ACTIVE: TSHA-102 study for girls aged 2–3 years – USA

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Status: Active, not recruitingStudy: Safety and preliminary efficacy of TSHA-102 in younger paediatric participants
Registry number: NCT07480564
Country: United States
Participants: Females older than 2 and younger than 4 years

What is being studied?
This very small early-stage study is evaluating a one-time intrathecal dose of the investigational regulated MECP2 gene therapy TSHA-102 in four young children with Rett syndrome.

The registry currently shows the study as active but not recruiting.

Official study record: https://clinicaltrials.gov/study/NCT07480564

Information checked 3 September 2026. Early gene-therapy studies primarily assess safety; clinical benefit is uncertain. Check the registry and speak with a Rett specialist for future updates.