drugset
drugset / Trial / NCT06152237

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

NCT06152237 ↗

Phase 1/2 Active not recruiting 6 enrolled Taysha Gene Therapies, Inc.
RandomizedSequentialOpen-labelTreatment

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.

Timeline

Start
2023-12-12
Primary completion
2028-11-02
Completion
2031-11-02

Drugs

EvaluationDrugModalityDoseRoute
Subject LONIMECGENE RENPARVOVEC Gene therapy (AAV / viral vector) — Intrathecal

Indications

Rett syndrome