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

  • STATUS
    Recruiting
  • End date
    May 19, 2028
  • participants needed
    20
  • sponsor
    Sanofi
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Updated on 6 February 2025
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Summary

This is an exploratory, single group, Phase 1, 1-arm 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 ≥18 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 a transition 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 up to 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.

Details
Condition Hemophilia A
Age 18years or above
Treatment Emicizumab, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC), Fitusiran (SAR439774)
Clinical Study IdentifierNCT06145373
SponsorSanofi
Last Modified on6 February 2025

Eligibility

 Yes No Not Sure

Inclusion Criteria

Male participants must be ≥18 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
Capable of giving signed informed consent 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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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