ACTIVE: TSHA-102 REVEAL pivotal gene-therapy study – Canada and USA

Status: Active, not recruitingStudy: REVEAL pivotal study of TSHA-102 gene therapy
Registry number: NCT05606614
Countries: Canada and United States
Participants: Females aged 6–21 years

What is being studied?
TSHA-102 is an investigational gene therapy designed to deliver a functional MECP2 gene using a regulated system intended to control MECP2 expression. This Phase 1/2/3 study is assessing safety and clinical effects after a one-time intrathecal dose.

The study remains active but is not currently recruiting according to the registry.

Official study record: https://clinicaltrials.gov/study/NCT05606614

Information checked 3 September 2026. Gene therapy carries important known and unknown risks, and benefit is not guaranteed. Contact the sponsor through the official record for updates and discuss any interest with a Rett specialist.