Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Running, not enrolling · Phase 3

Conditions studied: Hemophilia

In brief

Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: * To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: * Bleeding episodes * Spontaneous bleeding episodes * Joint bleeding episodes * To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age

Key facts

Study ID

NCT03754790

Run by

Genzyme, a Sanofi Company

People needed

281

Starts

2019-01-09

Expected to finish

2026-11-05

Last updated by the study team

2025-12-17

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
• Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
• Male
• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative

You may not qualify if…

• Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis
• Current participation in immune tolerance induction treatment (ITI)
• Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period
• Use of compounds other than factor concentrates or BPAs for hemophilia treatment
• Current or prior participation in a gene therapy trial
• Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) >1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST > 5 x ULN for patients who were in the fitusiran arm in the parent study
• Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort:
• Clinically significant liver disease
• History of arterial or venous thromboembolism
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Where it is running

• Children's Hospital Los Angeles Site Number : 8400019 — Los Angeles, California, United States
• Center for Inherited Blood Disorders (CIBD) Site Number : 8400016 — Orange, California, United States
• Nemours Children's Clinic Site Number : 8400008 — Jacksonville, Florida, United States
• St Joseph's Children's Hospital of Tampa Site Number : 8400002 — Tampa, Florida, United States
• Rush University Medical Center -1725 W Harrison St Site Number : 8400001 — Chicago, Illinois, United States
• ~Massachusetts General Hospital Site Number : 8400011 — Boston, Massachusetts, United States
• University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012 — Ann Arbor, Michigan, United States
• Alliance for Childhood Diseases Site Number : 8400007 — Las Vegas, Nevada, United States
• Childrens Hospital Medical Center of Akron Site Number : 8400006 — Akron, Ohio, United States
• Investigational Site Number : 0360001 — Camperdown, New South Wales, Australia
• Investigational Site Number : 0360003 — Prahran, Victoria, Australia
• Investigational Site Number : 0360002 — Murdoch, Western Australia, Australia
• Investigational Site Number : 1240001 — Montreal, Quebec, Canada
• Investigational Site Number : 1560004 — Beijing, China
• Investigational Site Number : 1560007 — Beijing, China
• Investigational Site Number : 1560014 — Changsha, China
• Investigational Site Number : 1560009 — Chengdu, China
• Investigational Site Number : 1560002 — Guangzhou, China
• Investigational Site Number : 1560011 — Guiyang, China
• Investigational Site Number : 1560005 — Hangzhou, China
• Investigational Site Number : 1560008 — Jinan, China
• Investigational Site Number : 1560012 — Kunming, China
• Investigational Site Number : 1560013 — Lanzhou, China
• Investigational Site Number : 1560010 — Qingdao, China
• Phoenix Childrens Hospital Site Number : 8400009 — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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