Gene therapy for severe Crigler-Najjar syndrome
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy for children with severe Crigler-Najjar syndrome type I. It aims to reduce the need for daily phototherapy by fixing the faulty gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of severe Crigler-Najjar syndrome type I caused by changes in the UGT1A1 gene
- You are at least 3 months old and no older than 10 years old
- You need regular phototherapy sessions to control your bilirubin levels
- You have not had a liver transplant
- You do not have active hepatitis B, hepatitis C, or HIV
- You do not have significant liver damage or brain problems from high bilirubin
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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