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

> **NCT06856759** · EARLY_PHASE1 · RECRUITING · sponsor: **Guangzhou Women and Children's Medical Center** · enrollment: 8 (estimated)

## Conditions studied

- Rett Syndrome

## Interventions

- **GENETIC:** Intrathecal injection of AAV-MECP2 for the treatment of Rett syndrome

## Key facts

- **NCT ID:** NCT06856759
- **Lead sponsor:** Guangzhou Women and Children's Medical Center
- **Sponsor class:** OTHER
- **Phase:** EARLY_PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2025-01-14
- **Primary completion:** 2029-10-23
- **Final completion:** 2029-10-23
- **Target enrollment:** 8 (ESTIMATED)
- **Last updated:** 2025-07-01


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06856759

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06856759, "Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome". Retrieved via AI Analytics 2026-07-19 from https://api.ai-analytics.org/clinical/NCT06856759. Licensed CC0.

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