Gene therapy for hemophilia B with AAV5 antibodies
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are an adult (age defined by your country's laws).
- You have severe or moderately severe hemophilia B (factor IX level 2% or less) and are on regular preventive factor IX treatment.
- You have tested positive for AAV5 antibodies during screening.
- You have used factor IX treatment for more than 150 days in the past.
- You have been on a stable factor IX preventive plan for at least 2 months before screening.
- You are able to complete an electronic diary during the lead-in 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 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 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 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.
This study checks long-term safety and outcomes in men with hemophilia B who previously received CSL222. It may help your care team better understand how well the treatment lasts over many years.
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