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ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

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

A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome

NCT06152237•Taysha Gene Therapies, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

5 - 8 years

Sex

FEMALE

Healthy Volunteers

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.

Locations (5)

Rush University Medical Center & Children's Hospital

Chicago, Illinois, United States

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, United States

Washington University, St. Louis

St Louis, Missouri, United States

CHU Ste-Justine

Montreal, Quebec, Canada

Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust

London, United Kingdom

Key Dates

Start Date

December 12, 2023

Primary Completion Date

November 2, 2028

Completion Date

November 2, 2031

Last Updated

October 15, 2025

Enrollment

ACTUAL participants

Conditions

Rett Syndrome

Interventions

TSHA-102

GENETIC

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267

Rett Syndrome

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RECRUITINGEARLY_PHASE1

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

NCT06856759

Rett Syndrome

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RECRUITING

GROWing Up With Rare GENEtic Syndromes

NCT04463316

Prader-Willi Syndrome

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 15, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

GROWing Up With Rare GENEtic Syndromes

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome