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

Recruiting Phase 1 Interventional Study
Glutaric Acidemia Type I Glutaric Aciduria Type I
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
Up to 6
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
12 (estimated)
Sponsor
Shanghai Vitalgen BioPharma Co., Ltd. · Industry
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About This Trial
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 statis…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Subjects must be ≤ 6 years; 2. History of diagnosis of GA-I, and confirmed by gene mutation analysis with biallelic GCDH mutation; 3. At the time of screening, there was one of the obvious neurological manifestations associated with the following diseases, including macroceph…
Contacts

Xianfeng Wang

18816250806

xianfeng.wang@vitalgen.com

CONTACT