Found 3 clinical trials
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B With or Without Inhibitory Antibodies to Factor VIII or IX
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: To characterize the long-term efficacy of fitusiran as assessed by the frequency of: Bleeding episodes Spontaneous bleeding episodes Target joint bleeding episodes To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants …
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- 19 Feb, 2024
- 41 locations
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
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)
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- 19 Feb, 2024
- 11 locations
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
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)
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- 19 Feb, 2024
- 6 locations