NCT05606614

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

An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Taysha Gene Therapies, Inc. · Industry
• Sex: Female
• Age: 6 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

REVEAL Part A (Phase 1/2) is an open-label safety and dose-finding study designed to evaluate the safety and preliminary efficacy of two dose levels of TSHA-102 to establish initial safety of TSHA-102 and select a safe and efficacious dose for further evaluation. Enrollment of 6 participants in Part A is complete. REVEAL Part B (Phase 3) will evaluate the efficacy and safety of TSHA-102 at the dose level 2 determined in Part A in 15 females ages 6 to \<22 years with typical Rett syndrome. TSHA-102 is designed to target the genetic root cause of Rett syndrome by regulating the expression of MECP2 in cells. Each participant will be followed for the observation period of 5 years after TSHA-102 administration in Part A and B.

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: 2023-03-06
• Primary completion: 2031-06-01
• Completion: 2031-06-01
• First posted: 2022-11-07
• Last updated: 2025-12-30

Locations

6 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study