Gene therapy for severe Crigler-Najjar syndrome
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy called GNT0003, given after a pre-treatment to help it work, for people with severe Crigler-Najjar syndrome. The goal is to see if it can reduce the need for daily light treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have severe Crigler-Najjar syndrome and need light therapy (phototherapy) for at least 6 hours each day.
- A genetic test must confirm you have a change in the UGT1A1 gene.
- You need to have detectable antibodies against AAV8 (a type of virus used in the treatment).
- Your lab test results must be within acceptable ranges.
- You must use a highly effective birth control method if you can become pregnant.
- You must be part of a health care system in the country where the trial is done.
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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