At a glance
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A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
In Brief
A Phase 3 clinical trial evaluating Cipaglucosidase Alfa, Miglustat, and 2 other interventions for Pompe Disease (Late-onset). Completed, enrolled 125 participants across 73 sites in 24 countries.
Detailed Summary
This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.
Study Details
Timeline
Interventions
Participants received an intravenous (IV) infusion dose over a 4-hour duration every 2 weeks (Q2W).
Participants received weight-based doses 1 hour prior to cipaglucosidase alfa infusion Q2W.
Participants received an IV infusion dose over a 4-hour duration Q2W.
Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W.