Status:
ACTIVE_NOT_RECRUITING
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
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Hemophilia
Eligibility:
MALE
12+ years
Phase:
PHASE3
Brief Summary
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 fre...
Detailed Description
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
Eligibility Criteria
Inclusion
- Inclusion criteria :
- 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
- Exclusion criteria:
- 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.
Exclusion
Key Trial Info
Start Date :
January 9 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 5 2026
Estimated Enrollment :
281 Patients enrolled
Trial Details
Trial ID
NCT03754790
Start Date
January 9 2019
End Date
November 5 2026
Last Update
December 17 2025
Active Locations (79)
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1
Phoenix Childrens Hospital Site Number : 8400009
Phoenix, Arizona, United States, 85016
2
Children's Hospital Los Angeles Site Number : 8400019
Los Angeles, California, United States, 90027
3
Center for Inherited Blood Disorders (CIBD) Site Number : 8400016
Orange, California, United States, 92868
4
Nemours Children's Clinic Site Number : 8400008
Jacksonville, Florida, United States, 32207