Last updated on June 2020

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis


Are you eligible to participate in this study?

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

  • Conditions: Hemophilia A | Hemophilia | antagonists & inhibitors | Factor IX Deficiency
  • Age: Between 12 - 100 Years
  • Gender: Male

Inclusion Criteria:

  • Males, 12 years of age
  • Severe hemophilia A or B (as evidenced by a central laboratory measurement at screening or documented medical record evidence of FVIII <1% or FIX level 2%)
  • A minimum of 2 bleeding episodes requiring BPA treatment within the last 6 months prior to Screening for patients with inhibitory antibodies to factor VIII or factor IX (Cohort A). A minimum of 1 bleeding episode requiring factor treatment within the last 12 months prior to Screening for patients without inhibitory antibodies to factor VIII or factor IX (Cohort B).
  • Must meet either the definition of inhibitor or non-inhibitor patient as below:
  • Inhibitor:Use of BPAs for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:
  • 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
  • The subgroup of patients in Cohort A patients must additionally meet the following criteria to be eligible to start treatment with fitusiran directly after the screening period:
  • Hemophilia B with inhibitory antibody to Factor IX as defined above
  • Not responding adequately to BPA treatment (historical ABR 20) prior to enrollment
  • In the opinion of the Investigator, with approval of Sponsor Medical Monitor, 6-month BPA prophylaxis period should be omitted.
  • Non-inhibitor:Use of factor concentrates for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following
    criterion
  • Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening and
  • No use of bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening and
  • No history of immune tolerance induction therapy within the past 3 years prior to Screening.
  • Documented prophylactic treatment with factor concentrates or bypassing agents for the treatment of hemophilia A or B for at least 6 months prior to Screening
  • Adherent to the prescribed prophylactic therapy for at least 6 months prior to Screening per Investigator assessment
  • Willing and able to comply with the study requirements and to provide written informed consent and assent

Exclusion Criteria:

  • Known co-existing bleeding disorders other than hemophilia A or B
  • AT activity <60% at Screening
  • Co-existing thrombophilic disorder
  • Clinically significant liver disease
  • Active Hepatitis C virus infection
  • Acute or chronic Hepatitis B virus infection
  • HIV positive with a CD4 count of <200 cells/L
  • History of arterial or venous thromboembolism
  • Inadequate renal function
  • History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
  • History of intolerance to SC injection(s)
  • Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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