Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) (NCT06152237) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
United States, Canada, United Kingdom6 participantsStarted 2023-12-12
Plain-language summary
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.
Who can participate
Age range5 Years – 8 Years
SexFEMALE
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Inclusion Criteria:
* Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.
* Participant is between ≥5 to ≤8 years of age at the time of consent.
* Participant must be up to date with all relevant local vaccination requirements, with last vaccination dose received at least 42 days prior to the start of the immunosuppression regimen.
* Participant's parent/caregiver must be willing to allow participant to receive blood or blood products for the treatment of an AE if medically needed.
Exclusion Criteria:
* Participant has another neurodevelopmental disorder independent of the MECP2 gene loss of function mutation, or any other genetic syndrome with a progressive course.
* Participant has a history of brain injury that causes neurological problems.
* Participant had grossly abnormal psychomotor development in the first 6 months of life.
* Participant has a diagnosis of atypical Rett syndrome.
* Participant has an MECP2 mutation that does not cause Rett syndrome.
* Participant requires non-invasive and invasive ventilatory support.
* Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, other medical conditions, or contraindications to any medications required for IT administration.
* Participant has acute or chronic hepatitis B or C infections.