Gene therapy trial for children with CTNNB1 syndrome
Treatments studied
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a one-time gene replacement therapy given into the fluid around the brain to help children with CTNNB1 neurodevelopmental syndrome. The goal is to see if replacing the faulty gene can improve development and reduce symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 2 and 12 years old and have a confirmed diagnosis of CTNNB1 syndrome.
- Your child must meet specific weight requirements based on their age (for example, a 2-year-old needs to weigh at least about 24 pounds).
- Parents must be able to stay within 1 hour of the study site for at least 4 months after treatment.
- Your child cannot have had prior gene or cell therapy or certain other medical conditions.
- Your child must not have certain lab abnormalities or active infections like hepatitis or HIV.
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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