Gene therapy trial for sickle cell disease using BAH243
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a gene therapy that uses a modified virus to deliver a healthy gene to your blood stem cells. It aims to help people with sickle cell disease produce normal red blood cells and reduce painful crises.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have sickle cell disease with certain genetic types (βS/βS, βS/β0, or βS/β+).
- You need to be between 2 and 50 years old, weigh at least 6 kg, and be in fairly good health (performance status 60% or higher).
- You must have had at least 4 severe pain crises (called VOEs) in the last 2 years and either failed or cannot tolerate hydroxyurea.
- You cannot have had a stroke, severe brain blood vessel problems, or need blood thinners during the treatment period.
- You cannot have certain infections (HIV, hepatitis, etc.), advanced liver disease, or low blood cell counts.
- You must agree to use effective birth control from screening until 6 months after the infusion.
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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