Gene Therapy for Girls with Rett Syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene therapy called GCB-002 for girls with Rett syndrome. The treatment aims to help with symptoms by targeting the genetic cause.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Female, ages 2 to 10 years old
- Diagnosis of Rett syndrome with a MECP2 gene mutation
- A parent or guardian must understand and sign a consent form
- Not currently in any other Rett syndrome or gene therapy trials
- No severe head injury that caused neurological problems
- No recent severe seizures (status epilepticus) within the last 3 months
- Not needing ventilation support (breathing machine)
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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