Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | |
| Rush University Medical Center | Chicago | Illinois | |
| Boston Children's Hospital | Boston | Massachusetts |
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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