Fitusiran for young boys with hemophilia A or B
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests if fitusiran, given as a shot, can help prevent bleeding in boys ages 1 to 12 with severe hemophilia (factor VIII under 1% or factor IX at 2% or lower). It includes boys with or without inhibitors and aims to understand safety and how well it works.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be a boy between 1 and 11 years old.
- Have severe hemophilia A (factor VIII under 1%) or B (factor IX at 2% or lower).
- In the last 3 months, you have been using bypassing agents (if you have inhibitors) or clotting factor concentrates (if you don't have inhibitors) to prevent or treat bleeding.
- Have adequate veins for blood draws.
- Parent or guardian must sign consent, and you may need to give your okay too.
- Not have other bleeding disorders, liver disease, or a history of blood clots.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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