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Found 1 Glycogen Storage Disease (GSD) trials

A listing of Glycogen Storage Disease (GSD) medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD) (Baby-COMET)
All genders
Phase 3
Interventional
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period …