Gene therapy for hemophilia B using a FIX gene treatment
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a one-time (or limited) gene therapy meant to help the body make more factor IX (FIX), which can reduce bleeding in people with hemophilia B. You may be able to qualify if you have significant bleeding history, no factor IX “blockers” (inhibitors), and can follow strict safety and pregnancy-prevention rules.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You can sign consent and follow all study visits and instructions
- You are male and have confirmed hemophilia B with low factor IX levels (≤2 IU/dL or ≤2% of normal)
- You’ve had enough bleeding episodes (about 4 or more per year) that required factor IX infusions or prevention infusions
- You do not have measurable factor IX inhibitors (and you’ve never had inhibitors before)
- You agree to use barrier contraception until test results show the gene therapy “vector” is not detectable for three samples in a row
- You are not in another drug or gene therapy study recently (gene transfer in last 6 months, or any investigational drug in last 12 weeks)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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 study tests a new gene therapy called ANB-002 for men with severe hemophilia B (factor IX levels at or below 2%). The goal is to see if it's safe and how well it might help your blood clot normally.
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.
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.
This trial tests a gene therapy called CSL222 for adults with hemophilia B who have antibodies against the AAV5 virus used to deliver the therapy. It aims to see if the treatment is safe and effective even when these antibodies are present.
This trial asks people with hemophilia who previously received certain gene therapies to have a liver biopsy. The goal is to improve understanding of how the gene therapy affects the liver and to support safer future care.
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