Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome — Stella

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Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome — Stella

Recruiting

Back to Rett Syndrome trials

Early Phase 1 — Very small initial tests in humans, typically just a few people, to make sure the treatment is safe enough to study further.

Trial locations(1 site)

China

Guangzhou Women and Children's Medical Center, Guangzhou, Guangdong

Key details

Phase

Very early testing

Sponsor

Guangzhou Women and Children's Medical Center

Enrollment target

~8 participants

Started

January 2025

Primary completion

October 2029

Age range

4 Years – 10 Years

Sex

Female only

Last updated July 2025

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