Gene therapy for teens with severe hemophilia B
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a single dose of CSL222, a gene therapy, in adolescent boys with severe or moderately severe hemophilia B. The goal is to see if it can reduce or eliminate the need for regular factor IX infusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be a boy between about 11 and a half and 17 years old.
- You have severe or moderately severe hemophilia B (factor IX level 2% or less).
- You have been on regular factor IX prophylaxis for at least 2 months before joining.
- You have had at least 75 prior factor IX treatments.
- You have never had a factor IX inhibitor (antibody that blocks treatment).
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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