Glycogen Storage Disease (GSD) Clinical 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.
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)
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 …
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- 31 Jan, 2023
- 3 locations