Gene therapy for Rett syndrome in girls
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene therapy called GCB-002 for girls with Rett syndrome caused by a MECP2 gene change. It may help improve symptoms by targeting the root genetic cause.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Girls between 2 and 10 years old
- Diagnosed with Rett syndrome and have a mutation in the MECP2 gene
- Not currently in another Rett syndrome drug trial or gene therapy study
- No history of head injury that caused seizures or physical disability
- No severe allergies to medicines like prednisolone or anesthetics
- No seizures lasting more than 5 minutes in the past 3 months
- Not needing a breathing machine or non-invasive breathing support
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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