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This study evaluates a treatment called GCB-002 in children with Rett Syndrome. Girls aged 2 to 10 with a disease-causing variant in the MECP2 gene confirmed by genetic testing can take part. It is a study with no applicable phase and is enrolling participants by invitation.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Age range from 2 to 10 years old, female; 2. The clinical diagnosis of the subject is RTT, and after genetic testing, it was found to be a pathogenic variant of the MECP2 gene; 3. The legal guardian is able to understand the requirements and procedures of the research plan, voluntarily participate, and sign an informed consent form. Exclusion Criteria: 1. Has participated in or is currently participating in other RTT drug clinical trials or other AAV gene therapy clinical studies; 2. The subject has a history of head injuries that can cause neurological disorders such as epilepsy, physical disabilities, etc; 3. The subject has MECP2 gene mutation but does not cause RTT; 4. Subjects with allergic constitution, including those allergic or hypersensitive to prednisolone, other glucocorticoids, their excipients, and local anesthetics; 5. The subjects had status epilepticus in the 3 months prior to enrollment; 6. Subjects require invasive or non-invasive ventilation support; 7. Serum anti AAV9 neutralizing antibody titer\>1:200; The researchers believe that it is not suitable to participate in this study.