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

StatusActive, Not Recruiting
PhaseEarly Phase 1
Started
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Record History

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  1. Primary completion date: Oct 23, 2029 to Oct 20, 2026
    • Trial status: Recruiting to Active, Not Recruiting
    • Site details revised
    v4
  2. Phase: Phase 1/2 to Early Phase 1
    • Eligibility criteria expanded, about 10 words
    • Secondary endpoints: details revised at 2 of 17 entries
    • Study description expanded, about 11 words
    v3
  3. Enrollment target: 6 to 8v2 Show 5 more changesShow less
    • Eligibility criteria expanded, about 41 words
    • Secondary endpoints: 16 to 17
    • Study description shortened, about 11 words
    • Site contact updated
    • Collaborators shortened, about 13 words
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Rett syndrome (RTT) is a serious neurodevelopmental disorder that has a significant impact on patients and their families. Patients suffer from severe social dysfunction and poor quality of life, and there is currently no effective treatment available. The MECP2 functional loss mutation is the clear pathogenic factor. In recent years, gene therapy has been applied in neuromuscular diseases such as SMA and has achieved good safety and effectiveness. Professor Qiu Zilong's self-developed AAV-MECP2 gene therapy product for RTT was found to significantly improve disease symptoms in RTT model mice, and demonstrated good safety in heath injection testing in monkeys. The dose exploration study of AAV-MECP2 initiated by our researchers is a multicenter, single arm, single intrathecal injection. The plan is to explore two target doses, with 5 subjects enrolled in dose 1 and 3 subjects enrolled in dose 2, to evaluate the safety, tolerability, and preliminary efficacy of single intrathecal injection of AAV-MECP2 in the treatment of RTT.
Trial Details
NCT Number NCT06856759
Lead Sponsor Guangzhou Women and Children's Medical Center
Conditions
(from sponsor)		 Rett Syndrome
Related terms Rett Syndrome
Enrollment 8 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov