Last updated on May 2019

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


Brief description of study

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 period in patients receiving fitusiran
  • To characterize the safety and tolerability of fitusiran
  • Annualized weight-adjusted consumption of factor/BPA

Detailed Study Description

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

Clinical Study Identifier: NCT03549871

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Recruitment Status: Open


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