Last updated on June 2020

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors


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 | Factor IX Deficiency
  • Age: Between 12 - 100 Years
  • Gender: Male

Inclusion Criteria:

  • Males, 12 years of age
  • Severe hemophilia A or B without inhibitors
  • (Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is 2% at Screening; Inhibitors defined as Nijmegen modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening)
  • A minimum of 6 bleeding episodes requiring factor concentrate 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

Exclusion Criteria:

  • Known co-existing bleeding disorders other than hemophilia A or B
  • Antithrombin (AT) activity <60% at Screening
  • Co-existing thrombophilic disorder
  • Clinically significant liver disease
  • Active HCV 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: Closed


Brief Description Eligibility Contact Research Team


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