Gene therapy for severe hemophilia B
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have severe or moderately severe hemophilia B (factor IX level of 2% or less or a genetic change that causes severe hemophilia B).
- You are currently taking factor IX replacement therapy to prevent bleeding (prophylaxis) and have used it before.
- You have been in this study's lead-in period or a separate lead-in study for at least 6 months, recording bleeding episodes while on prophylaxis.
- You have never had a factor IX inhibitor (antibody) detected two or more times.
- You do not have antibodies against the AAV8 virus (a common virus used in gene therapy).
- You have not had any previous gene therapy using an AAV virus.
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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