Gene therapy study for teens with severe hemophilia B
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are male and between 11.5 and 17 years old at the start.
- You have severe or moderately severe hemophilia B (factor IX level 2% or less).
- You have been on regular factor IX replacement therapy for at least 2 months before screening.
- You have had at least 75 days of factor IX treatment in the past.
- You do not have a history of factor IX inhibitors (antibodies that block treatment).
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 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 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 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 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 trial tests a single dose of ANB-002, an experimental therapy for hemophilia B. It aims to see if the drug is safe and effective for people with severe hemophilia B who have been treated with factor IX before and do not have inhibitors.
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