Gene therapy for hemophilia B
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be male and at least 2 years old.
- Must have a confirmed diagnosis of hemophilia B with very low factor IX (less than 2% of normal).
- Must have at least 4 bleeding episodes a year that need factor IX treatment, or use factor IX regularly to prevent bleeding.
- Must not have ever had a factor IX inhibitor (antibodies that block the treatment).
- Must have adequate liver function and no active hepatitis B or C infections.
- Must agree to use reliable barrier contraception during the study period.
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 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.
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 Phase 3 trial is checking how safe and effective two gene therapies are for people with hemophilia A or hemophilia B. It only includes people who already received the study medicines through specific Pfizer studies, so it’s mainly for that existing participant group.
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