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Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial is testing a new medication called to help prevent bleeding episodes in children with hemophilia A or B. is given as a weekly injection under the skin. Researchers want to see if this treatment works better than the standard care that participants have been receiving.
Children with hemophilia struggle with frequent, serious bleeding episodes that can be painful and dangerous. Current treatments either require frequent infusions or don't always prevent bleeds well, especially in patients who have developed inhibitors (resistance to standard factor replacement). This medication aims to offer a simpler, more effective way to prevent bleeds.
You likely qualify if…
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You would participate in this trial for about 14 months total: one month of screening and baseline visits, 12 months of receiving the medication, and one month of follow-up. During the 12-month treatment phase, you would receive a weekly injection of the medication, either at home or at the study clinic — whatever works best for you. The study team will track how many bleeding episodes you have and monitor your safety throughout.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
Argentina
Australia
Austria
Canada
China