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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT06856759 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Rett Syndrome
Disease Ontology Term DOID:1206
Compound Name AAV-MECP2
Compound Description AAV9.CAG.MECP2
Sponsor Guangzhou Women and Children's Medical Center
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 6
Results Posted Not Available

Therapy Information


Target Gene/Variant MECP2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 7E14 vg
Dose 2 1.5E15 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-12-29
Completion Date 2029-10-23
Last Update 2025-03-04

Participation Criteria


Eligible Age 4 Years - 10 Years
Standard Ages Child
Sexes Eligible for Study FEMALE

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links