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Alnylam Pharmaceuticals clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK, Ireland, China, India and EU (CTIS) registers with Alnylam Pharmaceuticals as lead sponsor. Synced 26 Sep 2026
25Current trials
17Recruiting now
7Drugs in these trials
6Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Alnylam Pharmaceuticals is the lead sponsor of 25 current clinical trials in our USA, UK, Ireland, China, India and EU (CTIS) registers. 17 are recruiting patients now. The most common phase is Phase 1. The drugs named most often in its trials are Aln-App, Vutrisiran and ALN-4324. The most-studied condition is Transthyretin Amyloidosis (ATTR) With Cardiomyopathy.

ClinicalTrials.gov trials by status

  • Recruiting1774%
  • Active, closed522%
  • By invitation14%

Trials by phase

  • Phase 1626%
  • Phase 3522%
  • Observational522%
  • Phase 2522%
  • Phase 1/229%
01

Clinical trials sponsored by Alnylam Pharmaceuticals

23 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
NCT07223203 · Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
Phase 3 Recruiting 2026-02 Ireland, UK, USA
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT07052903 · Transthyretin Amyloidosis With Cardiomyopathy
Phase 3 Recruiting 2025-07 Ireland, UK, USA, China
A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
NCT06585449 · Huntington's Disease
Phase 1 Recruiting 2024-10 UK
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
NCT05040373 · Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Observational Recruiting 2020-08 USA
Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease
NCT07181109 · ClinicalTrials.gov ↗ · High Risk Cardiovascular Disease, Hypertension, High Cardiovascular Risk
Phase 3 Recruiting 2025-09 UK, USA, India, China
A Study to Evaluate ALN-4285 in Adult Healthy Volunteers
NCT07295717 · ClinicalTrials.gov ↗ · Healthy Volunteers
Phase 1 Recruiting 2025-12 UK
A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease
NCT07214727 · ClinicalTrials.gov ↗ · Alzheimer's Disease
Phase 1 Recruiting 2025-10 UK
A Study to Evaluate ALN-4915 in Adult Healthy Volunteers
NCT07535606 · ClinicalTrials.gov ↗ · Healthy Volunteers
Phase 1 Recruiting 2026-03 UK
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
NCT05231785 · ClinicalTrials.gov ↗ · Early-Onset Alzheimer Disease
Drug: Aln-App
Phase 1 Recruiting 2022-02 UK, USA
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
NCT07575308 · ClinicalTrials.gov ↗ · Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
Drug: ALN-6400
Phase 2 Recruiting 2026-07 UK, USA
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
NCT07358078 · ClinicalTrials.gov ↗ · Transthyretin Amyloidosis With Cardiomyopathy
Observational Recruiting 2026-01 USA
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
NCT04883905 · ClinicalTrials.gov ↗ · Acute Hepatic Porphyria
Observational Recruiting 2021-04 UK, USA
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
NCT04561518 · ClinicalTrials.gov ↗ · Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
Observational Recruiting 2020-11 USA
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
NCT06600321 · ClinicalTrials.gov ↗ · Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma
Phase 1 Recruiting 2024-12 USA
A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus
NCT07465224 · ClinicalTrials.gov ↗ · Type 2 Diabetes Mellitus (T2DM)
Drug: ALN-4324
Phase 2 Recruiting 2026-01 USA
A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran
NCT07553442 · ClinicalTrials.gov ↗ · Hypertension
Phase 2 Recruiting 2026-04 USA
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
NCT07636811 · ClinicalTrials.gov ↗ · Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Drug: Aln-App
Phase 2 Recruiting 2026-08 USA
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT06679946 · ClinicalTrials.gov ↗ · Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Phase 3 By invitation 2024-12 UK, USA
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
NCT04982393 · Primary Hyperoxaluria Type 1
Observational Active, closed 2021-12 UK, USA
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT04153149 · Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Phase 3 Active, closed 2019-11 Ireland, UK, USA
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
NCT06393712 · ClinicalTrials.gov ↗ · Cerebral Amyloid Angiopathy
Drug: Aln-App
Phase 2 Active, closed 2024-05 UK, USA
A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM
NCT06845202 · Obese or Overweight Healthy Volunteers, Type 2 Diabetes Mellitus (T2DM)
Drug: ALN-4324
Phase 1/2 Active, closed 2025-03 USA
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
NCT06659640 · ClinicalTrials.gov ↗ · Hereditary Hemorrhagic Telangiectasia
Drug: ALN-6400
Phase 1/2 Active, closed 2024-11 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Alnylam Pharmaceuticals in the EU trials register (CTIS)

2 trials · 1 in Ireland

Authorised or ongoing trials in the EU Clinical Trials Information System with Alnylam Pharmaceuticals as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

StudyPhaseStatusCountries
A Phase 2, Multicenter, Randomized, Double-blind, Placebocontrolled 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)
2025-523390-42-00 · Nervous System Diseases
Phase 2 Authorised Ireland + 4 more
HMBeacon: A Phase 2, Randomized, Double-blind Study of the Safety, Tolerability, Efficacy, and Pharmacodynamics of Multiple Dose ALN-6400 in Female Patients with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)
2025-524967-19-00 · CTIS ↗ · Cardiovascular Diseases
Phase 2 Authorised Netherlands, Sweden, Germany
03

Drugs in Alnylam Pharmaceuticals trials

7 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

04

Conditions in Alnylam Pharmaceuticals trials

26 conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy2Transthyretin Amyloidosis With Cardiomyopathy2Healthy Volunteers2Hypertension2Type 2 Diabetes Mellitus (T2DM)2Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy1hATTR-PN1Primary Hyperoxaluria Type 11Huntington's Disease1Cerebral Amyloid Angiopathy1Alzheimer's Disease1High Risk Cardiovascular Disease1High Cardiovascular Risk1Early-Onset Alzheimer Disease1Von Willebrand Disease (VWD)1Heavy Menstrual Bleeding (HMB)1Acute Hepatic Porphyria1Hereditary Transthyretin-mediated (hATTR) Amyloidosis1Polyneuropathy1Obese or Overweight Healthy Volunteers1Transthyretin-Mediated Amyloidosis1ATTR Amyloidosis1Advanced Hepatocellular Carcinoma1Metastatic Hepatocellular Carcinoma1
05

By trial register

RegisterTrialsRecruiting
Clinical trials in USA1913
Clinical trials in UK1410
Clinical trials in Ireland32
Clinical trials in China22
Clinical trials in India11
EU register (CTIS)2—

A trial with sites in more than one country appears in each of those registers.

06

Questions about Alnylam Pharmaceuticals trials

How many clinical trials does Alnylam Pharmaceuticals sponsor?

Alnylam Pharmaceuticals is the lead sponsor of 23 current trials on ClinicalTrials.gov in our USA, UK, Ireland, China and India registers, and 2 authorised or ongoing trials in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Alnylam Pharmaceuticals trials recruiting?

Yes. 17 of its 23 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Alnylam Pharmaceuticals testing?

Its current trials name 7 drugs and biologicals as interventions. The most frequent are Aln-App (3), Vutrisiran (3), ALN-4324 (2), ALN-6400 (2) and Nucresiran (2). The number in brackets is how many of its trials name each drug.

Does Alnylam Pharmaceuticals have clinical trials in Ireland?

Yes. 3 trials in our Ireland register (ClinicalTrials.gov), and 1 trial in the EU register (CTIS) with a site in Ireland. Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.