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RECRUITINGPhase 3

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

NCT04808505

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.

Glycogen Storage Disease Type II Infantile Onset

Amicus Therapeutics36 participantsStarted Jul 2023

Gainesville, Florida, United States • Atlanta, Georgia, United States • Durham, North Carolina, United States +11 more locations

Data from ClinicalTrials.govTerms