Amicus ATB200-03 POMPE CDA

Brief description of study

This is a double-blind, randomized, multicenter, international switch study where adult subjects with LOPD who have been in the prospective, non-interventional Study POM-003 (receiving alglucosidase alfa) will be randomized to ATB200/AT2221 or alglucosidase alfa/placebo oral capsule. The primary measure of efficacy is the comparison of the change in clinical parameters such as endurance (primary endpoint), motor performance, pulmonary function, and patient-reported outcomes (secondary endpoints) in individual subjects who will switch from alglucosidase alfa in Study POM-003 to the ATB200/AT2221 arm in this randomized study. The primary purpose of the alglucosidase alfa/placebo oral capsule arm in this study is to ensure the double-blind aspect of the study.


Clinical Study Identifier: s18-01115
ClinicalTrials.gov Identifier: NCT03729362
Principal Investigator: Heather Ann Lau
Currently Recruiting

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