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Active, not recruiting Phase 3

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

NCT07480564 · tracked via the Priya Life Science USA tracker
Phase
Phase 3
Started
2026-05-08
Last updated
2026-08-19

Condition(s) studied

Rett Syndrome

Investigational drug(s) / intervention(s)

TSHA-102

TSHA-102: TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.

Study summary

The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.

Eligibility

Sex
FEMALE
Min age
2 Years
Max age
3 Years
Healthy volunteers
No
Inclusion Criteria: * Pediatric females between the ages of 2 and less than 4 years old. * Participant has a clinical diagnosis of classic/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function. * Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed. * Participants and parent/caregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment. Exclusion Criteria: * Participant has another neurodevelopmental disorder independent of the MECP2 loss-of-function mutation, or any other genetic syndrome with a progressive course. * Participant has a history of brain injury that causes neurological problems or had grossly abnormal psychomotor development in the first 6 months of life. * Participant has a diagnosis of atypical Rett syndrome or a MECP2 gene mutation that does not cause Rett syndrome. * Participant requires invasive ventilatory support. Note: Other protocol defined inclusion/exclusion criteria may apply

Primary outcome measure(s)

  • Primary Safety — Baseline through Week 25
    Safety and Tolerability of TSHA-102 Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)

Trial sites (3)

FacilityCityRegionStatus
University of Alabama at Birmingham Birmingham Alabama
Rush University Medical Center Chicago Illinois
Boston Children's Hospital Boston Massachusetts
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 NCT07480564 on ClinicalTrials.gov ↗ ← All trials in the USA