Clinical Trial

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

Study acronym: ASPIRE
Recruiting Phase 3
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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.
Protocol Amendment History 1 change
critical Recruitment opened 2026-05-23
Trial Details
NCT Number NCT07480564
Lead Sponsor Taysha Gene Therapies, Inc.
Conditions Rett Syndrome
Enrollment 3 participants
Start Date 2026-05-08
Primary Completion 2031-06 (estimated)
Study Completion 2031-06 (estimated)
Updated on ClinicalTrials.gov 2026-05-22