for females ages 5-8 (full criteria)
at La Jolla, California and other locations
study started
completion around



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.

Official Title

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


Rett Syndrome, Neurodevelopmental Disorder, MECP2, Syndrome, TSHA-102


You can join if…

Open to females ages 5-8

  • 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.

You CAN'T join if...

  • 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.


  • University of California San Diego (UCSD) not yet accepting patients
    La Jolla California 92037 United States
  • University of Texas Southwestern Medical Center (UTSW) not yet accepting patients
    Dallas Texas 75390 United States
  • Rush University Medical Center accepting new patients
    Chicago Illinois 60612 United States


accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
Taysha Gene Therapies, Inc.
Phase 1 Rett Syndrome Research Study
Study Type
Expecting 6 study participants
Last Updated