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

Study on Investigational Medication for Hemophilia

Recruiting
12 years or above
Male
Phase 3
75 participants needed
11 Locations

Study Overview

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

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hemophilia
  • Age: 12 years or above
  • Gender: Male

Inclusion Criteria:

  • 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.
  • 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.
  • 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:

  • Known co-existing bleeding disorders other than congenital hemophilia A or B
  • History of arterial or venous thromboembolism, not associated with an indwelling venous access
  • History of intolerance to SC injection(s).
  • Current participation in immune tolerance induction therapy (ITI)
  • Prior gene therapy
  • Current or prior participation in a fitusiran trial
  • Current or prior participation in a gene therapy trial
  • 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
  • Presence of clinically significant liver disease AT activity <60% at Screening
  • Co-existing 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.

Updated on 10 May 2024. Study ID: NCT05662319

This study investigates the treatment of hemophilia using an investigational medication. Hemophilia is a condition where blood does not clot properly, leading to excessive bleeding. The purpose of this study is to measure how often bleeding episodes occur in male adults and adolescents aged 12 and older with hemophilia A or B. Participants in this study will switch from their previous treatment to the investigational medication.

Participants in this study will undergo several procedures. They will have regular telephone visits every two weeks and site visits approximately every eight weeks during the standard care period, and every four weeks during the investigational medication treatment period. Some visits may be conducted at home or remotely, depending on local regulations.

  • Who can participate: Males aged 12 years and older with severe hemophilia A or B are eligible. Participants must have had at least 4 bleeding episodes in the past 6 months requiring treatment. They must be able to comply with study requirements and provide consent.
  • Study details: Participants will switch from their current treatment to the investigational medication. They will have regular visits and be monitored for bleeding episodes.
  • Study Timelines: The study will last 50 months.

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