRI exams with GBCA exposure in a 12 month period. Research MRI exams performed outside of this protocol will be included in the 12 month period.\n\n* Subjects meeting this exclusion criterion, or without eGFR determination, may still be included in the study but may not be exposed to gadolinium-based contrast agents\n* Breast feeding (unless subject is willing to discard breast milk for 24 hours)\n* Does not wish to be exposed to gadolinium.\n\nExclusion Criteria for Oxygen Inhalation:\n\n* Severe chronic obstructive pulmonary disease defined as requiring more than one bronchodilator medication every day or continuous oxygen requiring\n* Prior treatment with bleomycin\n\nExclusion Criteria for Ferumoxytol Contrast\n\n* A history of allergic reaction to any intravenous iron product\n* Allergy to ferumoxytol or to mannitol excipient\n* Breast feeding\n* Does not wish to be exposed to ferumoxytol\n* Iron overload\n\nExclusion Criteria for oral contrast agent:\n\n* A history of reaction to oral contrast (if using barium sulfate)\n* Breast feeding unless subject is willing to discard breast milk for 24 hours (if using barium sulfate)\n* Allergy to pineapple (if using pineapple juice)\n* Does not wish to be exposed to oral contrast\n\nExclusion Criteria for Healthy Volunteers (Objectives 1 and 2)\n\n-Important past medical illness\n\nExclusion Criteria for Adults with heart Disease (Objective 3)\n\n* Unstable angina, acute coronary syndrome, or myocardial infarction not attributable to PCI, within 2 weeks unless after coronary revascularization of the culprit lesion.\n* Any hemodynamic instability or decompensated heart failure as determined by the enrolling physician.\n* Adenosine: Patients with asthma or chronic obstructive pulmonary disease of any severity are ineligible for vasodilator stress CMR with adenosine.\n* Regadenoson: Only patients with severe or uncontrolled asthma or severe or uncontrolled chronic obstructive pulmonary disease are ineligible for vasodilator stress CMR with regadenoson. This will be determined at the discretion of the supervising provider based on medical records and physical exam.\n* Patients with advanced heart block on baseline ECG are ineligible for vasodilator stress CMR\n\nExclusion Criteria for Adults with Non-Cardiac Disease (Objective 4)\n\n* Acute illness for which investigational imaging might delay care (such as acute stroke before treatment), as determined by the enrolling physician\n* Any hemodynamic instability as determined by the enrolling physician.\n\nExclusion criteria for bronchodilators:\n\n\\- Patient refuses bronchodilator administration.\n\nExclusion criteria for adults with cardiac implanted electronic devices (CIED, pacemakers or defibrillators:\n\nLow-SAR MRI:\n\n* Subjects with MRI-conditional and legacy CIEDs are not excluded, based on the intrinsic safety of low-SAR MRI.\n* Subjects with CIEDs are excluded if they have pacemakers implanted before 1998; ICDs implanted before 2000; temporary, epicardial or abandoned leads; and CIEDs implanted \\\u003C4 weeks prior to MRI exam.\n\nConventional MRI:\n\n-Subjects with CIED are excluded from conventional MRI unless they have CIEDs that are labeled as MRI conditional or MRI safe, and that are implanted greater than or equal to 4 weeks prior to MRI.",{"count":366,"type":21},2950,[368],"NA","Background:\n\nResearchers are testing version of a system known as a magnetic resonance imagining (MRI) scanner that uses strong magnetic fields, radio waves and the like to create images of the organs in the body. It uses lower energy levels than other MRI scanners. This may help scan people with metal devices in their body, or in invasive heart procedures using metal tools.\n\nObjective:\n\nTo test a new MRI scanner and software changes to create better pictures.\n\nEligibility:\n\nPeople with disease and healthy volunteers, ages 18 and older.\n\nDesign:\n\nParticipants will be screened with blood tests.\n\nParticipants may have both the new MRI and a conventional MRI or only the new one. If 2 are done, they must be within 60 days.\n\nFor both MRI versions, participants lie on a table that slides into a large tube. During scans, they will hold their breath for up to 20 seconds at a time. Heart activity will be measured by wires connected to pads on the skin. A flexible belt may be used to monitor their breathing. They will be in the scanner up to 2 hours.\n\nParticipants can agree to have a dye called gadolinium injected into their arm during the scan. This brightens the pictures.\n\nParticipants can agree to take a drug called a vasodilator. This helps detect areas of the heart with abnormal blood supply. Scans of the heart are taken before, during, and after they get the medicine. The drug may cause temporary chest pain or shortness of breath. They may get other drugs to relieve these symptoms.\n\nSponsoring Institution: National Heart, Lung, and Blood Institute",[371],"CAD",[373,374,375,376,377],"1.5 T CMR","Low SAR CMR","Gadolinium","Regadenoson","Adenosine",{"date":199,"type":40},{"date":380,"type":40},"2018-01-05",{"date":382,"type":21},"2028-05-31",{"name":46,"class":47},{"id":385,"slug":4,"hasResults":11,"nctId":386,"briefTitle":387,"officialTitle":388,"acronym":4,"eligibilityCriteria":389,"healthyVolunteers":11,"sex":16,"minAge":170,"maxAge":342,"enrollmentInfo":390,"targetDuration":4,"studyType":22,"phases":392,"briefSummary":393,"conditions":394,"keywords":398,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":401,"startDateStruct":402,"completionDateStruct":404,"leadSponsor":406,"locationsCount":48},"100299122","NCT03173937","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count \\\u003C=1000 cells\u002F uL or for patients receiving granulocyte transfusions, absolute neutrophil count \\\u003C=1000 cells\u002F uL before beginning granulocyte transfusions).\n\nOR\n\nHistory of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C 5% myeloblasts and \\\u003C 30% of cellularity in the bone marrow on screening morphologic analysis.\n\n-Intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n\nIdentification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>=5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>=9\u002F10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn(TM) unit has been rejected or b) umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the CordIn(TM) unit has been rejected.\n\n-Availability of at least one \\>=4\u002F8 HLA matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).\n\nThe cord blood unit must contain a minimum TNC of at least 1.8 x 10\\^9 and at least 1.5x10\\^7\u002Fkg TNC and at least 8 x 10\\^6 CD34+ cells (all doses prior to thawing).\n\nException: Cord units containing at least 8 x 10\\^6 CD34+ cells but less than 1.8 x 10\\^9 TNC may be eligible for use on this trial if\n\n1. the pre-expansion CD34\u002Fkg recipient cell number is at least 1.5 x 10\\^5 \u002Fkg\n\n   AND\n2. approval for use of this cord unit for expansion is granted by Gamida Cell.\n3. the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.\n\n   * The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.\n   * Ages 4-60 years inclusive.\n   * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C 40% (evaluated by ECHO).\n* Transaminases \\> 5x upper limit of normal.\n* Serum bilirubin \\>4 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n* Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.\n* Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.",{"count":391,"type":21},37,[59,175],"Background:\n\nSevere aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective.\n\nObjective:\n\nTo test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.\n\nEligibility:\n\nRecipients ages 4-60 with SAA or MDS\n\nDonors ages 4-75\n\nDesign:\n\nRecipients will be screened with:\n\n* Blood, lung, and heart tests\n* Bone marrow biopsy\n* CT scan\n\nRecipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose.\n\nRecipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits:\n\n* First 3-4 months: 1-2 times weekly\n* Then every 6 months for 5 years\n\nDonors will be screened with:\n\n* Physical exam\n* Medical history\n* Blood tests\n\nDonors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein.\n\nDonors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.",[395,396,397],"Severe Aplastic Anemia","Hypo-Plastic MDS","Myelodysplastic Syndrome (MDS)",[399,400],"Haploidentical","Nonmyeloablative",{"date":199,"type":40},{"date":403,"type":40},"2017-06-13",{"date":405,"type":21},"2032-03-01",{"name":46,"class":47},{"id":408,"slug":4,"hasResults":11,"nctId":409,"briefTitle":410,"officialTitle":411,"acronym":4,"eligibilityCriteria":412,"healthyVolunteers":144,"sex":16,"minAge":413,"maxAge":18,"enrollmentInfo":414,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":416,"conditions":417,"keywords":419,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":422,"startDateStruct":423,"completionDateStruct":425,"leadSponsor":427,"locationsCount":48},"100250836","NCT02543996","Human Biospecimen Procurement Protocol: Biorepository to Support Translational Research to Identify Disease Mechanism(s)","Human Biospecimen Procurement and Analysis to Support Translational Research to Identify Genetic Etiology and Disease Mechanism(s) in Rare Genetic Vascular\u002FCardiovascular Diseases","* INCLUSION CRITERIA (SUBJECTS MUST MEET ONE OF THE FOLLOWING):\n* Age: older than 1 month of age\n* Affected pregnant women if they have been referred with a known or suspected pathology or if they become pregnant while on study.\n* Unaffected related pregnant women (including spouses\u002Fpartners) for cord blood and tissue collection (surgical waste) only at the time of delivery.\n* Cognitively impaired individuals that are affected\n* Cognitively impaired individuals that are related to an affected subject.\n* Subjects willing to provide informed consent.\n\nEXCLUSION CRITERIA:\n\n* Healthy volunteers unable to give informed consent\n* Cognitively impaired individuals who are not affected\n* Cognitively impaired individuals who are not related to affected subjects.","1 Month",{"count":415,"type":21},10000,"Background:\n\nStudies show that rare genetic variants might lead to diseases. Researchers want to collect blood and tissue samples so they can study them and better understand diseases.\n\nObjective:\n\nTo collect blood and tissue samples for studies to identify underlying causes of disease.\n\nEligibility:\n\nPeople of all ages\n\nDesign:\n\nParticipants will have blood and\u002For tissue samples collected.\n\nSamples can be collected at the NIH Clinical Center. Participants doctors can collect the samples and send them to NIH. NIH staff can collect samples off site.\n\nFor blood samples, blood is taken from an arm vein using a needle.\n\nTissue collection may involve:\n\nBuccal smear: Cells are collected by scraping the inside of the cheek with a cotton swab.\n\nSaliva collection: Participants spit into a cup.\n\nSkin biopsy: A special needle takes a very small skin sample.\n\nSurgical waste tissue: If participants have surgery, NIH may receive samples of tissue that\n\nwould routinely be removed.\n\nUmbilical cord or cord blood collection: If a participant has a baby, NIH may receive a\n\nsmall piece of the umbilical cord or blood from the cord once the baby is delivered.\n\n...",[418,320],"Undiagnosed Diseases",[420,327,326,421,106],"Sample Collection","Rare Diseases",{"date":199,"type":40},{"date":424,"type":40},"2015-09-17",{"date":426,"type":21},"2050-05-22",{"name":46,"class":47},{"id":429,"slug":4,"hasResults":11,"nctId":430,"briefTitle":431,"officialTitle":431,"acronym":4,"eligibilityCriteria":432,"healthyVolunteers":144,"sex":433,"minAge":17,"maxAge":434,"enrollmentInfo":435,"targetDuration":4,"studyType":22,"phases":437,"briefSummary":438,"conditions":439,"keywords":441,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":444,"lastUpdatePostDateStruct":445,"startDateStruct":446,"completionDateStruct":448,"leadSponsor":450,"locationsCount":48},"100518621","NCT06032923","Double-blind Placebo Controlled Study to Evaluate the Effect of NAD+ Boosting With Nicotinamide Riboside on Immunometabolism and Immunity in Systemic Lupus Erythematosus","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\nSLE subjects:\n\n* Female subjects 18 years or older who meets \\> 3 of 11 modified Am. Coll. of Rheumatology (ACR) (1997) Revised Criteria for SLE and mild\u002Fmoderate disease activity defined as an SLE Disease Activity Index 2000(SLEDAI 2K) between zero and less than or equal to 14 at screening;\n* If on glucocorticoids, the dose must be less than or equal to 20 mg daily and stable for at least 4 weeks prior to screening;\n* If on hydroxychloroquine or other antimalarials such as chloroquine or quinacrine, dose must have been stable for the 12 weeks prior to screening. The max. allowed doses - hydroxychloroquine 400 mg\u002Fday, chloroquine phosphate 500 mg\u002Fday and quinacrine 100 mg\u002Fday;\n* If on immunosuppressive drugs (methotrexate, azathioprine, mycophenolate mofetil, cyclosporine, tacrolimus); dose must have been stable for the 12 weeks prior to screening\n* Subjects of childbearing potential must agree to practice effective birth control for the duration of the study;\n* Stated willingness to comply with all study procedures and availability for the duration of the study;\n* Agreement to adhere to Lifestyle Considerations throughout study duration;\n* Ability of subject to understand and the willingness to sign a written informed consent document.\n* If on vitamin B3 or tryptophan supplementation at screening, willing to stop it at least 6 weeks before the baseline visit.\n\nControl subjects:\n\n* Female subjects 18 years or older\n* No history of autoimmune or inflammatory disease\n* If on vitamin B3 or tryptophan supplementation at screening, willing to stop it at least 6 weeks before the blood draw visit.\n\nEXCLUSION CRITERIA:\n\nSLE Subjects:\n\n* Active renal or central nervous system disease or major renal or hepatic dysfunction;\n* Treatment with rituximab, belimumab or any other biologic agent within the 6 months prior to screening\n* Treatment with cyclophosphamide or IVIG within the 6 months prior to screening and or increase in glucocorticoid dose within 4 weeks of screening;\n* Pregnancy or lactation (nursing)\n* Treatment with another investigational drug or other intervention within 6 months of screening\n\nControl Subjects:\n\n* Inability to sign consent\n* Pregnancy or nursing\n\nPregnant women are excluded from participation on this study. Self-reported pregnancy status may be accepted from female control participants of child-bearing potential for a blood draw which is considered a minimal risk procedure.","FEMALE","120 Years",{"count":436,"type":21},78,[59,175],"Study Description:\n\nSystemic lupus erythematosus (SLE) occurs predominantly in women and is driven by type I interferon dysregulation and neutrophil hyperresponsiveness. Neutrophils in females have reduced mitochondrial bioenergetic capacity which affects immunometabolism. Nicotinamide adenine dinucleotide (NAD)+ boosting with nicotinamide riboside blunts type 1 IFN activation in-vivo in monocytes of healthy subjects and ex-vivo in SLE subjects. These findings support the proposal of the hypothesis that NAD+ boosting by NR supplementation will modulate metabolic pathways in lupus and blunt type 1 interferon signaling. Moreover, as type 1 interferon drives endothelial dysfunction, linked to increased cardiovascular risk, the effect of NR on endothelial function will be examined.\n\nObjectives:\n\nPrimary Objective: Evaluate the effect of NR vs. placebo on immunometabolic and inflammatory remodeling in female SLE subjects:\n\nExploratory Objective: Compare and characterize myeloid cell bioenergetic and immunometabolic profiles in healthy control and SLE female subjects\n\nEndpoints:\n\nPrimary Endpoint:\n\nThe primary end point will be to assess the effect of NR on blunting type I IFN signaling by measuring monocytic secretion of IFN-beta secretion compared to baseline in response to placebo vs. NR supplemented in SLE study subjects.\n\nExploratory Endpoints:\n\nHealthy control vs. SLE subjects:\n\n* Compare type I IFN transcript profiles in monocytes and neutrophils at baseline and in response to activation.\n* Assess cell bioenergetics including: 1) monocyte and neutrophil metabolic flux mass spectroscopy of 13C-glucose and 13Cglutamine analysis to investigate their metabolic fates; (iii) Mitochondrial oxygen consumption (using glucose, amino acid, and fatty acid substrates) and glycolysis rates.\n\nSLE baseline vs. NR\u002Fplacebo supplementation:\n\nBaseline vs. 6 weeks of NR\u002Fplacebo:\n\n-Assess effect of NR on bioenergetics by measuring steady-state metabolite levels comparing changes in placebo vs. NR groups in monocytes and neutrophils.\n\nBaseline vs. 12 weeks of NR\u002Fplacebo:\n\n* Whole blood NAD+ levels (batched and measured at the end of study enrollment period)\n* Explore effects of NR on gene regulation using monocyte and neutrophils by RNA-seq and chromatin remodeling analysis.\n* Determine the effect of NR vs placebo on endothelial dysfunction in SLE subjects",[440],"Systemic Lupus Erythematosus (Sle)",[442,443],"Lupus","Chronic Inflammation","2026-06-30",{"date":197,"type":40},{"date":447,"type":40},"2024-03-13",{"date":449,"type":21},"2028-08-01",{"name":46,"class":47},{"id":452,"slug":4,"hasResults":11,"nctId":453,"briefTitle":454,"officialTitle":455,"acronym":4,"eligibilityCriteria":456,"healthyVolunteers":144,"sex":16,"minAge":457,"maxAge":18,"enrollmentInfo":458,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":460,"conditions":461,"keywords":468,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":444,"lastUpdatePostDateStruct":474,"startDateStruct":475,"completionDateStruct":4,"leadSponsor":477,"locationsCount":478},"100143838","NCT01143454","Characterization of Patients With Uncommon Presentations and\u002For Uncommon Diseases Associated With the Cardiovascular System","Cardiovascular Disease Discovery Protocol","* INCLUSION CRITERIA:\n\nEligible subjects may include anyone over 1 year of age who is affected with diseases\u002Fdisorders (index cases), or who is a relative of a person who is affected with diseases\u002Fdisorders. Relatives may include genetic carriers and non-carriers.\n\n* Healthy adult volunteers must be 18 years of age or older, and must agree to have blood or tissue samples studied, and potentially stored for future research.\n* Index cases enrolled in this protocol will have been referred with a known or suspected pathology that may be associated with cardiovascular dysfunction or risk with a suspected atypical presentation, heritable disorder, or genetic predisposition. The investigator with expertise in the presentation of the subject, along with consulting specialists, will review the medical history and may review any medical records that are available of prospective subjects and offer admission based upon the potential to help the individual, to learn from the subject, or to initiate clinical or basic research suggested by the subject s workup.\n\nEXCLUSION CRITERIA:\n\n* Persons of less than 1 year of age or greater than 100 years of age\n* Healthy volunteers unable to give informed consent or who decline to have blood and\u002For tissue studies, or who do not consent to have samples stored for future research may be excluded from this study.\n* Pregnant women\n* Persons who are not fluent in the English language will be excluded from Patient Reported Outcome Questionnaires. Such persons would be unable to properly complete questionnaires that are only valid in the English language.","1 Year",{"count":459,"type":21},5000,"Background:\n\n\\- Researchers are interested in studying individuals who have known or suspected metabolic, inflammatory or genetic diseases that may put them at a high risk for heart diseases or diseases of their blood vessels. Depending on the condition being studied, both affected and nonaffected individuals may be asked to provide blood and other samples and may undergo tests to evaluate the heart, blood vessels and lung function. The testing is tailored to the individual and\u002For condition being studied. Nonaffected individuals may include relatives of affected individuals and healthy nonrelated volunteers.\n\nObjectives:\n\n\\- To study individuals who have or are at risk for cardiovascular diseases, and in some cases their unaffected relatives and healthy volunteers.\n\nEligibility:\n\n\\- Individuals between 1 and 100 years of age. Participants may be healthy volunteers, individuals with cardiovascular diseases, or unaffected relatives of individuals with cardiovascular diseases.\n\nDesign:\n\n* Participants will have some or all of the following tests, as directed by the study researchers:\n* Photography of the face and full body\n* Body measurements\n* Radiography, including chest or limb x-rays\n* Metabolic stress testing to study heart and muscle function\n* Echocardiography to study heart function\n* Magnetic resonance imaging (MRI) studies, including cardiovascular MRI, angiography, and contrast MRI, to study heart function and performance\n* Computed tomography (CT) angiogram to obtain images of the heart and lungs\n* Positron emission tomography (PET) imaging to study possible fat infiltration of the heart\n* Six-minute walk test to study heart, lung, and muscle function and performance\n* Vascular ultrasound to study blood vessel walls\n* Blood, tissue, and other specimens will be collected for research and testing, and will be taken either as part of the clinical study or during surgical procedures.\n* Follow-up studies may be performed under separate research protocols.",[462,463,464,465,466,467],"Metabolic Disease","Obesity","Li-Fraumeni Syndrome","Cardiomyopathy","Atherosclerosis","Inflammatory Disease",[469,470,471,472,106,104,473],"Cardiac Disease","iPS Cells","Cardiac Risk Factors","Cardiac Disease Discovery","Heart Disease Risk",{"date":197,"type":40},{"date":476,"type":40},"2010-07-21",{"name":46,"class":47},3,{"id":480,"slug":4,"hasResults":11,"nctId":481,"briefTitle":482,"officialTitle":483,"acronym":4,"eligibilityCriteria":484,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":55,"enrollmentInfo":485,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":487,"conditions":488,"keywords":490,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":494,"lastUpdatePostDateStruct":495,"startDateStruct":496,"completionDateStruct":498,"leadSponsor":499,"locationsCount":48},"100241170","NCT02417714","Prospective Evaluation of Next Generation CT Reconstruction (NextGenIR)","Prospective Evaluation of Next Generation CT Reconstruction","* INCLUSION CRITERIA:\n\nInclusion Criteria for Body CT:\n\n1. Clinical indication for a chest, abdomen and pelvis CT with contrast\n2. Age equal to or greater than 18 years\n3. Able to understand and willing to sign the Informed Consent Form\n\nInclusion Criteria for Chest CT:\n\n1. Clinical indication for a CT with or without contrast that includes the chest\n2. Age equal to or greater than 18 years\n3. Able to understand and willing to sign the Informed Consent Form\n\nEXCLUSION CRITERIA:\n\nExclusion Criteria for Body CT:\n\n1. Pregnancy\n2. Serum Cr \\> 1.5 mg\u002FdL\n3. Clinical CT scan with more than one contrast imaging phase (for example three phase liver)\n\nExclusion Criteria for Chest CT\n\n1\\. Pregnancy",{"count":486,"type":21},215,"Background:\n\n\\- Computed Tomography (CT) is a scan that makes detailed pictures of the body. It uses radiation to do that. In 2011, about 85.3 million CT scans were done in the United States. But there is growing concern about the health effects of radiation. There are new and different ways to make CT pictures that use less radiation. Researchers want to learn whether these new methods create images that are similar to images from a traditional CT.\n\nObjective:\n\n\\- To learn if new CT imaging methods with less radiation make images that are similar to traditional ways.\n\nEligibility:\n\n\\- Adults 18 years of age and older who are scheduled for a CT of the chest, abdomen and pelvis with contrast.\n\nDesign:\n\n* Researchers will review participants medical records to see if they can be in this study.\n* Participants may have blood drawn from an arm vein by a needle stick. The blood will be used to make sure they can be in this study and that it is safe for them to have contrast.\n* During the participants CT scan appointment, one extra scan will be done using low radiation methods.\n* During a CT scan, the participant lies on a table. A large x-ray machine takes pictures of the body.",[489],"Healthy Subjects",[491,492,493,106],"Computed Tomography","Radiation Reduction","Iterative Reconstruction","2026-06-27",{"date":444,"type":40},{"date":497,"type":40},"2014-09-08",{"date":197,"type":21},{"name":46,"class":47},{"id":501,"slug":4,"hasResults":11,"nctId":502,"briefTitle":503,"officialTitle":504,"acronym":4,"eligibilityCriteria":505,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":506,"targetDuration":4,"studyType":22,"phases":507,"briefSummary":508,"conditions":509,"keywords":511,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":494,"lastUpdatePostDateStruct":514,"startDateStruct":515,"completionDateStruct":517,"leadSponsor":519,"locationsCount":48},"100180310","NCT01621594","Evaluating New Radiation Techniques for Cardiovascular Imaging","Prospective Evaluation of New Techniques in Radiation Reduction for Cardiovascular Computed Tomographic Angiography","* INCLUSION CRITERIA:\n\n  1. Clinical indication for a coronary CT angiography exam\n  2. Age equal to or greater than 18 years\n  3. Able to understand and willing to sign the Informed Consent Form\n  4. Able and willing to provide follow-up information\n\nEXCLUSION CRITERIA:\n\n1. Pregnancy\n2. If receiving intravenous contrast, severe renal excretory dysfunction, estimated glomerular filtration rate \\\u003C 30 mL\u002Fmin\u002F1.73m2 body surface area according to the Modification of Diet in Renal Disease criteria or as reported by the clinical lab. Glomerular filtration rate will be estimated using the 2021 CK-EPI equation that eliminate the use of race coefficient as reported in CRIS.\n\nScreen Failures: Subjects who are screen failures can be re-enrolled once they meet eligibility criteria. Rescreened participants will be assigned the same participant number as for the initial screening.",{"count":459,"type":21},[368],"Title: Evaluating New Radiation Techniques for Cardiovascular Imaging\n\nBackground:\n\nCardiac CT angiography is associated with radiation exposure. Different methods of creating CT pictures have been developed to reduce the radiation dose to the subject. The purpose of this research study is to learn whether these low dose research imagings are accurate or predict subject outcomes.\n\nCardiac CT is also used for diagnostic imaging of coronary artery disease and identification of abnormal cardiac structures. An additional purpose of this study is to monitor the progression of cardiac disease.\n\nCardiac imaging software and AI are constantly evolving and requires validation for accuracy. Using existing scan data, updated image software reconstruction can be applied and compared to previous existing standard of care images.\n\nObjectives:\n\n\\- To study new ways of taking pictures of the heart or blood vessels using computed tomography.\n\nEligibility:\n\n\\- Adults at least 18 years of age who will be having imaging studies to help detect heart or blood vessel problems.\n\nDesign:\n\n* Participants will be screened with a physical exam and medical history. Blood samples will be taken to check kidney function.\n* Participants will have a CT scan of the heart and blood vessels. A contrast agent may be used to improve the quality of the images. The scanning session may last up to 2 hours.\n* Timing of and the need for follow up contact will depend on results from the initial scan and may be repeated to assess for late events. Telephone, office contact, or other follow-up of subjects may be done after CCTA to evaluate if the subject had subsequent cardiovascular testing. Further follow up will be based on reported test results.",[510],"Coronary Disease",[512,513,492,493],"computer tomography","Cardiac",{"date":444,"type":40},{"date":516,"type":40},"2012-06-21",{"date":518,"type":21},"2027-04-01",{"name":46,"class":47},{"id":521,"slug":4,"hasResults":11,"nctId":522,"briefTitle":523,"officialTitle":524,"acronym":4,"eligibilityCriteria":525,"healthyVolunteers":11,"sex":16,"minAge":170,"maxAge":526,"enrollmentInfo":527,"targetDuration":4,"studyType":22,"phases":529,"briefSummary":530,"conditions":531,"keywords":533,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":494,"lastUpdatePostDateStruct":536,"startDateStruct":537,"completionDateStruct":539,"leadSponsor":541,"locationsCount":112},"100146179","NCT01174108","Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells","Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-Cells","* INCLUSION CRITERIA:\n* Recipient:\n\n  * Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional BMT may delay or prevent such treatment:\n\n    * 1\\) Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and\u002For cytopenia, and\u002For transfusion dependence and\u002For recurrent and debilitating hemolytic crisis\n    * 2\\) Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA \\[acquired or congenital\\]) with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count \\\u003C=1000 cells\u002FuL or for patients receiving granulocyte transfusions, absolute neutrophil count \\\u003C=1000 cells\u002F uL before beginning granulocyte transfusions). in newly diagnosed patients and\u002For in patients who have failed immunosuppressive therapy.\n    * 3\\) Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and\u002For neutropenia.\n  * Ages 4 to 80 (both inclusive), and weight \\>15 kg\n  * Availability of HLA identical or single HLA locus mismatched family donor or 10\u002F10 matched unrelated donor at the allelic level (HLA alleles A, B, C, DR, and DQ).\n  * 9\u002F10 donors where all the HLA sequences have the same antigen\u002Fpeptide binding domains in key exons to the patient. This can result in identical protein sequences between patient and donor. Allele mismatches in p and g groups can be considered acceptable due to the exact matching which exists in the binding domains.\n  * Telomere Length Testing\n  * Germline\u002FInherited gene panel in patients where a suspicion for a familial bone marrow failure syndrome (BMFS) exist, hTERC and hTERT, GATA2 mutation testing will be performed on protocol 04-H-0012 or performed elsewhere prior to enrolling on 04-H-0012.\n\nEXCLUSION CRITERIA:\n\n\\- Recipient: any of the following\n\n* Major anticipated illness or organ failure incompatible with survival from PBSC transplant\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% predicted (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed) using DL Adj and DL\u002FVA\u002FAdj.\n* Left ventricular ejection fraction \\\u003C40% (evaluated by ECHO)\n* Serum creatinine greater than 2.5mg\u002Fdl or creatinine clearance less than 50 ml\u002Fmin by 24 hr urine collection\n* Serum bilirubin greater than 4 mg\u002Fdl, transaminases greater than 5 times the upper limit of normal\n* Pregnant or lactating\n* Fanconi s anemia (test to be performed at a CLIA-certified laboratory)\n* ECOG performance status of 3 or more (See NIH Bone \\& Marrow Transplant Consortium Supportive Care Guidelines for HSCT Recipients or Institutional Guidelines for bone and marrow transplants)\n* Other malignant diseases liable to relapse or progress within 5 years, with the exception of a separate hematologic malignancy where allogeneic stem cell transplant has been shown to be potentially curative.\n* Presence of an active infection not adequately responding to appropriate therapy.\n* Inability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects age 8 -17 years with formal consent being obtained from parents or legal guardian.\n\nINCLUSION CRITERIA:\n\n-Related Donor:\n\n* Related donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood samples for research. Related donors will be evaluated in accordance with existing Standard Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all related donors, but study participation is not required for a donor to make a stem cell donation, so it is possible that not all related donors will enroll onto this study\n* Age greater than or equal to 4 and less than or equal to 80 years old\n\nEXCLUSION CRITERIA:\n\n-Related Donor: None\n\nINCLUSION CRITERIA \\& EXCLUSION CRITERIA: Unrelated Donor\n\n\\- The NMDP unrelated donor inclusion criteria will be used as outlined in document (http:\u002F\u002Fbethematch.org\u002FWorkArea\u002FDownloadAsset.aspx?id=1960). Donor eligibility will be completed per NMDP standards and in accordance with most recent and stringent FDA guidelines.","80 Years",{"count":528,"type":21},120,[175],"Background:\n\n* Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications.\n* By reducing the number of white blood cells included in the blood taken during the stem cell collection process, and replacing them with a smaller amount of white blood cells collected prior to stem cell donation, the stem cell transplant may be less likely to cause severe complications for the recipient. Researchers are investigating whether altering the stem cell transplant donation procedure in this manner will improve the likelihood of a successful stem cell transplant with fewer complications.\n\nObjectives:\n\n\\- To evaluate a new method of stem cell transplantation that may reduce the possibly of severe side effects or transplant rejection in the recipient.\n\nEligibility:\n\n* Recipient: Individuals between 4 and 80 years of age who have been diagnosed with a blood disease that can be treated with allogenic stem cell transplants.\n* Donor: Individuals between 4 and 80 years of age who are related to the recipient and are eligible to donate blood. OR unrelated donors found through the National Marrow Donor Program.\n\nDesign:\n\n* All participants will be screened with a physical examination and medical history.\n* DONORS:\n* Donors will undergo an initial apheresis procedure to donate white blood cells.\n* After the initial donation, donors will receive injections of filgrastim to release bone marrow cells into the blood.\n* After 5 days of filgrastim injections, donors will have apheresis again to donate stem cells that are present in the blood.\n* RECIPIENTS:\n* Recipients will provide an initial donation of white blood cells to be used for research purposes only.\n* From 7 days before the stem cell transplant, participants will be admitted to the inpatient unit of the National Institutes of Health Clinical Center and will receive regular doses of cyclophosphamide, fludarabine, and anti-thymocyte globulin to suppress their immune system and prepare for the transplant.\n* After the initial chemotherapy, participants will receive the donated white blood cells and stem cells as a single infusion.\n* After the stem cell and white blood cell transplant, participants will have regular doses of cyclosporine and methotrexate to prevent rejection of the donor cells. Participants will have three doses of methotrexate within the week after the transplant, but will continue to take cyclosporine for up to 4 months after the transplant.\n* Participants will remain in inpatient care for up to 1 month after the transplant, and will be followed with regular visits for up to 3 years with periodic visits thereafter to evaluate the success of the transplant and any side effects.",[395,532],"MDS (Myelodysplastic Syndrome)",[397,395,534,350,535],"Pure Red Cell Aplasia","Miltenyi CD34 Reagent System",{"date":444,"type":40},{"date":538,"type":40},"2010-12-10",{"date":540,"type":21},"2028-06-30",{"name":46,"class":47},""]