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First received: November 21, 2023

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

Study Status: Completed

Recruit Status: RECRUITING

Condition: Rett Syndrome

Study Type: INTERVENTIONAL


Official Title: 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 The

Brief 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.

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