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Clinical trials
catalyst calendar across sponsors →Studies where ALNY is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
69 interventional · 11 observational · 4 expanded access · 24 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)NCT04561518observationalIncidence of Adverse Events | — | Recruiting | — | Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis | 1,500 | 2033-12-31 | in 2,690 d | 2026-05-29 |
| DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World SettingNCT07358078observationalNew York Heart Association (NYHA) Class | — | Recruiting | — | Transthyretin Amyloidosis With Cardiomyopathy | 2,000 | 2030-10-23 | in 1,525 d | 2026-04-24 |
| Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance ProgramNCT05040373observationalPrevalence of Major Congenital Malformations | — | Recruiting | — | Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy | 10 | 2030-10-12 | in 1,514 d | 2026-06-01 |
| Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular DiseaseNCT07181109Time to First Occurrence of a Composite Endpoint of Cardiovascular (CV) Death, Nonfatal Myocardial Infarction (MI), Nonfatal Stroke, or Heart Failure (HF) Event (Hospitalization for HF or Urgent HF Visit)Randomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: Zilebesiran, Placebo | High Risk Cardiovascular Disease, Hypertension, High Cardiovascular Risk | 11,000 | 2030-09-30 | in 1,502 d | 2026-08-19 |
| TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNCT07052903Composite outcome of all-cause mortality and recurrent cardiovascular [CV] events (CV hospitalizations and urgent heart failure [HF] visits)Randomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: Nucresiran, Sterile Normal Saline (0.9% NaCl) | Transthyretin Amyloidosis With Cardiomyopathy | 1,750 | 2030-05-28 | in 1,377 d | 2026-08-14 |
| A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)NCT07636811Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs)Randomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: Mivelsiran, Placebo | Down Syndrome-Associated Alzheimer's Disease (DS-AD) | 58 | 2030-05-18 | in 1,367 d | 2026-08-12 |
| A Study to Evaluate ALN-5288 in Patients With Alzheimer's DiseaseNCT07214727Frequency of Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ALN-5288, Placebo | Alzheimer's Disease | 50 | 2030-03-06 | in 1,294 d | 2026-08-14 |
| A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOADNCT05231785Part A: Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ALN-APP, Placebo | Early-Onset Alzheimer Disease | 60 | 2029-04-20 | in 974 d | 2026-08-12 |
| BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)NCT04982393observationalIncidence of Adverse Events in Lumasiran Treated Patients | — | Active, not recruiting | — | Primary Hyperoxaluria Type 1 | 207 | 2028-09-01 | in 743 d | 2026-04-24 |
| A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's DiseaseNCT06585449Frequency of Adverse Events (AEs) in the Double-blind Part of the StudyRandomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ALN-HTT02, Placebo | Huntington's Disease | 66 | 2028-07-05 | in 685 d | 2026-08-12 |
| A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With ZilebesiranNCT07553442Part A: Percentage recovery from baseline in serum AGTRandomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: ALN-AGT01 RVR, Placebo, Zilebesiran | Hypertension | 93 | 2028-04-01 | in 590 d | 2026-08-14 |
| A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNCT06679946Frequency of Adverse Events (AEs)Open-label · Treatment | Phase 3 | Enrolling by invitation | Drug: Vutrisiran | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 700 | 2028-03-31 | in 589 d | 2026-02-20 |
| A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)NCT06659640Part A: Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 1/2 | Active, not recruiting | Drug: ALN-6400, Placebo | Hereditary Hemorrhagic Telangiectasia | 89 | 2028-01-05 | in 503 d | 2026-08-12 |
| TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With PolyneuropathyNCT07223203Change from Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9Randomized · Open-label · Treatment | Phase 3 | Recruiting | Drug: Nucresiran, Vutrisiran | Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN | 125 | 2027-12-27 | in 494 d | 2026-08-14 |
| Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With PolyneuropathyNCT06360289observationalNfL Levels in Asymptomatic Carriers of a TTR Variant and Symptomatic hATTR Patients With Polyneuropathy at Baseline | — | Active, not recruiting | Other: Standard of Care | Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State | 346 | 2027-12-15 | in 482 d | 2026-08-12 |
| A Study to Evaluate ALN-6222 in Participants With ObesityNCT07624071Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ALN-6222, Placebo | Obesity | 88 | 2027-12-14 | in 481 d | 2026-08-12 |
| A Study to Evaluate ALN-BCAT in Patients With Hepatocellular CarcinomaNCT06600321Frequency of Adverse Events (AEs)Non-randomized · Open-label · Treatment | Phase 1 | Recruiting | Drug: ALN-BCAT, Pembrolizumab | Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma | 158 | 2027-09-30 | in 406 d | 2026-06-17 |
| A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DMNCT06845202Part A: Frequency of Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 1/2 | Active, not recruiting | Drug: ALN-4324, Placebo | Obese or Overweight Healthy Volunteers, Type 2 Diabetes Mellitus (T2DM) | 105 | 2027-08-31 | in 376 d | 2026-08-14 |
| A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid AngiopathyNCT06393712Double-blind Treatment Period: Annualized Rate of New Lobar Cerebral Microbleeds (CMBs) Assessed on Magnetic Resonance Imaging (MRI) of Brain in Patients with Sporadic Cerebral Amyloid Angiopathy (sCAA)Randomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: Placebo, ALN-APP | Cerebral Amyloid Angiopathy | 200 | 2027-08-09 | in 354 d | 2026-07-15 |
| A Study to Evaluate ALN-4915 in Adult Healthy VolunteersNCT07535606Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ALN-4915, Placebo | Healthy Volunteers | 44 | 2027-07-31 | in 345 d | 2026-08-12 |
| HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMBNCT07575308Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 2 | Recruiting | Drug: ALN-6400 | Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB) | 24 | 2027-06-30 | in 314 d | 2026-08-12 |
| A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes MellitusNCT07465224Change from Baseline in M-valueRandomized · Triple-masked · Treatment | Phase 2 | Recruiting | Drug: ALN-4324, Placebo | Type 2 Diabetes Mellitus (T2DM) | 24 | 2027-05-28 | in 281 d | 2026-08-12 |
| A Study to Evaluate ALN-2232 in Participants With ObesityNCT07463846Part A: Frequency of Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 1/2 | Recruiting | Drug: ALN-2232, Placebo, Tirzepatide | Obesity | 156 | 2027-04-27 | in 250 d | 2026-08-12 |
| ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)NCT04883905observationalIncidence of Selected Events of Interest in AHP Patients | — | Recruiting | — | Acute Hepatic Porphyria | 150 | 2027-04-01 | in 224 d | 2026-04-27 |
| A Study to Evaluate ALN-4285 in Adult Healthy VolunteersNCT07295717Frequency of Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ALN-4285, Placebo | Healthy Volunteers | 76 | 2026-12-29 | in 131 d | 2026-08-12 |
| HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNCT04153149results2025-10-21Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall PopulationRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Active, not recruiting | Drug: Vutrisiran, Sterile Normal Saline (0.9% NaCl) | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 655 | 2024-05-08actual | — | 2026-01-12 |
| A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy SubjectsNCT05661916Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: ALN-TTRSC04, Placebo | Transthyretin-Mediated Amyloidosis | 96 | 2025-09-30actual | — | 2026-02-17 |
| A Study to Evaluate ALN-AGT01 RVR in Adult Healthy VolunteersNCT06675565Frequency of Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: ALN-AGT01 RVR, Placebo | Healthy Volunteers | 42 | 2025-09-08actual | — | 2025-09-19 |
| A Study to Evaluate Zilebesiran in Japanese Patients With Mild to Moderate HypertensionNCT06423352results2026-04-30Number of Participants With Adverse Events (AEs)Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: Zilebesiran, Placebo | Mild to Moderate Hypertension | 36 | 2025-07-17actual | — | 2026-04-30 |
| Zilebesiran as Add-on Therapy in Patients With High Cardiovascular Risk and Hypertension Not Adequately Controlled by Standard of Care Antihypertensive Medications (KARDIA-3)NCT06272487Change from Baseline at Month 3 in Mean Seated Office Systolic Blood Pressure (SBP)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Zilebesiran, Placebo | High Cardiovascular Risk, Hypertension | 375 | 2025-06-19actual | — | 2026-03-02 |
| A Phase 1/2 Study to Evaluate ALN-KHK in Overweight to Obese Healthy Volunteers and Obese Patients With T2DMNCT05761301Part A: Frequency of Adverse EventsRandomized · Triple-masked · Treatment | Phase 1/2 | Terminatedsponsor's stated reason: Business reasons (not safety related) | Drug: ALN-KHK, Placebo | Type 2 Diabetes Mellitus (T2DM) | 49 | 2025-04-03actual | — | 2025-05-07 |
| Zilebesiran as Add-on Therapy in Patients With Hypertension Not Adequately Controlled by a Standard of Care Antihypertensive Medication (KARDIA-2)NCT05103332results2025-07-09Indapamide: Change From Baseline at Month 3 in 24-hour Mean SBP Assessed by ABPM - Censored DataRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Indapamide, Amlodipine, Olmesartan, Placebo, Zilebesiran | Hypertension | 663 | 2023-12-11actual | — | 2025-11-03 |
| A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate HypertensionNCT04936035results2024-12-27Change From Baseline at Month 3 in 24-hour Mean SBP Assessed by ABPMRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Placebo, ALN-AGT01 | Hypertension | 394 | 2023-04-04actual | — | 2025-12-23 |
| An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1NCT03350451results2024-04-25Number of Participants With at Least One Adverse Event (AE)Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Lumasiran | PH1, Primary Hyperoxaluria, RNAi Therapeutic, siRNA, AGT | 20 | 2023-02-07actual | — | 2024-04-25 |
| A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With GoutNCT05256810Part A: Frequency of Adverse EventsRandomized · Quadruple-masked · Treatment | Phase 1/2 | Terminatedsponsor's stated reason: The Sponsor elected not to continue with the study | Drug: ALN-XDH, Placebo | Gout | 44 | 2023-01-25actual | — | 2024-03-29 |
| A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)NCT04565717Parts A and B: Frequency of Adverse EventsRandomized · Quadruple-masked · Treatment | Phase 1 | Terminatedsponsor's stated reason: Study ALN-HSD-001 was terminated due to business reasons and changes in the development strategy of the product. No safety issues were found. | Drug: ALN-HSD, Placebo | Nonalcoholic Steatohepatitis, NASH | 6 | 2023-01-06actual | — | 2024-05-17 |
| The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)NCT02510261results2023-12-06Percentage of Participants With Adverse Events (AEs) Leading to Study DiscontinuationNon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Patisiran | Amyloidosis | 211 | 2022-11-23actual | — | 2023-12-06 |
| A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate LevelsNCT05161936results2024-05-22Percent Change in 24-hour Urinary Oxalate From Baseline to Month 6Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Study terminated early due to low enrollment | Drug: Lumasiran, Placebo | Recurrent Calcium Oxalate Kidney Stone Disease, Elevated Urinary Oxalate Levels | 2 | 2022-11-01actual | — | 2024-05-22 |
| APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)NCT03997383results2023-10-18Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Placebo, Patisiran | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 360 | 2022-06-20actual | — | 2026-04-20 |
| A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A MutationNCT04201418observationalPercentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline | — | Completed | Drug: Patisiran | Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy | 67 | 2022-05-24actual | — | 2022-06-06 |
| A Study to Evaluate ALN-AGT01 in Patients With HypertensionNCT03934307Number of Participants with Adverse Events (AEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: ALN-AGT01, ALN-AGT01-Matching Placebo, Irbesartan, Irbesartan-Matching Placebo | Hypertension | 124 | 2022-04-20actual | — | 2023-01-17 |
| A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN)NCT03841448results2023-12-08Percent Change From Baseline in UPCR as Measured in 24-hour Urine at Week 32Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Study was terminated early having identified clinically meaningful magnitude of proteinuria reduction due to cemdisiran (study goal). Participants completed DBT period\&were in OLE period. Sponsor had no concerns with safety\&integrity of participants. | Drug: Placebo, Cemdisiran | IgA Nephropathy (IgAN), Berger Disease, Glomerulonephritis, IgA | 31 | 2022-03-17actual | — | 2024-08-09 |
| A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)NCT02949830results2024-03-12Percentage of Participants With Adverse Events (AEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: Givosiran | Acute Intermittent Porphyria | 16 | 2021-11-05actual | — | 2024-03-12 |
| A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1NCT04152200results2022-07-12Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Lumasiran | Primary Hyperoxaluria Type 1, Primary Hyperoxaluria | 21 | 2021-05-18actual | — | 2025-07-18 |
| EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)NCT02240784observationalMedical history of AHP patients (Part A only) | — | Completed | — | Acute Hepatic Porphyria | 136 | 2021-04-26actual | — | 2021-05-17 |
| HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)NCT03759379results2022-08-11Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]Randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Patisiran, Vutrisiran | Amyloidosis, Hereditary, Transthyretin Amyloidosis | 164 | 2020-11-10actual | — | 2026-01-12 |
| Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver TransplantNCT03862807results2021-12-21Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Patisiran | Amyloidosis, Familial, Transthyretin Amyloidosis | 24 | 2020-10-06actual | — | 2024-04-22 |
| A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1NCT03905694results2021-07-19Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Lumasiran | Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1) | 18 | 2020-06-29actual | — | 2025-02-14 |
| A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseNCT03767829Percentage of Participants with Treatment Emergent Adverse Events (TEAEs)Randomized · Double-masked · Treatment | Phase 1/2 | Terminatedsponsor's stated reason: The sponsor decided not to proceed with Part B for business reasons. | Drug: ALN-AAT02, Placebo, ALN-AAT02 | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | 32 | 2020-06-25actual | — | 2021-04-27 |
| A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) DiseaseNCT03774784observationalChange in Estimated Glomerular Filtration Rate (eGFR) from Baseline Up to End of Study (Month 48) | — | Terminatedsponsor's stated reason: The study was terminated due to the impacts of COVID-19 on patient safety and enrollment. | — | Amyloidosis, Leukocyte Chemotactic Factor 2 Amyloidosis | 26 | 2020-05-18actual | — | 2020-06-16 |
| A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1NCT03681184results2021-01-19Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Placebo, Lumasiran | Primary Hyperoxaluria Type 1 (PH1) | 39 | 2019-11-05actual | — | 2024-08-12 |
| ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)NCT03338816results2020-02-11Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Givosiran, Placebo | Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria, Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP) | 94 | 2019-01-31actual | — | 2024-04-22 |
| Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1NCT02706886results2020-01-30Number of Participants With Adverse Events (AEs)Randomized · Single-masked · TreatmentStatistical Analysis Plan ↗ · Study Protocol ↗ | Phase 1/2 | Completed | Drug: Lumasiran, Placebo | Primary Hyperoxaluria Type 1 (PH1) | 52 | 2019-01-23actual | — | 2020-01-30 |
| INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHPNCT03547297observationalProportion of participants with current or past clinically symptomatic AHP with urinary aminolevulinic acid (ALA) or porphobilinogen (PBG) values above the upper limit of normal (ULN) | — | Terminatedsponsor's stated reason: Sponsor elected not to continue with study | — | Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP), Porphyria Acute | 212 | 2019-01-15actual | — | 2019-01-25 |
| A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)NCT03505853Profile of Pharmacokinetics (PK) of Cytochrome P450 (CYP) probe cocktailOpen-label · Treatment | Phase 1 | Completed | Drug: Givosiran, 5-probe cocktail | Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP), Porphyria, Acute Intermittent, Acute Porphyria | 10 | 2018-11-16actual | — | 2019-03-12 |
| A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic SyndromeNCT03303313The effect of Cemdisiran on platelet countOpen-label · Treatment | Phase 2 | Withdrawnsponsor's stated reason: Study terminated early due to lack of enrollment. | Drug: Cemdisiran | Atypical Hemolytic Uremic Syndrome | 0 | 2018-09-12actual | — | 2018-10-01 |
| A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy SubjectsNCT02797847Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)Randomized · Single-masked · Treatment | Phase 1 | Completed | Drug: ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl) | Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) | 80 | 2018-01-12actual | — | 2018-09-21 |
| A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseNCT02503683The safety of ALN-AAT evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuationRandomized · Single-masked · Treatment | Phase 1 | Terminatedsponsor's stated reason: Observation of low incidence of asymptomatic, transiently elevated liver enzymes in a subset of study subjects | Drug: ALN-AAT, Sterile Normal Saline (0.9% NaCl) | Antitrypsin Deficiency Liver Disease | 26 | 2018-01-03actual | — | 2019-01-07 |
| A Study of ALN-HBV in Healthy Adult Volunteers and Non-cirrhotic Patients With Chronic Hepatitis B Virus (HBV) InfectionNCT02826018Proportion of subjects experiencing adverse eventsRandomized · Single-masked · Treatment | Phase 1 | Terminated | Drug: ALN-HBV, Sterile Normal Saline (0.9% NaCl) | Hepatitis B, Chronic Hepatitis B, Hepatitis B, Chronic, Hepatitis B Infection, HBV | 24 | Oct 2017actual | — | 2018-09-24 |
| A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)NCT02452372The safety of givosiran evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuationRandomized · Single-masked · Treatment | Phase 1 | Completed | Drug: givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl) | Acute Intermittent Porphyria | 40 | 2017-09-06actual | — | 2018-06-14 |
| APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated AmyloidosisNCT01960348results2018-09-06Modified Neuropathy Impairment Score +7 (mNIS+7)Randomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) | TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related | 225 | Aug 2017actual | — | 2024-04-22 |
| ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)NCT02319005results2018-07-186 Minute Walk Distance (6-MWD)Randomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl) | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis | 206 | 2017-03-30actual | — | 2018-07-18 |
| A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac AmyloidosisNCT02292186results2018-06-15Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Revusiran (ALN-TTRSC) | TTR-mediated Amyloidosis | 25 | 2017-02-22actual | — | 2020-07-09 |
| The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver TransplantNCT02595983results2019-03-28Percentage Change From Baseline in Serum TTR at Month 6Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Revusiran | Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis, Familial Amyloid Neuropathies | 12 | 2017-02-06actual | — | 2019-03-28 |
| The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)NCT01961921results2018-10-30The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug DiscontinuationOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion | TTR-mediated Amyloidosis | 27 | Jul 2016actual | — | 2024-04-19 |
| A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHNCT02352493results2020-03-30Number of Participants With Adverse EventsRandomized · Double-masked · TreatmentStatistical Analysis Plan ↗ · Study Protocol: Clinical Study Protocol ↗ · Study Protocol: Amendment Summary of Changes ↗ | Phase 1/2 | Completed | Drug: ALN-CC5, Sterile Normal Saline (0.9% NaCl) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | 62 | Apr 2016actual | — | 2020-03-30 |
| DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac AmyloidosisNCT02252653observationalProportion of patients with of Transthyretin (TTR) mutations | — | Completed | — | Familial Amyloidotic Cardiomyopathy (FAC) | 1,010 | Jan 2016actual | — | 2016-07-20 |
| A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)NCT02314442The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuationRandomized · Single-masked · Treatment | Phase 1 | Completed | Drug: ALN-PCSSC, Sterile Normal Saline (0.9% NaCl) | Hypercholesterolemia | 70 | May 2015actual | — | 2015-12-17 |
| A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy VolunteersNCT01814839The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: ALN-TTRSC (revusiran), Sterile Normal Saline (0.9% NaCl) | TTR-mediated Amyloidosis | 85 | Apr 2015actual | — | 2016-02-05 |
| Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac AmyloidosisNCT01981837The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.Open-label · Treatment | Phase 2 | Completed | Drug: ALN-TTRSC (revusiran) for subcutaneous administration | TTR-mediated Amyloidosis | 26 | Oct 2014actual | — | 2023-03-20 |
| A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy VolunteersNCT02053454The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuationRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) | Transthyretin (TTR)-Mediated Amyloidosis | 12 | Mar 2014actual | — | 2015-05-25 |
| Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) AmyloidosisNCT01617967results2018-11-21Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug DiscontinuationOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Patisiran | TTR-mediated Amyloidosis | 29 | Oct 2013actual | — | 2024-04-19 |
| Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 TreatmentNCT01158079Collect long term ALN-VSP02 safety dataOpen-label · Treatment | Phase 1 | Completed | Drug: ALN-VSP02 | Solid Tumors | 7 | Aug 2012actual | — | 2012-10-12 |
| Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer SubjectsNCT01559077The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.Randomized · Single-masked · Treatment | Phase 1 | Completed | Drug: ALN-TTR02, Sterile Normal Saline (0.9% NaCl) | TTR-mediated Amyloidosis | 17 | Jun 2012actual | — | 2013-01-15 |
| Phase 2b Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)NCT01065935The occurrence of new or progressive BOS grade 0p through 3 in RSV-infected lung transplant recipientsRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: ALN-RSV01, Normal Saline | Respiratory Syncytial Virus Infections | 87 | Apr 2012actual | — | 2018-02-05 |
| Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)NCT01437059The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.Randomized · Single-masked · Treatment | Phase 1 | Completed | Drug: ALN-PCS02, Sterile Normal Saline (0.9% NaCl) | Elevated LDL-Cholesterol (LDL-C) | 32 | Mar 2012actual | — | 2012-10-12 |
| Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) AmyloidosisNCT01148953The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.Randomized · Single-masked · Treatment | Phase 1 | Completed | Drug: ALN-TTR01, Sterile Normal Saline (0.9% NaCl) | Transthyretin Mediated Amyloidosis (ATTR) | 32 | Jan 2012actual | — | 2012-05-24 |
| Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ALN-VSP02 In Patients With Advanced Solid Tumors With Liver InvolvementNCT00882180Safety and Tolerability of intravenous ALN-VSP02Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: ALN-VSP02 | Solid Tumors | 41 | Jul 2011actual | — | 2011-08-24 |
| Phase 2 Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)NCT00658086Assess safety and tolerability of aerosolized ALN-RSV01 versus placebo in lung transplant patients infected with RSVRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: ALN-RSV01, normal saline | Respiratory Syncytial Virus Infections | 24 | Apr 2009actual | — | 2018-01-29 |
| Intranasal ALN-RSV01 Administered to Adult Volunteers Experimentally Inoculated With Respiratory Syncytial VirusNCT00496821Safety and tolerability of intranasal ALN-RSV01 versus placebo, administered in a multiple-dose schedule (once daily for 5 days) to healthy adult volunteers experimentally inoculated with respiratory syncytial virusRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ALN-RSV01 | Respiratory Syncytial Virus Infections | 88 | — | — | 2007-11-30 |
| Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)NCT02939820expanded access | — | Approved for marketing | Drug: patisiran (ALN-TTR02) | TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related | — | — | — | 2024-05-20 |
| Expanded Access Protocol of Givosiran for Patients With Acute Hepatic PorphyriaNCT04056481expanded access | — | Approved for marketing | Drug: Givosiran | Acute Hepatic Porphyria | — | — | — | 2024-05-20 |
| Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1NCT04125472expanded access | — | Approved for marketing | Drug: Lumasiran | Primary Hyperoxaluria | — | — | — | 2024-02-16 |
| Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With CardiomyopathyNCT05505838expanded access | — | No longer available | Drug: Patisiran | Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy | — | — | — | 2024-02-16 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19