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Glutaric Aciduria Type I

Tundra lists 1 Glutaric Aciduria Type I clinical trial. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.

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RECRUITING

NCT06217861

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

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.

Gender: All

Ages: Any - 6 Years

Updated: 2024-05-17

1 state

Glutaric Acidemia Type I

Glutaric Aciduria Type I