Clinical Trial

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

Recruiting Phase 1
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Summary
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.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2024-01-11; most recent amendment 2026-05-09.
Status change: Not Yet Recruiting → Recruiting 2024-05-17
Trial Details
NCT Number NCT06217861
Lead Sponsor Shanghai Vitalgen BioPharma Co., Ltd.
Conditions Glutaric Acidemia Type I, Glutaric Aciduria Type I
Enrollment 12 participants
Start Date 2024-04-29
Primary Completion 2027-08 (estimated)
Study Completion 2027-08 (estimated)
Updated on ClinicalTrials.gov 2026-05-13