Current Environment:


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.


Rett Syndrome

Recruitment Status


Eligibility Criteria

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.


Intervention Type

Intervention Name




Phase 1



Min Age

5 Years

Max Age

8 Years

Download Date

February 14, 2024

Principal Investigator

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Primary Contact Information

Grace Correa

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For more information on this trial, visit


For more information and to contact the study team:

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) NCT06152237 Grace Correa