HomeTrialNCT04808505
🔬NCT04808505Phase 3RECRUITING

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

Study Summary

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.

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment36 participants
Primary Completion
Lead SponsorAmicus Therapeutics
Data Retrieved2026-04-11 04:14:12.640544+00:00
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