49 completed, 19 recruiting, 10 terminated, 7 active not recruiting. 36 Phase 1, 35 Phase 2, 15 Phase 3, 2 Early Phase 1, 1 Not applicable.
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD) Mivelsiran, Placebo - Down Syndrome-Associated Alzheimer's Disease (DS-AD) | Phase 2 | Recruiting | Alnylam Pharmaceuticals | 58 | 2026-08-17 | 2026-10-13 |
| HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB ALN-6400 - Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB) | Phase 2 | Recruiting | Alnylam Pharmaceuticals | 24 | 2026-07-30 | 2026-09-18 |
| A Study to Evaluate ALN-6222 in Participants With Obesity ALN-6222, Placebo - Obesity | Phase 1 | Recruiting | Alnylam Pharmaceuticals | 88 | 2026-06-04 | 2026-10-21 |
| A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran ALN-AGT01 RVR, Placebo, Zilebesiran - Hypertension | Phase 2 | Recruiting | Alnylam Pharmaceuticals | 93 | 2026-04-27 | 2026-10-08 |
| A Study to Evaluate ALN-2232 in Participants With Obesity ALN-2232, Placebo, Tirzepatide - Obesity | Phase 1 / Phase 2 | Recruiting | Alnylam Pharmaceuticals | 156 | 2026-03-02 | 2026-10-13 |
| A Study to Evaluate ALN-4915 in Adult Healthy Volunteers ALN-4915, Placebo - Healthy Volunteers | Phase 1 | Active not recruiting | Alnylam Pharmaceuticals | 36 | 2026-03-02 | 2026-10-15 |
| TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy Nucresiran, Vutrisiran - Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN | Phase 3 | Recruiting | Alnylam Pharmaceuticals | 125 | 2026-02-03 | 2026-10-08 |
| A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus ALN-4324, Placebo - Type 2 Diabetes Mellitus (T2DM) | Phase 2 | Recruiting | Alnylam Pharmaceuticals | 24 | 2026-01-15 | 2026-10-20 |
| DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting Transthyretin Amyloidosis With Cardiomyopathy | Recruiting | Alnylam Pharmaceuticals | 2000 | 2026-01-09 | 2026-10-09 | |
| A Study to Evaluate ALN-4285 in Adult Healthy Volunteers ALN-4285, Placebo - Healthy Volunteers | Phase 1 | Recruiting | Alnylam Pharmaceuticals | 76 | 2025-12-12 | 2026-10-20 |
| A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease ALN-5288, Placebo - Alzheimer's Disease | Phase 1 | Recruiting | Alnylam Pharmaceuticals | 50 | 2025-10-15 | 2026-10-08 |
| Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease Zilebesiran, Placebo - High Risk Cardiovascular Disease, Hypertension | Phase 3 | Recruiting | Alnylam Pharmaceuticals | 11000 | 2025-09-06 | 2026-10-15 |
| TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Nucresiran, Sterile Normal Saline (0.9% NaCl) - Transthyretin Amyloidosis With Cardiomyopathy | Phase 3 | Recruiting | Alnylam Pharmaceuticals | 1750 | 2025-07-02 | 2026-10-20 |
| A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM ALN-4324, Placebo - Obese or Overweight Healthy Volunteers, Type 2 Diabetes Mellitus (T2DM) | Phase 1 / Phase 2 | Active not recruiting | Alnylam Pharmaceuticals | 105 | 2025-03-03 | 2026-10-20 |
| A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma ALN-BCAT, Pembrolizumab - Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma | Phase 1 | Recruiting | Alnylam Pharmaceuticals | 158 | 2024-12-30 | 2026-06-17 |
| A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Vutrisiran - Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Phase 3 | Enrolling by invitation | Alnylam Pharmaceuticals | 700 | 2024-12-03 | 2026-10-21 |
| A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT) ALN-6400, Placebo - Hereditary Hemorrhagic Telangiectasia | Phase 1 / Phase 2 | Active not recruiting | Alnylam Pharmaceuticals | 89 | 2024-11-07 | 2026-10-15 |
| A Study to Evaluate ALN-AGT01 RVR in Adult Healthy Volunteers ALN-AGT01 RVR, Placebo - Healthy Volunteers | Phase 1 | Completed | Alnylam Pharmaceuticals | 42 | 2024-11-05 | 2025-09-19 |
| A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease ALN-HTT02, Placebo - Huntington's Disease | Phase 1 | Recruiting | Alnylam Pharmaceuticals | 66 | 2024-10-14 | 2026-10-15 |
| A Study to Evaluate Zilebesiran in Japanese Patients With Mild to Moderate Hypertension Zilebesiran, Placebo - Mild to Moderate Hypertension | Phase 1 / Phase 2 | Completed | Alnylam Pharmaceuticals | 36 | 2024-06-05 | 2026-04-30 |
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Amyloidosis, Hereditary, Transthyretin-Related (16) · Amyloidosis (15) · Amyloid Neuropathies, Familial (11) · Amyloidosis, Familial (11) · Cardiomyopathies (10) · Hypertension (7) · Amyloid Neuropathies (6) · Coproporphyria, Hereditary (6) · Hyperoxaluria, Primary (6) · Polyneuropathies (6) · Porphyria, Acute Intermittent (6) · Porphyrias (6) · Alzheimer Disease (5) · Primary hyperoxaluria type 1 (5) · Heart Failure (4)
patisiran (13) · lumasiran (8) · givosiran (5) · revusiran (5) · Saline Solution (2) · ALN-PCS (1) · Amlodipine (1) · Indapamide (1) · Irbesartan (1) · Sodium Chloride (1) · Standard of Care (1) · Tirzepatide (1) · olmesartan (1) · peginterferon alfa-2a (1) · pembrolizumab (1)
United Kingdom (54) · United States (54) · France (31) · Germany (30) · Spain (28) · Canada (27) · Sweden (23) · Netherlands (21) · Italy (20) · Australia (19) · Portugal (16) · Belgium (15) · South Korea (15) · Taiwan (13) · Brazil (12)
Jared Gollob, MD (10) · Akshay Vaishnaw, MD PhD (3) · Nader Najafian, MD (3) · Amy Simon, MD (2) · Martin Zamora, MD (2) · Alan Glanville, MB BS MD Syd, FRACP (1) · Allan Glanville, MB BS MD Syd, FRACP (1) · Edward Miller, MD (1) · Felix Knauf, MD (1) · Hanna K Gaggin, MD (1) · Jared Gollob (1) · Jens T Gottlieb, MD (1) · John Vest, MD (1)
PMID 41388232, PMID 23984729, PMID 37290591, PMID 38332115, PMID 38548524, PMID 39213194, PMID 39363243, PMID 20851929, PMID 24094767, PMID 26338094, PMID 27959715, PMID 28893208, PMID 29972753, PMID 30586695, PMID 30726693, PMID 30878017, PMID 31215818, PMID 31322739, PMID 32062791, PMID 32521132, PMID 32641071, PMID 33047216, PMID 33212063, PMID 33789010, PMID 33985991, PMID 34741731, PMID 34906487, PMID 35188911, PMID 35213769, PMID 35749284, PMID 35843439, PMID 35875890, PMID 35913563, PMID 36028858, PMID 36120830, PMID 37467498, PMID 37479139, PMID 37523143, PMID 37639169, PMID 37888916, PMID 38044204, PMID 38363577, PMID 38512694, PMID 39355649, PMID 39389081, PMID 39566871, PMID 39804640, PMID 40099776, PMID 40243852, PMID 40380962
2018 (7), 2025 (6), 2023 (5), 2019 (3), 2015 (2), 2016 (2), 2009 (1), 2014 (1)