[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Alnylam Pharmaceuticals\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":576},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,23,0,[8,50,75,105,134,156,181,199,218,237,261,287,298,330,351,377,404,427,448,471,497,531,552],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100054235",false,"NCT07181109","Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease","ZENITH: A Phase 3 Global, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Zilebesiran in Addition to Standard of Care in Reducing Major Adverse Cardiovascular Events in Adult Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease","ZENITH","Inclusion Criteria:\n\n* Is 18 years or older for patients with established cardiovascular disease (CVD)\n* Is 55 years or older for patients with high risk for CVD\n* Has established CVD (defined as coronary, cerebrovascular, or peripheral artery disease) or high risk for CVD\n* Has treated hypertension on stable therapy with at least 2 standard of care antihypertensive medications, one of which must be a thiazide, thiazide-like, or loop diuretic\n\nExclusion Criteria:\n\n* Has known history of secondary hypertension\n* Has symptomatic orthostatic hypotension\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>3×upper limit of normal (ULN)\n* Has total serum bilirubin \\>1.5×ULN\n* Has international normalized ratio (INR) \\>1.5\n* Has serum potassium \\>4.8 mEq\u002FL\n* Has estimated glomerular filtration rate (eGFR) \\\u003C30 mL\u002Fmin\u002F1.73m\\^2","ALL","18 Years",{"count":19,"type":20},11000,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","The purpose of this study is to evaluate whether zilebesiran versus placebo reduces the risk of cardiovascular (CV) death, nonfatal myocardial infarction (MI), nonfatal stroke, or heart failure (HF) events. This is an event-driven study that will continue until the targeted number of positively adjudicated primary endpoint clinical outcome events (COEs) have been reached.",[26,27,28],"High Risk Cardiovascular Disease","Hypertension","High Cardiovascular Risk",[30,31,32,33,34,35,36],"High blood pressure","siRNA","Angiotensinogen","AGT","Cardiovascular disease","Uncontrolled hypertension","RNAi","RECRUITING","2026-07-10",{"date":40,"type":41},"2026-07-13","ACTUAL",{"date":43,"type":41},"2025-09-22",{"date":45,"type":20},"2030-09-30",{"name":47,"class":48},"Alnylam Pharmaceuticals","INDUSTRY",995,{"id":51,"slug":4,"hasResults":10,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":56,"enrollmentInfo":57,"targetDuration":4,"studyType":21,"phases":59,"briefSummary":61,"conditions":62,"keywords":64,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":74},"100639878","NCT07624071","A Study to Evaluate ALN-6222 in Participants With Obesity","A Phase 1, Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of ALN-6222 in Adult Participants With Obesity","Inclusion Criteria -\n\n* Is an adult patient with a body mass index ≥30 kg\u002Fm\\^2 to \\\u003C40 kg\u002Fm\\^2\n* Has a hemoglobin A1c \\\u003C6.5%\n\nExclusion Criteria -\n\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>1.5 times the upper limit of normal (ULN)\n* Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection\n* Has an estimated glomerular filtration (eGFR) of \\\u003C60 mL\u002Fmin\u002F1.73m\\^2 at screening\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","65 Years",{"count":58,"type":20},88,[60],"PHASE1","This is a first in human, randomized, double-blind (DB), placebo-controlled, single ascending dose (SAD), Phase 1 study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of ALN-6222 administered subcutaneously (SC) in adult participants with obesity.",[63],"Obesity",[65],"siRNA, RNAi therapeutic","2026-06-25",{"date":68,"type":41},"2026-06-29",{"date":70,"type":41},"2026-06-04",{"date":72,"type":20},"2027-12-14",{"name":47,"class":48},1,{"id":76,"slug":4,"hasResults":10,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":21,"phases":83,"briefSummary":84,"conditions":85,"keywords":88,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":5},"100562219","NCT06600321","A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma","A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma","Inclusion Criteria:\n\n* Has HCC confirmed histologically or cytologically, or, for patients with liver cirrhosis, clinically by the American Association for the Study of Liver Diseases (AASLD) criteria\n* Has had at least one line of systemic therapy for unresectable advanced or metastatic disease\n* Has at least one wingless-related integration site (WNT)-pathway activating mutation\n* Child-Pugh class A or B7\n\nExclusion Criteria:\n\n* Has fibrolamellar HCC, sarcomatoid HCC, or mixed cholangio-HCC tumors\n* Has symptomatic extrahepatic disease\n* Has received anti-cancer therapy or investigational drugs ≤3 weeks prior to the first dose of study drug\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply",{"count":82,"type":20},158,[60],"The purpose of the dose escalation part of the study is to characterize the safety and tolerability of ALN-BCAT as monotherapy and in combination with pembrolizumab; and to determine the recommended dose(s) for expansion (RDFE) of ALN-BCAT as monotherapy and in combination with pembrolizumab. The purpose of the dose expansion part of the of the study is to evaluate the antitumor activity of ALN-BCAT as monotherapy and in combination with pembrolizumab; to characterize the safety and tolerability of ALN-BCAT as monotherapy and in combination with pembrolizumab.",[86,87],"Advanced Hepatocellular Carcinoma","Metastatic Hepatocellular Carcinoma",[89,90,91,92,93,94,95,96],"Hepatocellular carcinoma (HCC)","Pembrolizumab","CTNNB1","Liver disease","Liver cancer","Liver neoplasms","Carcinoma, hepatocellular","WNT pathway activating","2026-06-16",{"date":99,"type":41},"2026-06-17",{"date":101,"type":41},"2024-12-30",{"date":103,"type":20},"2027-10-31",{"name":47,"class":48},{"id":106,"slug":4,"hasResults":10,"nctId":107,"briefTitle":108,"officialTitle":109,"acronym":110,"eligibilityCriteria":111,"healthyVolunteers":10,"sex":112,"minAge":113,"maxAge":114,"enrollmentInfo":115,"targetDuration":4,"studyType":21,"phases":117,"briefSummary":119,"conditions":120,"keywords":123,"overallStatus":125,"whyStopped":4,"lastUpdateSubmitDate":126,"lastUpdatePostDateStruct":127,"startDateStruct":129,"completionDateStruct":131,"leadSponsor":133,"locationsCount":74},"100643166","NCT07575308","HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB","HMBeacon: A Phase 2, Randomized, Double-blind Study of the Safety, Tolerability, Efficacy, and Pharmacodynamics of Multiple Dose ALN-6400 in Adult and Adolescent Female Patients With Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)","HMBeacon","Inclusion Criteria -\n\n* Is an adolescent or adult patient with a diagnosis of VWD (including Type 1, Type 2, Type 3, and platelet-type VWD) and HMB for 2 cycles during screening\n\nExclusion Criteria -\n\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2 times the upper limit of normal (ULN)\n* Has total bilirubin greater than 1.5×ULN (except for patients with Gilbert's syndrome)\n* Has an estimated glomerular filtration (eGFR) less than 30 mL\u002Fmin\u002F1.73m\\^2 at screening\n* Is not willing to comply with the contraceptive requirements during the study period\n* Used routine factor prophylaxis (greater than or equal to 1 infusion of factor per week over 12 weeks) within 4 weeks of screening or plans use for routine factor prophylaxis during the study\n* New placement of any hormonal or nonhormonal intrauterine device (IUD) or hormonal implants within 24 weeks of screening\n* Known current gynecological conditions causing abnormal uterine bleeding (including infection, fibroids, endometriosis, polycystic ovary syndrome, or dysplasia)\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","FEMALE","16 Years","45 Years",{"count":116,"type":20},24,[118],"PHASE2","The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB",[121,122],"Von Willebrand Disease (VWD)","Heavy Menstrual Bleeding (HMB)",[124],"siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB","NOT_YET_RECRUITING","2026-06-11",{"date":128,"type":41},"2026-06-15",{"date":130,"type":20},"2026-06-30",{"date":132,"type":20},"2028-11-29",{"name":47,"class":48},{"id":135,"slug":4,"hasResults":10,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":140,"sex":16,"minAge":17,"maxAge":56,"enrollmentInfo":141,"targetDuration":4,"studyType":21,"phases":143,"briefSummary":144,"conditions":145,"keywords":147,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":155,"locationsCount":74},"100634124","NCT07535606","A Study to Evaluate ALN-4915 in Adult Healthy Volunteers","A Phase 1, Randomized, Double-Masked, Placebo-controlled, Single Ascending Dose Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALN-4915 in Adult Healthy Volunteers","Inclusion Criteria:\n\n* Is willing and able to complete all study assessments\n\nExclusion Criteria:\n\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>upper limit of normal (ULN)\n* Has total bilirubin \\>ULN\n* Has no history of invasive infection by an encapsulated organism\n* Has no known complement or immunologic deficiency",true,{"count":142,"type":20},44,[60],"The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of ALN-4915.",[146],"Healthy Volunteers",[31,36],"2026-06-10",{"date":150,"type":41},"2026-06-12",{"date":152,"type":41},"2026-03-02",{"date":154,"type":20},"2027-07-31",{"name":47,"class":48},{"id":157,"slug":4,"hasResults":10,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":4,"eligibilityCriteria":161,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":162,"enrollmentInfo":163,"targetDuration":4,"studyType":21,"phases":165,"briefSummary":166,"conditions":167,"keywords":168,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":174,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":180},"100635496","NCT07553442","A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran","A Phase 2, Randomized, Double-blind, Placebo-controlled, Single Dose Study of the Efficacy, Pharmacodynamics, and Safety of ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran","Part A: Inclusion Criteria\n\n* Is an adult participant with a mean seated office systolic blood pressure (SBP) of at least 130 mmHg and no more than than 170 mmHg\n* Either not taking antihypertensive medication or on stable therapy with up to 2 antihypertensive medications\n\nPart B: Inclusion Criteria\n\n* Is an adult participant with a mean seated office SBP of at least 140 mmHg and no more than 170 mmHg\n* Has discontinued all prior antihypertensive medication other than a calcium channel blocker (CCB) and\u002For thiazide\u002Fthiazide-like diuretic (if taking) for at least 3 weeks\n\nBoth Parts: Exclusion Criteria\n\n* Has known secondary hypertension or serum potassium more than 5 mmol\u002FL\n* Has an estimated glomerular filtration rate (eGFR) less than 30 mL\u002Fmin\u002F1.73m\\^2\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","75 Years",{"count":164,"type":20},93,[118],"The purpose of this study is to evaluate the efficacy, pharmacodynamics (PD), and safety of ALN-AGT01 RVR in participants with mild to moderate hypertension pretreated with zilebesiran.",[27],[169,170,171,172,173],"RNAi therapeutic","oligonucleotide","Reversir","Zilebesiran","ALN-AGT01",{"date":150,"type":41},{"date":176,"type":41},"2026-04-27",{"date":178,"type":20},"2028-10-01",{"name":47,"class":48},3,{"id":182,"slug":4,"hasResults":10,"nctId":183,"briefTitle":184,"officialTitle":185,"acronym":4,"eligibilityCriteria":186,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":56,"enrollmentInfo":187,"targetDuration":4,"studyType":21,"phases":189,"briefSummary":190,"conditions":191,"keywords":192,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":194,"startDateStruct":195,"completionDateStruct":196,"leadSponsor":198,"locationsCount":74},"100628608","NCT07463846","A Study to Evaluate ALN-2232 in Participants With Obesity","A Phase 1\u002F2, Randomized, Double-blind, Placebo-controlled, Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALN-2232 as Monotherapy and Co-initiated With Tirzepatide in Adult Participants With Obesity","Inclusion Criteria:\n\nAll Parts:\n\n* Has a body mass index (BMI) of ≥30 kg\u002Fm\\^2 and \\\u003C40 kg\u002Fm\\^2\n* Has a hemoglobin A1c (HbA1c) \\\u003C6.5%\n\nExclusion Criteria:\n\nAll Parts:\n\n* Has any clinically significant concomitant disease, medical condition, or abnormal laboratory finding that could compromise participant safety or confound interpretation of study results\n* Receiving therapies for chronic weight management or antidiabetic medications\n\nNote: other protocol defined inclusion\u002Fexclusion criteria apply",{"count":188,"type":20},156,[60,118],"The purpose of this study is to:\n\n* evaluate the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending doses of ALN-2232 in patients with obesity\n* evaluate the safety, tolerability, efficacy, PK, and PD of multiple doses of ALN-2232 in patients with obesity\n* evaluate the safety, tolerability, efficacy, PK, and PD of multiple doses of ALN-2232 co-initiated with tirzepatide in patients with obesity",[63],[193],"siRNA, RNAi therepeutic",{"date":150,"type":41},{"date":152,"type":41},{"date":197,"type":20},"2028-03-02",{"name":47,"class":48},{"id":200,"slug":4,"hasResults":10,"nctId":201,"briefTitle":202,"officialTitle":203,"acronym":4,"eligibilityCriteria":204,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":56,"enrollmentInfo":205,"targetDuration":4,"studyType":21,"phases":207,"briefSummary":208,"conditions":209,"keywords":211,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":212,"startDateStruct":213,"completionDateStruct":215,"leadSponsor":217,"locationsCount":74},"100628713","NCT07465224","A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus","A Randomized, Double-blind, Placebo-controlled Study Investigating the Effect of ALN-4324 on Insulin Sensitivity in Patients With Type 2 Diabetes Mellitus","Inclusion Criteria:\n\n* Is an adult patient with a confirmed diagnosis of T2DM\n* Has a body mass index (BMI) of ≥25 kg\u002Fm\\^2 and \\\u003C39.9 kg\u002Fm\\^2\n* Has a hemoglobin A1c (HbA1c) ≥6.5% to \\\u003C10.5%\n* Is on a stable dose of metformin\n\nExclusion Criteria:\n\n* Has any clinically significant concomitant disease, medical condition, or abnormal laboratory finding that could compromise participant safety or confound interpretation of study results\n* Receiving therapies known to interfere with glucose or insulin metabolism other than current treatment for T2DM or birth control methods\n\nNote: other protocol defined inclusion\u002Fexclusion criteria apply",{"count":206,"type":20},40,[118],"The purpose of this study is to evaluate the effect of a single dose of ALN-4324 on whole-body insulin sensitivity in participants with T2DM",[210],"Type 2 Diabetes Mellitus (T2DM)",[31,36],{"date":150,"type":41},{"date":214,"type":41},"2026-01-15",{"date":216,"type":20},"2027-05-28",{"name":47,"class":48},{"id":219,"slug":4,"hasResults":10,"nctId":220,"briefTitle":221,"officialTitle":222,"acronym":4,"eligibilityCriteria":223,"healthyVolunteers":140,"sex":16,"minAge":17,"maxAge":56,"enrollmentInfo":224,"targetDuration":4,"studyType":21,"phases":226,"briefSummary":227,"conditions":228,"keywords":229,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":231,"startDateStruct":232,"completionDateStruct":234,"leadSponsor":236,"locationsCount":74},"100615677","NCT07295717","A Study to Evaluate ALN-4285 in Adult Healthy Volunteers","A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-4285 in Adult Healthy Volunteers","Inclusion Criteria:\n\n* Has a body mass index (BMI) of ≥18.0 kg\u002Fm\\^2 and ≤30 kg\u002Fm\\^2\n* Has 12-lead ECG within normal limits or with no clinically significant abnormalities in the opinion of the Investigator\n\nExclusion Criteria:\n\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>upper limit of normal (ULN)\n* Has total bilirubin \\>ULN\n* Has systolic blood pressure \\>140 mmHg and\u002For a diastolic blood pressure \\>90 mmHg\n* Has an estimated glomerular filtration (eGFR) of \\\u003C90 mL\u002Fmin\u002F1.73m\\^2 at screening\n* Has received an investigational agent within the last 30 days or 5 half-lives",{"count":225,"type":20},76,[60],"The purpose of this study is to:\n\n* evaluate the safety and tolerability of single ascending doses of ALN-4285 in healthy volunteers\n* characterize the single-dose pharmacokinetics (PK) of ALN-4285",[146],[31,230],"RNAi therapeutics",{"date":150,"type":41},{"date":233,"type":41},"2025-12-12",{"date":235,"type":20},"2027-03-23",{"name":47,"class":48},{"id":238,"slug":4,"hasResults":10,"nctId":239,"briefTitle":240,"officialTitle":241,"acronym":4,"eligibilityCriteria":242,"healthyVolunteers":10,"sex":16,"minAge":243,"maxAge":244,"enrollmentInfo":245,"targetDuration":4,"studyType":21,"phases":247,"briefSummary":248,"conditions":249,"keywords":251,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":254,"startDateStruct":255,"completionDateStruct":257,"leadSponsor":259,"locationsCount":260},"100609449","NCT07214727","A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease","A Phase 1, Randomized, Placebo-controlled Study With a Double Blind Period With Open-label Extension Period to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-5288 in Adult Patients With Alzheimer's Disease","Inclusion Criteria:\n\n* Is able and willing to meet all study requirements in the opinion of the Investigator\n* Has a diagnosis of Alzheimer's disease (AD) based on clinical findings supported by cerebrospinal fluid (CSF) biomarkers or positive positron emission tomography (PET) amyloid imaging within 7 years prior to screening\n* Has mild cognitive impairment (MCI) or dementia due to AD\n\nExclusion Criteria:\n\n* Has non-AD dementia\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2× upper limit of normal (ULN)\n* Has total bilirubin \\>1.5×ULN\n* Has known human immunodeficiency virus infection\n* Has history of hepatitis C virus or current hepatitis B virus infection\n* Has systolic blood pressure \\>160 mmHg and\u002For a diastolic blood pressure \\>100 mmHg after 10 minutes of rest at screening\n* Has an estimated glomerular filtration (eGFR) of \\\u003C45 mL\u002Fmin\u002F1.73 m\\^2 at screening\n* Has clinically significant ECG abnormalities at screening\n* Has uncontrolled psychiatric disease, including patients deemed by the Investigator to be at significant risk of suicide, major depressive episode, psychosis, confusional state, or violent behavior\n* Has history of bleeding diathesis or coagulopathy due to chronic conditions\n* Has a medical history of brain or spinal disease that would interfere with the IT injection and LP procedures\n* Has history of uncontrolled seizures within the last 6 months prior to Screening","40 Years","80 Years",{"count":246,"type":20},50,[60],"The purpose of this study is to:\n\n* Evaluate the safety and tolerability of intrathecal (IT) ALN-5288 in patients with Alzheimer's Disease (AD)\n* Evaluate the pharmacodynamic (PD) and pharmacokinetic (PK) effects of ALN-5288 after dose administration",[250],"Alzheimer's Disease",[252,253],"AD","Dementia",{"date":150,"type":41},{"date":256,"type":41},"2025-10-15",{"date":258,"type":20},"2030-03-06",{"name":47,"class":48},13,{"id":262,"slug":4,"hasResults":10,"nctId":263,"briefTitle":264,"officialTitle":265,"acronym":266,"eligibilityCriteria":267,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":268,"enrollmentInfo":269,"targetDuration":4,"studyType":21,"phases":271,"briefSummary":272,"conditions":273,"keywords":276,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":281,"startDateStruct":282,"completionDateStruct":283,"leadSponsor":285,"locationsCount":286},"100610101","NCT07223203","TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy","TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy (hATTR-PN)","TRITON-PN","Inclusion Criteria:\n\n* Has documented diagnosis of hATTR-PN\n* Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant\n* Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive)\n* Has a Karnofsky Performance Status (KPS) of ≥60%\n\nExclusion Criteria:\n\n* Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study\n* Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis\n* Has a New York Heart Association (NYHA) heart failure classification \\>2\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2.5 upper limit of normal (ULN)\n* Has total bilirubin \\>1.5 ULN\n* Has estimated glomerular filtration rate (eGFR) ≤30 mL\u002Fmin\u002F1.73m\\^2\n* Has other known causes of sensorimotor or autonomic neuropathy","85 Years",{"count":270,"type":20},125,[23],"The purpose of this study is to:\n\n* Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed\n* Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels",[274,275],"Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy","hATTR-PN",[277,278,279,280,169],"Polyneuropathy","Amyloidosis","Transthyretin","TTR",{"date":150,"type":41},{"date":233,"type":41},{"date":284,"type":20},"2031-06-12",{"name":47,"class":48},29,{"id":288,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":289,"targetDuration":4,"studyType":21,"phases":290,"briefSummary":24,"conditions":291,"keywords":292,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":293,"startDateStruct":294,"completionDateStruct":295,"leadSponsor":296,"locationsCount":297},"100606865",{"count":19,"type":20},[23],[26,27,28],[30,31,32,33,34,35,36],{"date":150,"type":41},{"date":43,"type":41},{"date":45,"type":20},{"name":47,"class":48},856,{"id":299,"slug":4,"hasResults":10,"nctId":300,"briefTitle":301,"officialTitle":302,"acronym":4,"eligibilityCriteria":303,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":268,"enrollmentInfo":304,"targetDuration":4,"studyType":21,"phases":306,"briefSummary":307,"conditions":308,"keywords":310,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":323,"startDateStruct":324,"completionDateStruct":326,"leadSponsor":328,"locationsCount":329},"100597009","NCT07052903","TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy","TRITON-CM: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)","Inclusion Criteria\n\n* Has documented diagnosis of ATTR amyloidosis with cardiomyopathy including those with hereditary ATTR (hATTR) or wild-type ATTR (wATTR) amyloidosis.\n* Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF or signs and symptoms that require treatment with a diuretic.\n* Has screening N-terminal prohormone B-type natriuretic peptide (NT-proBNP) \\>300 ng\u002FL and \\\u003C8500 ng\u002FL; In patients with permanent or persistent atrial fibrillation, screening NT-proBNP \\>600 ng\u002FL and \\\u003C8500 ng\u002FL.\n* Patients may be receiving approved TTR stabilizers for ATTR amyloidosis (eg, tafamidis, acoramidis) and may be receiving background therapy for HF at the discretion of the Investigator.\n\nExclusion Criteria\n\n* Has New York Heart Association (NYHA) Class IV HF; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3.\n* Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV.\n* Has an estimated glomerular filtration rate eGFR of \\\u003C30 mL\u002Fmin\u002F1.73m\\^2 at screening.\n* Has received prior or currently receiving TTR-lowering therapy",{"count":305,"type":20},1250,[23],"The purpose of this study is to:\n\n* Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events\n* Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and\u002For death\n* Evaluate the efficacy of nucresiran compared to placebo on patient-reported health status and health-related quality of life",[309],"Transthyretin Amyloidosis With Cardiomyopathy",[311,312,313,314,315,316,317,318,278,280,279,319,36,169,320,321,322],"ATTR-CM","ATTR amyloidosis with cardiomyopathy","ATTR","Heriditary ATTR","hATTR","Wild-type ATTR","wATTR","Cardiomyopathy","TTR-mediated amyloidosis","TTR cardiomyopathy","V122i","TTR amyloidosis",{"date":150,"type":41},{"date":325,"type":41},"2025-07-02",{"date":327,"type":20},"2032-11-30",{"name":47,"class":48},233,{"id":331,"slug":4,"hasResults":10,"nctId":332,"briefTitle":333,"officialTitle":334,"acronym":4,"eligibilityCriteria":335,"healthyVolunteers":140,"sex":16,"minAge":17,"maxAge":162,"enrollmentInfo":336,"targetDuration":4,"studyType":21,"phases":338,"briefSummary":339,"conditions":340,"keywords":342,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":344,"startDateStruct":345,"completionDateStruct":347,"leadSponsor":349,"locationsCount":350},"100581045","NCT06845202","A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM","A Phase 1\u002F2, Randomized, Double-blind, Placebo-controlled 2-part Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-4324 in Overweight to Obese Adult Healthy Volunteers and Multiple Dose ALN-4324 in Overweight to Obese Patients With Type 2 Diabetes Mellitus (T2DM)","Inclusion Criteria:\n\nPart A:\n\n* Has a body mass index (BMI) of ≥27 kg\u002Fm\\^2 and \\\u003C40 kg\u002Fm\\^2\n\nPart B:\n\n* Is an adult patient with a confirmed diagnosis of T2DM\n* Has a hemoglobin A1c (HbA1c) ≥7% to \\\u003C10.5%\n* Has a BMI of ≥25 kg\u002Fm\\^2 and \\\u003C45 kg\u002Fm\\^2\n* Is on a stable dose of either metformin or metformin and a sodium-glucose cotransporter 2 inhibitor (SGLT2i)\n\nExclusion Criteria:\n\nPart A:\n\n* Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection\n\nPart B:\n\n* Receiving therapies for chronic weight management or antidiabetic medications other than metformin and SGLT2i\n\nNote: other protocol defined inclusion\u002Fexclusion criteria apply",{"count":337,"type":20},144,[60,118],"The purpose of this study is to:\n\n* evaluate the safety and tolerability of single ascending doses of ALN-4324 in healthy volunteers and to characterize the single-dose PK of ALN-4324\n* evaluate the efficacy, safety, tolerability, and pharmacodynamics (PD) of multiple doses of ALN-4324 in adult overweight to obese patients with T2DM",[341,210],"Obese or Overweight Healthy Volunteers",[31,343],"RNAi therepeutic",{"date":150,"type":41},{"date":346,"type":41},"2025-03-03",{"date":348,"type":20},"2027-08-31",{"name":47,"class":48},22,{"id":352,"slug":4,"hasResults":10,"nctId":353,"briefTitle":354,"officialTitle":355,"acronym":356,"eligibilityCriteria":357,"healthyVolunteers":140,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":358,"targetDuration":4,"studyType":21,"phases":360,"briefSummary":361,"conditions":362,"keywords":364,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":370,"startDateStruct":371,"completionDateStruct":373,"leadSponsor":375,"locationsCount":376},"100566781","NCT06659640","A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)","InsigHHT: A Phase 1\u002F2, Randomized, Double-blind, Placebo-controlled, 2-part Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-6400 in Adult Healthy Volunteers and Multiple Dose ALN-6400 in Adult Patients With Hereditary Hemorrhagic Telangiectasia (HHT)","InsigHHT","Inclusion Criteria -\n\nPart A:\n\n* Is a healthy adult volunteer\n\nPart B:\n\n* Is an adult patient with a clinical diagnosis of HHT\n\nExclusion Criteria -\n\nPart A:\n\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> upper limit of normal (ULN)\n* Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection\n* Has an estimated glomerular filtration (eGFR) of \\\u003C90 mL\u002Fmin\u002F1.73m\\^2 at screening\n\nPart B:\n\n* Has ALT or AST \\>2×ULN\n* Has total bilirubin \\>1.5×ULN\n* Has eGFR of \\\u003C30 mL\u002Fmin\u002F1.73m\\^2 at screening\n\nParts A and B:\n\n* Is not willing to comply with the contraceptive requirements during the study period\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply",{"count":359,"type":20},120,[60,118],"The purpose of this study is to:\n\n* evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers\n* evaluate the efficacy, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT",[363],"Hereditary Hemorrhagic Telangiectasia",[31,169,365,366,367,368,369],"Plasminogen","PLG","HHT","Osler-Weber-Rendu","epistaxis",{"date":150,"type":41},{"date":372,"type":41},"2024-11-07",{"date":374,"type":20},"2028-06-22",{"name":47,"class":48},16,{"id":378,"slug":4,"hasResults":10,"nctId":379,"briefTitle":380,"officialTitle":381,"acronym":4,"eligibilityCriteria":382,"healthyVolunteers":10,"sex":16,"minAge":383,"maxAge":384,"enrollmentInfo":385,"targetDuration":4,"studyType":21,"phases":387,"briefSummary":388,"conditions":389,"keywords":391,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":397,"startDateStruct":398,"completionDateStruct":400,"leadSponsor":402,"locationsCount":403},"100561076","NCT06585449","A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease","A Randomized, Double-blind, Placebo-controlled Study, to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered Single Ascending Doses of ALN-HTT02 in Adult Patients With Huntington's Disease","Inclusion Criteria\n\n* Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)\n\nExclusion Criteria\n\n* Has significant structural or degenerative neurologic disease other than Huntington's Disease (HD) at screening\n* Has primary or secondary immune compromise at screening due to infections, medical conditions, or chronic therapies\n* Has alanine aminotransferase or aspartate aminotransferase \\>2× upper limit of normal (ULN)\n* Has an estimated glomerular filtration rate (eGFR) of \\\u003C60 mL\u002Fmin\u002F1.73m\\^2 at screening\n* Has received an investigational agent within the last 1 year or 5 half-lives (if known)\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","25 Years","70 Years",{"count":386,"type":20},66,[60],"The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.",[390],"Huntington's Disease",[31,392,393,394,395,396],"ALN-HTT02","Intrathecal","HD-ISS Stage 2","HD-ISS early Stage 3","Neurodegenerative disorder",{"date":150,"type":41},{"date":399,"type":41},"2024-10-14",{"date":401,"type":20},"2028-07-05",{"name":47,"class":48},19,{"id":405,"slug":4,"hasResults":10,"nctId":406,"briefTitle":407,"officialTitle":408,"acronym":409,"eligibilityCriteria":410,"healthyVolunteers":10,"sex":16,"minAge":411,"maxAge":4,"enrollmentInfo":412,"targetDuration":4,"studyType":21,"phases":414,"briefSummary":415,"conditions":416,"keywords":418,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":420,"startDateStruct":421,"completionDateStruct":423,"leadSponsor":425,"locationsCount":426},"100546344","NCT06393712","A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy","A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)","cAPPricorn-1","Inclusion Criteria (sporadic CAA patients):\n\n* Is 50 years or older\n* Has probable CAA per the Boston Criteria Version 2.0\n\nInclusion Criteria (Dutch-type CAA patients):\n\n* Is 30 years or older\n* Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA\n\nExclusion Criteria:\n\n* Moderate or severe stage Alzheimer's disease (AD) or significant cognitive impairment (CI)\n* Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less than 90 days prior to anticipated randomization in the study\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>3×upper limit of normal (ULN) at Screening\n* Has estimated glomerular filtration rate (eGFR) \\\u003C30 mL\u002Fmin\u002F1.73m\\^2 at Screening\n* Has recently received an investigational agent\n* Has had treatment with amyloid-targeting antibody\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","30 Years",{"count":413,"type":20},200,[118],"The purpose of the study is to evaluate the effect of ALN-APP on measures of CAA disease progression and to characterize the safety, tolerability, and pharmacodynamics (PD) of ALN-APP in adult patients with sporadic CAA (sCAA) and Dutch-type CAA (D-CAA). The study will be conducted over 2 periods: a 24-month double-blind treatment period and an optional 18-month open-label extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 50 months.",[417],"Cerebral Amyloid Angiopathy",[419],"CAA",{"date":150,"type":41},{"date":422,"type":41},"2024-05-17",{"date":424,"type":20},"2029-12-14",{"name":47,"class":48},57,{"id":428,"slug":4,"hasResults":10,"nctId":429,"briefTitle":430,"officialTitle":431,"acronym":4,"eligibilityCriteria":432,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":433,"targetDuration":4,"studyType":21,"phases":435,"briefSummary":436,"conditions":437,"keywords":439,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":441,"startDateStruct":442,"completionDateStruct":444,"leadSponsor":446,"locationsCount":447},"100457068","NCT05231785","A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD","A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)","Inclusion Criteria:\n\n* Has mild cognitive impairment or mild dementia due to EOAD\n* Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) \\>20\n\nExclusion Criteria:\n\n* Has Non-Alzheimer's disease dementia\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2×upper limit of normal (ULN)\n* Has estimated glomerular filtration rate (eGFR) \\\u003C45 mL\u002Fmin\u002F1.73m\\^2 at Screening\n* Has recently received an investigational agent\n* Has recent treatment with amyloid-targeting antibody\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply",{"count":434,"type":20},60,[60],"The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.",[438],"Early-Onset Alzheimer Disease",[440],"EOAD",{"date":150,"type":41},{"date":443,"type":41},"2022-02-04",{"date":445,"type":20},"2029-04-20",{"name":47,"class":48},8,{"id":449,"slug":4,"hasResults":10,"nctId":450,"briefTitle":451,"officialTitle":452,"acronym":4,"eligibilityCriteria":453,"healthyVolunteers":10,"sex":16,"minAge":243,"maxAge":454,"enrollmentInfo":455,"targetDuration":4,"studyType":21,"phases":457,"briefSummary":458,"conditions":459,"keywords":461,"overallStatus":125,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":463,"startDateStruct":465,"completionDateStruct":467,"leadSponsor":469,"locationsCount":470},"100643681","NCT07636811","A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)","A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study, to Evaluate Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)","Inclusion Criteria:\n\n* Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21\n* Positive amyloid PET scan\n* Cognitively stable in the opinion of the investigator\n* Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening\n\nExclusion Criteria:\n\n* Has severe intellectual disability (ID)\n* Has a history of DS regression disorder\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2×upper limit of normal (ULN) at Screening\n* Has estimated glomerular filtration rate (eGFR) \\\u003C45 mL\u002Fmin\u002F1.73m\\^2 at Screening\n* Has recently received an investigational agent\n* Has had treatment with amyloid-targeting antibody\n* Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","55 Years",{"count":456,"type":20},58,[118],"The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.",[460],"Down Syndrome-Associated Alzheimer's Disease (DS-AD)",[462],"Alzheimer's; DS-AD; Down Syndrome; ALZ",{"date":464,"type":41},"2026-06-09",{"date":466,"type":20},"2026-07-01",{"date":468,"type":20},"2031-07-23",{"name":47,"class":48},2,{"id":472,"slug":4,"hasResults":10,"nctId":473,"briefTitle":474,"officialTitle":475,"acronym":4,"eligibilityCriteria":476,"healthyVolunteers":10,"sex":112,"minAge":4,"maxAge":4,"enrollmentInfo":477,"targetDuration":4,"studyType":479,"phases":4,"briefSummary":480,"conditions":481,"keywords":483,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":488,"lastUpdatePostDateStruct":489,"startDateStruct":491,"completionDateStruct":493,"leadSponsor":495,"locationsCount":496},"100442360","NCT05040373","Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program","Patisiran-LNP Pregnancy Surveillance Program","Inclusion Criteria:\n\n* Documentation that the patient was exposed to patisiran-LNP at any point starting from 12 weeks before LMP or at any point during pregnancy\n\nExclusion Criteria:\n\n* There are no exclusion criteria for participation in this program.",{"count":478,"type":20},10,"OBSERVATIONAL","The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal\u002Fneonatal\u002Finfant outcomes in women exposed to patisiran-LNP.",[482,277],"Hereditary Transthyretin-mediated (hATTR) Amyloidosis",[484,485,278,486,487],"Hereditary neuropathic amyloidosis","Familial amyloidosis","Amyloidosis senile","Cardiac amyloidosis","2026-05-28",{"date":490,"type":41},"2026-06-01",{"date":492,"type":41},"2020-08-01",{"date":494,"type":20},"2030-10-12",{"name":47,"class":48},7,{"id":498,"slug":4,"hasResults":10,"nctId":499,"briefTitle":500,"officialTitle":501,"acronym":502,"eligibilityCriteria":503,"healthyVolunteers":10,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":504,"targetDuration":4,"studyType":479,"phases":4,"briefSummary":506,"conditions":507,"keywords":510,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":523,"lastUpdatePostDateStruct":524,"startDateStruct":526,"completionDateStruct":528,"leadSponsor":530,"locationsCount":206},"100405602","NCT04561518","ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)","ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)","ConTTRibute","Inclusion Criteria:\n\n* Diagnosis of ATTR amyloidosis or documented known disease-causing TTR variant for the cohort of pre-symptomatic carriers\n* Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis\n\nExclusion Criteria:\n\n* Current enrollment in a clinical trial for any investigational agent",{"count":505,"type":20},1500,"The purpose of this study is to:\n\n* Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients\n* Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting\n* Describe disease emergence\u002Fprogression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant",[508,509],"Transthyretin-Mediated Amyloidosis","ATTR Amyloidosis",[169,279,280,278,482,511,512,513,514,515,516,277,517,313,518,319,519,520,521,522],"hATTR amyloidosis","Hereditary ATTR amyloidosis","Wild-type amyloidosis","wtATTR amyloidosis","ATTRv amyloidosis","ATTRwt amyloidosis","Familial amyloid polyneuropathies","Transthyretin amyloidosis","Polyneuropathies","Amyloid neuropathies","Amyloid neuropathies, familial","Amyloidosis, familial","2026-05-27",{"date":525,"type":41},"2026-05-29",{"date":527,"type":41},"2020-11-23",{"date":529,"type":20},"2033-12-31",{"name":47,"class":48},{"id":532,"slug":4,"hasResults":10,"nctId":533,"briefTitle":534,"officialTitle":535,"acronym":536,"eligibilityCriteria":537,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":538,"targetDuration":4,"studyType":479,"phases":4,"briefSummary":540,"conditions":541,"keywords":542,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":543,"lastUpdatePostDateStruct":544,"startDateStruct":546,"completionDateStruct":548,"leadSponsor":550,"locationsCount":551},"100620473","NCT07358078","DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting","DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy in a Real-World Setting","DemonsTTRate","Inclusion Criteria:\n\n* Has a diagnosis of ATTR-CM\n* Is initiating treatment with vutrisiran or another approved ATTR-CM therapy\n\nExclusion Criteria:\n\n* Is enrolled in an interventional clinical trial",{"count":539,"type":20},2000,"The purpose of this study is to:\n\n* Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care\n* Describe treatment patterns of adult patients with ATTR-CM treated with vutrisiran in routine clinical care\n* Assess health-related quality of life (HRQOL) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care\n* Assess healthcare resource use (HCRU) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care Compare the long-term effectiveness of vutrisiran versus other approved ATTR-CM treatments in routine clinical care",[309],[311,279,278,313,318],"2026-04-23",{"date":545,"type":41},"2026-04-24",{"date":547,"type":41},"2026-01-09",{"date":549,"type":20},"2030-10-23",{"name":47,"class":48},4,{"id":553,"slug":4,"hasResults":10,"nctId":554,"briefTitle":555,"officialTitle":556,"acronym":557,"eligibilityCriteria":558,"healthyVolunteers":10,"sex":16,"minAge":559,"maxAge":4,"enrollmentInfo":560,"targetDuration":4,"studyType":479,"phases":4,"briefSummary":562,"conditions":563,"keywords":565,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":543,"lastUpdatePostDateStruct":569,"startDateStruct":570,"completionDateStruct":572,"leadSponsor":574,"locationsCount":575},"100430348","NCT04883905","ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)","ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)","ELEVATE","Inclusion Criteria:\n\n* Documented diagnosis of AHP, per physician's determination\n\nExclusion Criteria:\n\n* Currently enrolled in a clinical trial for any investigational agent","12 Years",{"count":561,"type":20},150,"This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.",[564],"Acute Hepatic Porphyria",[566,567,568],"Givosiran","AHP","Acute hepatic porphyria",{"date":176,"type":41},{"date":571,"type":41},"2021-04-26",{"date":573,"type":20},"2027-04-01",{"name":47,"class":48},30,""]