Status:
RECRUITING
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
Lead Sponsor:
Sanofi
Conditions:
Hemophilia A
Eligibility:
MALE
12+ years
Phase:
PHASE4
Brief Summary
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis. This study aims to evaluate the safety and tolerabilit...
Eligibility Criteria
Inclusion
- Male participants must be ≥12 years of age inclusive, at the time of signing the informed consent
- Diagnosis of severe congenital hemophilia A (FVIII \< 1%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
- Inhibitor titer of ≥0.6 BU/mL at Screening, or
- Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, or
- Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of anamnestic response.
- Participants who are currently on the full labeled dose of emicizumab prophylaxis, irrespective of inhibitor/non-inhibitor status.
- Signed informed consent/assent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Exclusion
- Participants are excluded from the study if any of the following criteria apply:
- Known coexisting bleeding disorders
- History of antiphospholipid antibody syndrome.
- History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants who have experienced thrombosis associated with indwelling venous access may be enrolled.
- Presence of clinically significant liver disease
- Current or prior participation in a fitusiran trial
- Current or prior participation in a gene therapy trial
- AT activity \<60% at Screening, as determined by central laboratory measurement
- Coexisting thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
- Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
- Presence of acute or chronic hepatitis B infection
- Known to be HIV positive with CD4 count \<200 cells/μL.
- Reduced renal function
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Key Trial Info
Start Date :
March 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 30 2029
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT06145373
Start Date
March 1 2024
End Date
November 30 2029
Last Update
September 30 2025
Active Locations (2)
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1
Children's Hospital Los Angeles- Site Number : 8400005
Los Angeles, California, United States, 90027
2
Investigational Site Number : 1580001
Taipei, Podlaskie, Taiwan, 10041