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Clinical Trials

Our Clinical Trials

Alnylam is committed to developing new, innovative medicines to treat diseases with high unmet medical need. A clinical trial involves research using human volunteers (trial participants, including healthy volunteers or patients), and is intended to answer specific questions about potential new treatments for certain diseases. These studies allow us to evaluate whether these potential new treatments are safe and effective.

If you have questions about our clinical trials, please contact us:
Alnylam Clinical Trial Information Line
clinicaltrials@alnylam.com
866-330-0326 (Toll-free within the United States)
+1 617-575-7400 (International)

Visit www.clinicaltrials.gov to learn more about eligibility to participate in a study, risks and benefits of study participation, the informed consent process, questions to ask when considering whether to participate in a study, and much more information regarding clinical trials.

Clinical Trial Transparency

We are committed to ensuring our clinical trial information is accessible to patients, health care providers, researchers and the general public as a way to further scientific discovery and advance public health.

Learn more about Alnylam’s Clinical Trial Transparency ›

  • PATISIRAN (hATTR AMYLOIDOSIS)

    • APOLLO B – Phase 3 Study
      Trial Status

      Active, Not Recruiting

      Active, Not Recruiting

      Evaluating the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

      Location(s):

      USA, Czech Republic, Denmark, Sweden, Argentina, Brazil, South Korea, Italy, Taiwan, UK, France, Japan, Hong Kong, Australia, Mexico, Bulgaria, Chile, Belgium, Netherlands, New Zealand

      For more info:

      ClinicalTrials.gov – NCT03997383
      ATTRResearch.com

    • Phase 3 Study
      Trial Status

      Completed

      Completed

      Evaluating the efficacy and safety of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.

      Location(s):

      Italy, Sweden, Portugal, UK, Germany, Spain, France

      For more info:

      ClinicalTrials.gov – NCT03862807

    • Global Open-Label Extension (OLE) Study
      Trial Status

      Active

      Active, Not Recruiting

      Evaluating the long-term safety of patisiran in adults with hATTR amyloidosis with symptomatic polyneuropathy who participated in either the Phase 2 OLE study or the APOLLO study.

      Location(s):

      USA, Argentina, Australia, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Italy, Japan, South Korea, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, UK

      For more info:

      ClinicalTrials.gov – NCT02510261

    • APOLLO – Phase 3 Study
      Trial Status

      Active

      Completed

      Evaluated the safety and efficacy of patisiran in adults with hereditary ATTR (hATTR) amyloidosis with symptomatic polyneuropathy (previously known as familial amyloidotic polyneuropathy [FAP]).

      Location(s):

      USA, Argentina, Australia, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Italy, Japan, South Korea, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, UK

      For more info:

      ClinicalTrials.gov – NCT01960348

    • Phase 2 Open-Label Extension (OLE) Study
      Trial Status

      Completed

      Completed

      Evaluated the safety and efficacy of patisiran in adults with hATTR amyloidosis with symptomatic polyneuropathy who participated in the Phase 2 trial.

      Location(s):

      USA, Brazil, France, Germany, Portugal, Spain, Sweden

      For more info:

      ClinicalTrials.gov – NCT01961921

    • Phase 2 Study
      Trial Status

      Completed

      Completed

      Evaluated the safety and efficacy of patisiran in adults with hATTR amyloidosis with symptomatic polyneuropathy.

      Location(s):

      USA, Brazil, France, Germany, Portugal, Spain, Sweden, Portugal

      For more info:

      ClinicalTrials.gov – NCT01617967

    • ConTTRibute – Phase 4 Observational Study
      Trial Status

      Enrolling

      Enrolling

      Long-term study of patients with transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).

      Location(s):

      USA, Portugal, France

      For more info:

      ClinicalTrials.gov – NCT04561518

    • Phase 4 Observational Study
      Trial Status

      Enrolling

      Active Not Recruiting

      A multicenter observational study to evaluate the effectiveness of patisiran in patients with hATTR amyloidosis with polyneuropathy who have a V122I or T60A mutation.

      Location(s):

      USA

      For more info:

      ClinicalTrials.gov – NCT04201418

    • Patisiran-LNP Pregnancy Surveillance Program
      Trial Status

      Recruiting

      Recruiting

      The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

      Location(s):

      USA

      For more info:

      ClinicalTrials.gov – NCT05040373

  • GIVOSIRAN (ACUTE HEPATIC PORPHYRIA)

    • ENVISION – Phase 3 Study
      Trial Status

      Completed

      Completed

      Evaluating the effect of givosiran (ALN-AS1) on the rate of porphyria attacks in patients with acute hepatic porphyria (AHP).

      Location(s):

      USA, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, South Korea, Mexico, Netherlands, Poland, Spain, Sweden, Taiwan, UK

      For more info:

      ClinicalTrials.gov – NCT03338816

    • EXPLORE Natural History Study
      Trial Status

      Completed

      Completed

      A prospective observational study of adults with acute hepatic porphyria (AHP), including acute intermittent porphyria (AIP), variegate porphyria (VP), and hereditary coproporphyria (HCP), with recurrent attacks.

      Location(s):

      USA, Australia, Belgium, Bulgaria, Czech Republic, Finland, France, Germany, Italy, Israel, Mexico, Netherlands, Norway, Poland, South Africa, South Korea, Spain, Switzerland, Taiwan, UK

      For more info:

      ClinicalTrials.gov – NCT02240784

    • Phase 2 Open-Label Extension (OLE) Study
      Trial Status

      Completed

      Completed

      Evaluating the long-term safety and tolerability of givosiran in adults with acute intermittent porphyria (AIP), who participated in the Phase 2 trial.

      Location(s):

      USA, Sweden, UK

      For more info:

      ClinicalTrials.gov – NCT02949830

    • Phase 1 Study
      Trial Status

      Completed

      Completed

      Evaluating the safety and tolerability of givosiran (ALN-AS1) in adults with acute intermittent porphyria (AIP).

      Location(s):

      USA, Sweden, UK

      For more info:

      ClinicalTrials.gov – NCT02452372

    • ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
      Trial Status

      Recruiting

      Recruiting

      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.

      Location(s):

      USA, France, Germany

      For more info:

      ClinicalTrials.gov – NCT04883905

  • INCLISIRAN (HYPERCHOLESTEROLEMIA)

    • Phase 1 Study
      Trial Status

      Completed

      Completed

      Evaluated the safety and efficacy of inclisiran in adults with elevated low density lipoprotein cholesterol (LDL-C).

      Location(s):

      UK

      For more info:

      ClinicalTrials.gov – NCT02314442

  • LUMASIRAN (PRIMARY HYPEROXALURIA)

      • Phase 2 Study
        Trial Status

        Recruiting

        Recruiting

        Evaluating Lumasiran in patients with recurrent calcium oxalate kidney stone disease and elevated urinary oxalate levels.

        Location(s):

        USA

        For more info:

        ClinicalTrials.gov – NCT05161936

      • ILLUMINATE-C – Phase 3 Study
        Trial Status

        Active

        Active, Not Recruiting

        Evaluating the safety and efficacy of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1).

        Location(s):

        Israel, USA, France, Belgium, United Arab Emirates, Netherlands, Turkey, Australia, Jordan, Lebanon, UK

        For more info:

        ClinicalTrials.gov – NCT04152200

      • ILLUMINATE-A – Phase 3 Study
        Trial Status

        Completed

        Evaluating the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).

        Location(s):

        USA, UK, France, Germany, Netherlands, Switzerland, Israel, UAE

        For more info:

        ClinicalTrials.gov – NCT03681184

      • Phase 1/2 Study
        Trial Status

        Completed

        Evaluating the safety and efficacy of lumasiran in healthy volunteers and in patients with primary hyperoxaluria type 1 (PH1).

        Location(s):

        USA, UK, Germany, Israel, Netherlands

        Interested in learning more?

        Visit PH1trial.com for more information about the PH1 trial.

        For more info:

        ClinicalTrials.gov – NCT02706886

      • Phase 2 Open-Label Extension (OLE) Study
        Trial Status

        Active

        Active, Not Recruiting

        Evaluating the long-term safety and tolerability of lumasiran in patients with primary hyperoxaluria type 1.

        Location(s):

        France, Germany, Netherlands, UK, Israel

        For more info:

        ClinicalTrials.gov – NCT03350451

      • ILLUMINATE-B – Phase 3 Study
        Trial Status

        Active

        Active, Not Recruiting

        Evaluating the safety and efficacy of lumasiran in infants and young children with primary hyperoxaluria type 1 (PH1).

        Location(s):

        USA, UK, France, Germany, Israel

        For more info:

        ClinicalTrials.gov – NCT03905694

      • BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
        Trial Status

        Active

        Recruiting

        The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.

        Location(s):

        USA

        For more info:

        ClinicalTrials.gov – NCT04982393

  • CEMDISIRAN (COMPLEMENT-MEDIATED DISEASES)

    • Phase 1/2 Study
      Trial Status

      Completed

      Completed

      Evaluated the safety and efficacy of cemdisiran in healthy volunteers and adults with paroxysmal nocturnal hemoglobinuria (PNH).

      Location(s):

      Spain, UK

      For more info:

      ClinicalTrials.gov – NCT02352493

    • Phase 2 Study
      Trial Status

      Active

      Active, not recruiting

      Evaluating the effect of cemdisiran on proteinuria in adults with immunoglobulin A nephropathy (IgAN), who excrete >1 gram (gm) of protein per day despite standard of care, which includes treatment with angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARBs).

      Location(s):

      USA, Canada, Philippines, Spain, Sweden, UK, Singapore, France, Malaysia, Taiwan

      For more info:

      ClinicalTrials.gov – NCT03841448

  • VUTRISIRAN (ATTR AMYLOIDOSIS)

    • HELIOS-A – Phase 3 Study
      Trial Status

      Active

      Active, Not Recruiting

      Evaluating the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis).

      Location(s):

      USA, Argentina, Bulgaria, France, Portugal, Malaysia, Cyprus, Germany, Japan, South Korea, Greece, Sweden, UK, Taiwan, Belgium, Mexico, Italy, Australia, Canada, Netherlands, Spain, Brazil

      For more info:

      ClinicalTrials.gov – NCT03759379

    • HELIOS-B – Phase 3 Study
      Trial Status

      Enrolling

      Enrolling

      Evaluating the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with transthyretin amyloidosis with cardiomyopathy (ATTR amyloidosis with cardiomyopathy).

      Location(s):

      USA, Latvia, Spain, France, Sweden, Lithuania, Portugal, Australia, Republic of Korea, Germany, Norway, UK, Israel, Hungary, Denmark, Ireland, Canada, Moldova, Slovenia, Austria, Poland, Netherlands, Belgium, Croatia, Peru, Argentina, Japan, Saudi Arabia, Lebanon, Malaysia, Thailand, Colombia, Czech Republic

      For more info:

      ClinicalTrials.gov – NCT04153149
      ATTRResearch.com

  • ALN-AAT02 (ALPHA-1 LIVER DISEASE)

    • Phase 1/2 Study
      Trial Status

      Terminated

      Terminated

      Evaluating the safety and tolerability of single or multiple doses of ALN-AAT02 in participants with ZZ type alpha-1 antitrypsin deficiency (PiZZ) and biopsy-proven alpha-1 antitrypsin (AAT) deficiency-associated liver disease.

      Location(s):

      UK

      For more info:

      ClinicalTrials.gov – NCT03767829

  • ZILEBESIRAN (HYPERTENSION)

    • Phase 1 Study
      Trial Status

      Active

      Active, Not Recruiting

      Evaluating the safety and tolerability of zilebesiran (ALN-AGT01) in patients with hypertension.

      Location(s):

      UK

      For more info:

      ClinicalTrials.gov – NCT03934307

    • KARDIA-1 – Phase 2 Study
      Trial Status

      Recruiting

      Recruiting

      Evaluating the efficacy and safety of zilebesiran (ALN-AGT01) in patients with mild-to-moderate hypertension (KARDIA-1).

      Location(s):

      USA

      For more info:

      Clinicaltrials.gov – NCT04936035

    • KARDIA-2 – Phase 2 Study
      Trial Status

      Recruiting

      Recruiting

      The purpose of this study is to evaluate the effect of zilebesiran on systolic and diastolic blood pressure and to characterize the pharmacodynamic (PD) effects and safety of zilebesiran as add-on therapy.

      Location(s):

      USA

      For more info:

      Clinicaltrials.gov – NCT05103332

  • ALN-LEC (LECT2 Amyloidosis)

    • REFLECTION – Natural History Study
      Trial Status

      Terminated

      Terminated

      An observational study to characterize the natural history of leukocyte chemotactic factor 2 amyloidotic (ALECT2) disease.

      Location(s):

      USA, UK, India, Malaysia, Mexico, Egypt

      For more info:

      ClinicalTrials.gov – NCT03774784

  • ALN-HSD (NONALCOHOLIC STEATOHEPATITIS)

    • Phase 1 Study
      Trial Status

      Recruiting

      Recruiting

      The purpose of this study is to evaluate the safety and tolerability of single ascending doses of ALN-HSD in healthy participants (Part A) and multiple doses of ALN-HSD in patients with NASH (Part B).

      Location(s):

      UK, USA, Belgium, Bulgaria, Turkey

      For more info:

      ClinicalTrials.gov – NCT04565717

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