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Clinical Trials in the USA / NCT06152237
Active, not recruiting Phase 1/2

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

NCT06152237 · tracked via the Priya Life Science USA tracker
Sponsor
Taysha Gene Therapies, Inc.
Phase
Phase 1/2
Started
2023-12-12
Last updated
2025-10-15

Condition(s) studied

Rett Syndrome

Investigational drug(s) / intervention(s)

TSHA-102

TSHA-102: TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.

Study 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

Sex
FEMALE
Min age
5 Years
Max age
8 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (5)

FacilityCityRegionStatus
Rush University Medical Center & Children's Hospital Chicago Illinois
Gillette Children's Specialty Healthcare Saint Paul Minnesota
Washington University, St. Louis St Louis Missouri
CHU Ste-Justine Montreal Quebec
Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust London United Kingdom

More Taysha Gene Therapies, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06152237 on ClinicalTrials.gov ↗ ← All trials in the USA