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Clinical Trial Summary

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT03911505
Study type Interventional
Source Amicus Therapeutics
Contact For Site
Phone 215-921-7600
Email PompeSiteInfo@amicusrx.com
Status Recruiting
Phase Phase 3
Start date February 13, 2020
Completion date June 2026

See also
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Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT01288027 - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Phase 4
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Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Not yet recruiting NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) Early Phase 1
Completed NCT00268944 - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support Phase 3
Active, not recruiting NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD Phase 3