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

  • End date
    Sep 1, 2022
  • participants needed
  • sponsor
    Genzyme, a Sanofi Company
Updated on 4 April 2021
For site information, send an email with site number to
Primary Contact
Investigational Site Number 0140 (2.3 mi away) Contact
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immune tolerance
factor ix
severe haemophilia a
antihemophilic factor
christmas disease
immune tolerance induction
bethesda assay


Primary Objective:

To characterize the frequency of bleeding episodes while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor or bypassing agent (BPA) prophylaxis

Secondary Objectives:

  • To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis:
  • the frequency of spontaneous bleeding episodes
  • the frequency of joint bleeding episodes
  • health related quality of life (HRQOL) in patients 17 years of age
  • To characterize the frequency of bleeding episodes during the onset and treatment periods in patients receiving fitusiran
  • To characterize the safety and tolerability of fitusiran
  • Annualized weight-adjusted consumption of factor/BPA


Study duration per participant is approximately 13 months including 6-Month factor or BPA prophylaxis period and 7-Month fitusiran treatment period. Study duration for patients in the subgroup of Cohort A is approximately 7 months corresponding to a 7-Month fitusiran treatment period.

Participants completing the treatment period will be proposed to enroll in an extension study

Condition Hemophilia A, Factor IX Deficiency, antagonists & inhibitors, Hemophilia, hema
Treatment fitusiran, Fitusiran (ALN-AT3SC)
Clinical Study IdentifierNCT03549871
SponsorGenzyme, a Sanofi Company
Last Modified on4 April 2021

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