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Gene Therapy Trial Report

Summary

GCB-002 in Treatment of Patients With Rett Syndrome


NCTID NCT06739434 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Rett Syndrome
Disease Ontology Term DOID:1206
Compound Name GCB-002
Sponsor Genecombio Ltd.
Funder Type Other
Recruitment Status
Enrolling by invitation
Enrollment Count 6 (ESTIMATED)
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
Delivery System Viral transduction
Vector Type scAAV9
Dose 1 2 cohort dose escalation, undisclosed concentrations

Study Record Dates


Current Stage Na
Submit Date 2024-12-12
Completion Date 2030-12
Last Update 2025-03-11

Participation Criteria


Eligible Age 2 Years - 10 Years
Standard Ages Child
Sexes Eligible for Study FEMALE
Eligibility Criteria
Inclusion Criteria: 1. Age range from 2 to 10 years old, female; 2. The clinical diagnosis of the subject is RTT, and after genetic testing, it was found to be a pathogenic variant of the MECP2 gene; 3. The legal guardian is able to understand the requirements and procedures of the research plan, voluntarily participate, and sign an informed consent form. Exclusion Criteria: 1. Has participated in or is currently participating in other RTT drug clinical trials or other AAV gene therapy clinical studies; 2. The subject has a history of head injuries that can cause neurological disorders such as epilepsy, physical disabilities, etc; 3. The subject has MECP2 gene mutation but does not cause RTT; 4. Subjects with allergic constitution, including those allergic or hypersensitive to prednisolone, other glucocorticoids, their excipients, and local anesthetics; 5. The subjects had status epilepticus in the 3 months prior to enrollment; 6. Subjects require invasive or non-invasive ventilation support; 7. Serum anti AAV9 neutralizing antibody titer\>1:200; The researchers believe that it is not suitable to participate in this study.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.