Actively Recruiting
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
Led by Sanofi · Updated on 2025-09-30
20
Participants Needed
2
Research Sites
300 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 tolerability of switching to fitusiran after a transition period from the last dose of emicizumab. The study will be conducted in male participants with severe hemophilia A, with or without inhibitors, aged ≥12 years, who were previously receiving emicizumab prophylaxis. Study details include: * The study duration will be up to approximately 28 months: * There will be an approximately 2-month screening period. * There will be an approximately 2-month period before fitusiran treatment starts (pre-fitusiran treatment period) * The fitusiran treatment duration will be up to 18-months (fitusiran treatment period) * The antithrombin (AT) follow-up (FU) period will be approximately 6 months after the last dose of fitusiran (during which the AT activity level will be monitored at approximately monthly intervals following the final fitusiran dose until AT activity levels return to at least 60%). * The study site visits are scheduled at monthly/ every 2 months intervals of 28 days (4 weeks) / 56 days (8 weeks), respectively, during the fitusiran treatment period.
CONDITIONS
Official Title
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants aged 12 years or older at the time of consent
- Diagnosis of severe congenital hemophilia A with factor VIII level less than 1%
- Inhibitor titer of 0.6 BU/mL or higher at screening, or
- Inhibitor titer less than 0.6 BU/mL at screening with medical record evidence of two consecutive titers 0.6 BU/mL or higher, or
- Inhibitor titer less than 0.6 BU/mL at screening with medical record evidence of anamnestic response
- Currently receiving the full labeled dose of emicizumab prophylaxis regardless of inhibitor status
- Signed informed consent or assent including agreement to comply with study requirements
You will not qualify if you...
- 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 (except thrombosis linked to indwelling venous access)
- Clinically significant liver disease
- Current or prior participation in a fitusiran trial
- Current or prior participation in a gene therapy trial
- Antithrombin activity less than 60% at screening
- Coexisting thrombophilic disorder or positive Hepatitis C antibody unless viral load is negative and no cirrhosis
- Presence of acute hepatitis A or E
- Presence of acute or chronic hepatitis B infection
- Known HIV positive status with CD4 count less than 200 cells/µL
- Reduced kidney function
AI-Screening
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Trial Site Locations
Total: 2 locations
1
Children's Hospital Los Angeles- Site Number : 8400005
Los Angeles, California, United States, 90027
Actively Recruiting
2
Investigational Site Number : 1580001
Taipei, Podlaskie, Taiwan, 10041
Actively Recruiting
Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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