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Clinical trials and research at Alnylam Pharmaceuticals

• 18Clinical trials
• 43Countries

Newest trials

All recruiting trials (18)

• Mivelsiran for early Down syndrome–associated Alzheimer's disease in adults

  A 2-phase study will test whether mivelsiran can help adults with early DS-AD and see how safe and how it behaves in the body.

  Recruiting at 10 sites Phase 2 Alnylam Pharmaceuticals · Maitland, Florida and 9 other locations · NCT07636811

• First-in-human test of ALN-6222 in adults with obesity

  A first-in-human, randomized, placebo-controlled study will test a subcutaneous injection of ALN-6222 in adults with obesity to see if it is safe, tolerated, and how the body processes and responds to it.

  Recruiting Phase 1 Alnylam Pharmaceuticals · Mount Royal · NCT07624071

• ALN-6400 for women with von Willebrand disease and heavy menstrual bleeding

  This study tests ALN-6400 to see if it can help reduce heavy menstrual bleeding in adult and adolescent women with von Willebrand disease.

  Recruiting Phase 2 Alnylam Pharmaceuticals · Orange, California and 20 other locations · NCT07575308

• A person-friendly look at ALN-AGT01 RVR in adults with mild to moderate high blood pressure who had prior zilebesiran

  This study tests whether ALN-AGT01 RVR can help adults with mild to moderate hypertension who previously used zilebesiran.

  Recruiting Phase 2 Alnylam Pharmaceuticals · Lake Forest, California and 2 other locations · NCT07553442

• ALN-4324 for insulin sensitivity in adults with type 2 diabetes

  This study will test whether a single dose of ALN-4324 improves whole-body insulin sensitivity in adults with type 2 diabetes who are on stable metformin therapy.

  Recruiting Phase 2 Alnylam Pharmaceuticals · Chula Vista, California · NCT07465224

• ALN-2232 treatment for people with obesity.

  This trial will test whether ALN-2232, alone or started with tirzepatide, is safe, well tolerated, and helps people with obesity (BMI 30–<40 and HbA1c <6.5%) lose weight while measuring how the drug behaves in the body.

  Recruiting Phase 1/2 Alnylam Pharmaceuticals · Mount Royal · NCT07463846

• Long-term global follow-up of adults with ATTR-CM treated with vutrisiran or other approved therapies

  This project will follow adults with ATTR-CM who start vutrisiran or another approved treatment to see how they do over up to five years in routine clinical care.

  Recruiting at 27 sites Alnylam Pharmaceuticals · Tucson, Arizona and 26 other locations · NCT07358078

• ALN-4285 single ascending doses in healthy adults

  This test will give single ascending doses of ALN-4285 or placebo to healthy adult volunteers to see if the drug is safe and how it behaves in the body.

  Recruiting Phase 1 Alnylam Pharmaceuticals · London · NCT07295717

• Nucresiran versus vutrisiran for hereditary transthyretin polyneuropathy

  This trial tests whether nucresiran helps people with hereditary transthyretin-mediated amyloidosis with polyneuropathy by improving nerve function, symptoms, and quality of life and whether it lowers blood TTR more than vutrisiran.

  Recruiting at 61 sites Phase 3 Alnylam Pharmaceuticals · Aurora, Colorado and 60 other locations · NCT07223203

• ALN-5288 injected into the spinal fluid for Alzheimer's disease

  This will test whether giving ALN-5288 into the spinal fluid is safe and how the medicine moves through and affects the body in people with mild cognitive impairment or dementia from Alzheimer's disease.

  Recruiting at 10 sites Phase 1 Alnylam Pharmaceuticals · Montreal and 12 other locations · NCT07214727

• Zilebesiran for uncontrolled high blood pressure in people with or at high risk for cardiovascular disease

  This will test whether zilebesiran versus placebo reduces the chance of heart attack, stroke, heart-failure events, or cardiovascular death in adults whose high blood pressure isn’t controlled and who have or are at high risk for cardiovascular disease.

  Recruiting at 1147 sites Phase 3 Alnylam Pharmaceuticals · Birmingham, Alabama and 1189 other locations · NCT07181109

• Nucresiran for transthyretin amyloidosis with cardiomyopathy

  This trial will test whether nucresiran can lower deaths and major heart-related events in people with transthyretin amyloidosis that affects the heart.

  Recruiting at 265 sites Phase 3 Alnylam Pharmaceuticals · La Jolla, California and 265 other locations · NCT07052903

• Evaluating ALN-BCAT for liver cancer treatment

  This study is testing a new treatment called ALN-BCAT, alone and with another drug, to see if it can safely help people with advanced liver cancer.

  Recruiting at 22 sites Phase 1 Alnylam Pharmaceuticals · Phoenix, Arizona and 22 other locations · NCT06600321

• Evaluating ALN-HTT02 for treating Huntington's disease in adults

  This study is testing a new drug called ALN-HTT02 to see if it is safe and effective for adults with early-stage Huntington's disease.

  Recruiting at 19 sites Phase 1 Alnylam Pharmaceuticals · Edmonton and 18 other locations · NCT06585449

• Evaluating ALN-APP for Early-Onset Alzheimer's Disease

  This study is testing a new treatment called ALN-APP to see if it is safe and can help people with early-onset Alzheimer's disease.

  Recruiting at 6 sites Phase 1 Alnylam Pharmaceuticals · La Jolla, California and 7 other locations · NCT05231785

• Monitoring pregnancy outcomes in women exposed to patisiran-LNP

  This study is looking to see how patisiran-LNP affects pregnancy and baby outcomes in women who have been treated with it.

  Recruiting at 7 sites Alnylam Pharmaceuticals · Iowa City, Iowa and 6 other locations · NCT05040373

• Registry for patients with Acute Hepatic Porphyria

  This study is creating a registry to track the experiences and treatment results of people with Acute Hepatic Porphyria to see how well givosiran and other approved therapies work in real life.

  Recruiting at 28 sites Alnylam Pharmaceuticals · Birmingham, Alabama and 29 other locations · NCT04883905

• Observational study of patients with ATTR amyloidosis

  This study looks at the characteristics of people with ATTR amyloidosis and tests how well existing treatments work for them in real life.

  Recruiting at 38 sites Alnylam Pharmaceuticals · La Jolla, California and 39 other locations · NCT04561518

Quick answers

How many clinical trials run by Alnylam Pharmaceuticals are recruiting right now?

18, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 43 countries. The most sites are in United States (11 trials), United Kingdom (9 trials), Germany (8 trials), Canada (8 trials), Spain (8 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 6, Phase 2: 4, Phase 3: 3, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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