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

Plain-language summary

This study examines the safety and efficacy of a gene therapy called TSHA-102 in girls with classical Rett syndrome who carry a loss-of-function mutation in the MECP2 gene. Children aged 5 to 8 years with a confirmed diagnosis are evaluated. The study is being conducted in Phase 1/Phase 2; it is not currently recruiting but is ongoing (active, not recruiting).

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

Taysha Gene Therapies, Inc.

Intervention

TSHA-102

Start date

12/12/2023

Estimated completion

11/2/2031

Countries

United States, Canada, United Kingdom

Related genes

Locations

• Chicago, United States
• Saint Paul, United States
• St Louis, United States
• Montreal, Canada
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.