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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

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Trial Profile

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: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 02 Jun 2025

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At a glance

  • Drugs TSHA 102 (Primary)
  • Indications Rett syndrome
  • Focus Adverse reactions
  • Acronyms REVEAL; REVEAL Pediatric Study
  • Sponsors Taysha Gene Therapies

Most Recent Events

  • 28 May 2025 According to a Taysha Gene Therapies media release, company will host three oral presentations related to TSHA-102 at the upcoming 2025 IRSF Rett Syndrome Scientific Meeting taking place in Boston from June 9-11, 2025.
  • 28 May 2025 Results presented in the Taysha Gene Therapies media release.
  • 28 May 2025 According to a Taysha Gene Therapies media release, companyannounced details of its planned pivotal Part B trial design for TSHA-102 following written alignment from the U.S. Food and Drug Administration (FDA).

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