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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Other trials that look related to this one.
This trial studies ZS801, a gene therapy, in adults with hemophilia B who have very low natural FIX (a clotting protein) and frequent bleeding or joint problems. It aims to see how well it works and whether it is safe, especially by monitoring blood counts, infections, and immune responses.
This Phase 1/2 study tests ZS801, a gene-related treatment, to see how safe it is and whether it helps prevent bleeding in adults with hemophilia B. You may be considered if you’ve had frequent bleeds or joint problems and meet several lab and health requirements.
This study tests a one-time gene therapy (CSL222) for teenage boys with severe or moderately severe hemophilia B. It aims to help their bodies produce the clotting factor they need, potentially reducing or eliminating the need for regular factor infusions.
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This trial tests a new gene therapy (REGV131-LNP1265) that aims to let your body produce its own factor IX, reducing or stopping the need for regular infusions. It is for children and adults with severe hemophilia B who are already using factor IX replacement therapy.
This gene therapy trial uses a modified virus to deliver a working copy of the gene that makes factor IX, the protein missing in hemophilia B. The goal is to help your body produce enough factor IX to reduce or prevent bleeding episodes, potentially reducing the need for regular factor IX infusions.
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