NCT06152237

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Taysha Gene Therapies, Inc. · Industry
• Sex: Female
• Age: 5 Years – 8 Years
• Healthy volunteers: Not accepted

Summary

The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
GENETIC	TSHA-102	TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.

Timeline

• Start date: 2023-12-12
• Primary completion: 2028-11-02
• Completion: 2031-11-02
• First posted: 2023-11-30
• Last updated: 2025-10-15

Locations

5 sites across 3 countries: United States, Canada, United Kingdom

Regulatory

• FDA-regulated drug study