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, but until avalglucosidase alfa is approved in the patient's country or up to 4.08 years, whichever comes first. ;
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