Last updated on June 2020

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Brief description of study

Primary Objective:

  • To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B

Secondary Objective:

  • To characterize the safety and tolerability
  • To characterize the pharmacokinetics (PK)

Detailed Study Description

Study duration per participant is approximately 160 weeks, including a 12-week fitusiran efficacy period

Clinical Study Identifier: NCT03974113

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Recruitment Status: Open

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