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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b


NCTID NCT06217861 (View at clinicaltrials.gov)
Description
Indication Glutaric Acidemia Type I
Compound Name VGM-R02b
Sponsor Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type Industry
Status
Recruiting
Enrollment Count 12

Therapy Information


Target Gene/Variant GCDH
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracerebroventricular
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 Undisclosed dose
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2023-12-12
Completion Date 2026-08
Last Update 2024-05-17

Participation Criteria


Eligible Age <=6 Years
Standard Ages Child
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
Recent Updates Granted CTA approval by NMPA on 7/13/23

Resources/Links