ACTIVE: AAV-MECP2 gene-therapy study – China
Posted: Thu Sep 03, 2026 10:48 pm
Status: Active, not recruitingStudy: Single-dose AAV-MECP2 safety, tolerability and efficacy study
Registry number: NCT06856759
Location: Guangzhou Women and Children’s Medical Center, Guangzhou, China
Participants: Females aged 4–10 years
What is being studied?
This small early-stage study is assessing the safety, tolerability and preliminary effects of a single-dose AAV-based MECP2 gene therapy in eight girls with Rett syndrome. Participants are followed for safety and clinical outcomes.
The registry lists the study as active but not currently recruiting.
Official study record: https://clinicaltrials.gov/study/NCT06856759
Information checked 3 September 2026. Gene therapy remains experimental and can involve serious or unknown risks. The official registry and treating Rett specialist should be consulted for updates.
Registry number: NCT06856759
Location: Guangzhou Women and Children’s Medical Center, Guangzhou, China
Participants: Females aged 4–10 years
What is being studied?
This small early-stage study is assessing the safety, tolerability and preliminary effects of a single-dose AAV-based MECP2 gene therapy in eight girls with Rett syndrome. Participants are followed for safety and clinical outcomes.
The registry lists the study as active but not currently recruiting.
Official study record: https://clinicaltrials.gov/study/NCT06856759
Information checked 3 September 2026. Gene therapy remains experimental and can involve serious or unknown risks. The official registry and treating Rett specialist should be consulted for updates.