Testing fitusiran to prevent bleeds in severe hemophilia A patients switching from emicizumab
An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors
This study is testing if fitusiran can help prevent bleeding in men with severe hemophilia A who are switching from another treatment called emicizumab.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 12 Years and up |
| Sex | Male |
| Sponsor | Sanofi Industry-sponsored |
| Drugs / interventions | emicizumab |
| Locations | 2 sites (Los Angeles, California and 1 other locations) |
| Trial ID | NCT06145373 on ClinicalTrials.gov |
What this trial studies
This exploratory Phase 1 study evaluates the safety and tolerability of fitusiran prophylaxis in male participants with severe hemophilia A who have previously received emicizumab. The study involves a transition period from emicizumab to fitusiran, followed by an 18-month treatment period with fitusiran. Participants will be monitored for antithrombin activity levels during a follow-up period after treatment. The study aims to gather data on the effectiveness of fitusiran in preventing bleeds in this patient population.
Who should consider this trial
Good fit: Ideal candidates are male adults aged 18 and older with severe hemophilia A who are currently on emicizumab prophylaxis.
Not a fit: Patients with known coexisting bleeding disorders will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce bleeding episodes in patients with severe hemophilia A.
How similar studies have performed: While this approach is novel, previous studies have shown promise in using fitusiran for hemophilia treatment.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Los Angeles, California and 1 other locations
- Children's Hospital Los Angeles- Site Number : 8400005 — Los Angeles, California, United States (Recruiting)
- Investigational Site Number : 1580001 — Taipei, Podlaskie, Taiwan (Recruiting)
Study contacts
- Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
- Email: Contact-US@sanofi.com
- Phone: 800-633-1610
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.