Gene therapy for severe Crigler-Najjar liver jaundice
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial studies a gene therapy for people with severe Crigler-Najjar syndrome, a condition that causes high bilirubin (jaundice) due to a UGT1A1 gene change. It may help reduce the need for constant bilirubin-lowering treatment like phototherapy.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have severe Crigler-Najjar syndrome with a confirmed UGT1A1 gene mutation
- You need ongoing phototherapy for symptom control
- You are at least 9 years old (or will be by the consent signing date)
- You (and your child, if relevant) can give consent/assent in writing
- You have not had a liver transplant
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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