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

NCTID	NCT06217861 (View at clinicaltrials.gov)
Description	Phase I, open-label, single-arm, single-dose, trial of VGM-R02b (gene replacement therapy) in patients with Glutaric Acidemia Type I (GA-I) who meet enrollment criteria and are genetically confirmed by GCDH gene mutation. 1 to 3 patients aged≤ 6 years at the time of screening will be enrolled in each dose group in the dose escalation part. In the dose expansion part, the sample size will be statistically calculated and adjusted according to the efficacy and safety data in the dose escalation part. (Show More)
Development Status	Active
Indication	Glutaric Acidemia Type I
Disease Ontology Term	DOID:0111254
Compound Name	VGM-R02b
Sponsor	Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	12
Results Posted	Not Available

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	none
Dose 1	Undisclosed dose
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Granted CTA approval by NMPA on 7/13/23