Glycogen Storage Disease Type II Clinical Trial
Official title:
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)
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 (ETP) - 52 weeks; - Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years. - Treatment duration: Up to 4 years - Visit frequency: every other week and potentially every week
Study duration may be variable by country, including at least completion of the PAP and ETP, and up to 4.08 years. ;
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