Testing fitusiran to prevent bleeds in severe hemophilia A patients switching from emicizumab

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

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

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
• Email: Contact-US@sanofi.com
• Phone: 800-633-1610

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.