Health Trials

Clinical trials

2

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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.

Participants needed: 3
Trial details
Phase: Phase 3Age: 2-3Biological sex: FemaleType: InterventionalSponsor: Taysha Gene Therapies, Inc.Updated: May 22, 2026Locations: 1
Eligibility criteria

Pediatric females between the ages of 2 and less than 4 years old. [+3]

Participant has another neurodevelopmental disorder independent of the MECP2 los... [+3]

Read more
Status: Recruiting

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

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.

Participants needed: 15
Trial details
Phase: Phase 3Age: 6-21Biological sex: FemaleType: InterventionalSponsor: Taysha Gene Therapies, Inc.Updated: Dec 30, 2025Locations: 6
Eligibility criteria

Females between the ages of 12 and <22 in Part A (closed) and females between th... [+3]

Participant has another neurodevelopmental disorder independent of the MECP2 los... [+3]

Read more