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Clinical Trials in Taiwan / NCT06145373
Recruiting Phase 4

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

NCT06145373 · tracked via the Priya Life Science Taiwan tracker
Sponsor
Sanofi
Phase
Phase 4
Started
2024-03-01
Last updated
2026-09-23

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

Fitusiran (SAR439774) →Clotting factor concentrates (CFC) or bypassing agents (BPA) →Antithrombin concentrate (ATIIIC) →Emicizumab →

Fitusiran (SAR439774): Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous (SC) injection

Clotting factor concentrates (CFC) or bypassing agents (BPA): Pharmaceutical form: Solution for injection-Route of administration: Intravenous (IV) injection

Antithrombin concentrate (ATIIIC): Pharmaceutical form:Solution for injection-Route of administration:Intravenous (IV) injection

Emicizumab: Pharmaceutical form:Solution for injection-Route of administration:SC injection

Study 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 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.

Eligibility

Sex
MALE
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * 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 Criteria: 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.

Primary outcome measure(s)

  • Number of participants with Adverse events (AEs) during the fitusiran treatment — From Day 1 up to Month 4
    Incidence, severity, and seriousness, of AEs occurred during fitusiran treatment period will be reported

Trial sites (3)

FacilityCityRegionStatus
Children's Hospital Los Angeles- Site Number : 8400005 Los Angeles California Recruiting
Southern Specialty Research- Site Number : 8400011 Flowood Mississippi Recruiting
Investigational Site Number : 1580001 Taipei Taiwan Recruiting

More Sanofi trials in Taiwan

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Long-term Safety and Efficacy Study of Riliprubart in Participants With CIDP Enrolling by invitation
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B Recruiting
A Maintenance Study to Investigate the Efficacy and Safety of Duvakitug in Participants With Moderately to Severely Active Crohn's Disease Recruiting
Long-term Safety and Efficacy Evaluation of Lunsekimig (SAR443765) in Adult Participants With Asthma Enrolling by invitation

Other trials for the same condition

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A Active, not recruiting
A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A Active, not recruiting
Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A Active, not recruiting
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A Recruiting
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06145373 on ClinicalTrials.gov ↗ ← All trials in Taiwan