Status:
ACTIVE_NOT_RECRUITING
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
Lead Sponsor:
Taysha Gene Therapies, Inc.
Conditions:
Rett Syndrome
Eligibility:
FEMALE
5-8 years
Phase:
PHASE1
PHASE2
Brief 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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 12 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 2 2031
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT06152237
Start Date
December 12 2023
End Date
November 2 2031
Last Update
October 15 2025
Active Locations (5)
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1
Rush University Medical Center & Children's Hospital
Chicago, Illinois, United States, 60612
2
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
3
Washington University, St. Louis
St Louis, Missouri, United States, 63110
4
CHU Ste-Justine
Montreal, Quebec, Canada, H3T 1C5