Status: Recruiting
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
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.
Participants needed: 36
Trial details
Phase: Phase 3Age: Up to 17Biological sex: AllType: InterventionalSponsor: Amicus TherapeuticsUpdated: Jan 22, 2026Locations: 14
Eligibility criteria
Male or female subjects who are aged 6 months to < 18 years on Day 1 [+9]
Subject requires invasive ventilation (eg, tracheostomy) [+4]