Treatment of Pediatric Late Onset Pompe Disease

This multicenter study will evaluate the safety and efficacy of cipaglucosidase alfa/miglustat treatment in pediatric subjects with late-onset Pompe disease, aged 0 to less than 18 years.

Cipaglucosidase alfa will be administered via IV infusion, and Miglustat will be supplied as capsules to be administered orally or used in the preparation of a solution for nasal or oral administration every other week. Both cipaglucosidase alfa and miglustat will be provided by Amicus Therapeutics, the sponsor of this study, at no cost to participants.

The starting dose regimens of cipaglucosidase alfa/miglustat will be based on age and weight. Cipaglucosidase alfa dose will either remain the same or may increase during the study. Subjects are expected to gain weight as they grow, so miglustat dose adjustments will be based on changes in weight category.