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.

  • PATISIRAN (hATTR AMYLOIDOSIS)

    • Expanded Access Protocol Study
      Trial Status

      Active

      Active, Enrolling

      Providing expanded access of patisiran to adults with hATTR amyloidosis with polyneuropathy who meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements.

      Location(s):

      USA (various)

      For more info:

      ClinicalTrials.gov – NCT02939820

    • 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

      For more info:

      ClinicalTrials.gov – NCT01617967

  • GIVOSIRAN (ACUTE HEPATIC PORPHYRIAS)

    • ENVISION – Phase 3 Study
      Trial Status

      Enrolling

      Enrolling

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

      Location(s):

      USA, Australia, Belgium, Denmark, Germany, Spain, UK

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT03338816

    • EXPLORE Natural History Study
      Trial Status

      Enrolling

      Enrolling

      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, Bulgaria, Czech Republic, Finland, France, Germany, Italy, Netherlands, Norway, Spain, Switzerland, UK

      For more info:

      ClinicalTrials.gov – NCT02240784

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

      Active

      Active, Not Recruiting

      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):

      US, Sweden, UK

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT02949830

    • Phase 1 Study
      Trial Status

      Completed

      Completed

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

      Location(s):

      USA, Sweden, UK

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT02452372

  • INCLISIRAN (HYPERCHOLESTEROLEMIA)

  • LUMASIRAN (PRIMARY HYPEROXALURIA)

    • ILLUMINATE-A – Phase 3 Study
      Trial Status

      Enrolling

      Enrolling

      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, Jordan, UAE, Japan

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT03681184

    • Phase 1/2 Study
      Trial Status

      Active, Not Recruiting

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

      Location(s):

      USA, France, UK, Germany, Israel, Netherlands, Jordan

      Interested in learning more?

      Visit PH1trial.com for more about the PH1 trial.

      For more info:

      ClinicalTrials.gov – NCT02706886

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

      Enrolling By Invitation

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

      Location(s):

      France

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT03350451

  • CEMDISIRAN (ALN-CC5)

    • Phase 2 Study
      Trial Status

      Enrolling

      Enrolling

      Evaluating the safety, tolerability, and pharmacokinetics of cemdisiran (ALN-CC5) in adults with atypical hemolytic uremic syndrome (aHUS).

      Location(s):

      Bosnia and Herzegovina, Estonia, Georgia, Latvia, Lithuania, Macedonia, Moldova, Serbia, Sweden

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT03303313

    • 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

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

      For more info:

      ClinicalTrials.gov – NCT02352493

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