Gene therapy for teens with hemophilia B
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1 study tests a one-time gene therapy treatment (BBM-H901) for boys ages 12 to 18 with hemophilia B. It aims to see if the treatment is safe and can help the body make more clotting factor over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (and your parent/guardian) must understand the study and sign consent forms.
- You must be a male age 12–17 (12 to under 18) and weigh at least 50 kg.
- You must have hemophilia B with very low factor FIX levels (≤2%), confirmed by lab testing.
- You must have used FIX replacement product before for at least 75 exposure days.
- You must have certain safe immune test results (not too many antibodies) against the gene-therapy “capsid.”
- Your recent health and labs must be safe: no current hepatitis B/C infection, no major liver disease, and blood counts/liver/kidney tests must meet study limits.
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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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.
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