For Healthcare Professionals

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

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About the study

This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment. The total study duration will be up to approximately 50 months (200 weeks, 1 study month is equivalent to 4 weeks) and will include: * A screening period up to approximately 60 days, * A standard of care (SOC) period of approximately 6 study months (24 weeks), * A fitusiran treatment period of approximately 36 study months (144 weeks), * An antithrombin (AT) follow-up period of approximately 6 study months (24 weeks) but may be shorter or longer depending on individual participants AT recovery. The frequency for telephone visits will be approximately every 2 weeks. For site visits the frequency will be approximately every 8 weeks during the SOC period and approximately every 4 weeks during the fitusiran treatment period. If applicable and if allowed by local regulation, home and/or remote visits may be conducted during the study
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Inclusion Criteria:

  1. Diagnosis of severe congenital hemophilia A or B (FVIII <1% or FIX level ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
  2. For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months prior to screening.
  3. Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of participants under the age of legal consent, per local and national requirements

EXCLUSION CRITERIA

Exclusion Criteria:

  1. Known co-existing bleeding disorders other than congenital hemophilia A or B
  2. History of arterial or venous thromboembolism, not associated with an indwelling venous access
  3. History of intolerance to SC injection(s).
  4. Current participation in immune tolerance induction therapy (ITI)
  5. Prior gene therapy
  6. Current or prior participation in a fitusiran trial
  7. Current or prior participation in a gene therapy trial
  8. Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer
  9. Presence of clinically significant liver disease AT activity <60% at Screening
  10. Co-existing thrombophilic disorder
  11. Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
  12. Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
  13. Presence of acute or chronic hepatitis B infection
  14. Known to be HIV positive with CD4 count <200 cells/μL.
  15. 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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Study Locations

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How to Apply


Contact the study center to learn if this study is a good match for you.
Phone iconCall 800-633-1610Email iconEmail Study Center

Study Details


Contition

Hemophilia

Age

12+

Phase

PHASE3

Participants Needed

75

Est. Completion Date

Mar 1, 2028

Treatment Type

INTERVENTIONAL


Sponsor

Sanofi

ClinicalTrials.gov NCT Identifier

NCT05662319

Study Number

EFC17574

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