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)

    • APOLLO
      Trial Status

      Active

      Active, Not Recruiting

      Evaluating 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, 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
      Visit APOLLOtrial.com for more about the APOLLO trial.

    • 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, 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

    • Expanded Access Protocol Study
      Trial Status

      Active

      Active, Not Recruiting

      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

    • 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

       

  • FITUSIRAN (HEMOPHILIA A OR B)

    • Phase 2 Open-Label Extension Study
      Trial Status

      Active

      On Hold

      Evaluating the long-term safety and tolerability of fitusiran in adult men with moderate or severe hemophilia A or B, with or without inhibitors, who participated in the Phase 1 study.

       

      Location(s):

      USA, Bulgaria, Russia, Switzerland, UK

       

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

       

      For more info:

      ClinicalTrials.gov – NCT02035605

       

  • GIVOSIRAN (ACUTE HEPATIC PORPHYRIAS)

    • Phase 1 Study
      Trial Status

      Enrolling

      Enrolling

      Evaluating the safety and tolerability of givosiran 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

    • EXPLORE Natural History Study
      Trial Status

      Active

      Active, Not Recruiting

      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, Poland, Spain, Switzerland, UK

       

      For more info:

      ClinicalTrials.gov – NCT02240784

       

  • INCLISIRAN (HYPERCHOLESTEROLEMIA)

    • ORION-1 Phase 2 Study
      Trial Status

      Active

      Active, Not Recruiting

      Evaluating the safety and tolerability of inclisiran in adults with atherosclerotic cardiovascular disease (ASCVD).

       

      Location(s):

      USA, Bulgaria, Russia, Switzerland, UK

       

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

       

      For more info:

      ClinicalTrials.gov – NCT02597127

    • 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

       

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

       

      For more info:

      ClinicalTrials.gov – NCT02314442

       

  • ALN-GO1 (PRIMARY HYPEROXALURIA)

    • Phase 1/2 Study
      Trial Status

      Enrolling

      Enrolling

      Evaluating the safety and efficacy of ALN-GO1 in healthy volunteers and in patients with primary hyperoxaluria type 1 (PH1).

       

      Location(s):

      France, UK

       

      Interested in learning more?

      Please email us at clinicaltrials@alnylam.com

       

      For more info:

      ClinicalTrials.gov – NCT02706886

       

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