Clinical Trial

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.
Protocol Amendment History 3 changes
critical Trial status changed: Recruiting → Active, Not Recruiting 2026-07-08
notable Enrollment increased: 15 -> 17 participants 2026-07-08
notable Trial sites expanded: 6 -> 9 locations 2026-07-08
Trial Details
NCT Number NCT05606614
Lead Sponsor Taysha Gene Therapies, Inc.
Conditions Rett Syndrome
Enrollment 17 participants
Start Date 2023-03-06
Primary Completion 2031-06 (estimated)
Study Completion 2031-06 (estimated)
Updated on ClinicalTrials.gov 2026-07-07