Found 264 clinical trials
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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- 04 Dec, 2020
- 6 locations
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia (ATLAS-NEO)
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory …
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- 24 May, 2024
- 54 locations
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting.
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- 16 Feb, 2024
- 95 locations
Pharmacokinetics Pharmacodynamics and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to …
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- 16 Feb, 2024
- 35 locations