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

RecruitingPhase 4Ages 12 Years - anyMaleSanofi
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In plain words

What the trial is, and whether it could suit you

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 โ‰ฅ12 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

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Male participants must be โ‰ฅ12 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 โ‰ฅ0.6 BU/mL at Screening, or * Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers โ‰ฅ0.6 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/ฮผL. * 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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What it tests

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

3 sites
Children's Hospital Los Angeles- Site Number : 8400005
Los Angeles, United States
Southern Specialty Research- Site Number : 8400011
Flowood, United States
Investigational Site Number : 1580001
Taipei, Taiwan
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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