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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Rett Syndrome

In brief

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.

Key facts

Study ID

NCT06152237

Run by

Taysha Gene Therapies, Inc.

People needed

6

Starts

2023-12-12

Expected to finish

2031-11-02

Last updated by the study team

2025-10-15

Who can join

Age: 5 and older, up to 8. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.