NCT04138277

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease (LOPD)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 119 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

Detailed description

This is an open-label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

Conditions

• Pompe Disease (Late-onset)

Interventions

Type	Name	Description
DRUG	AT2221	Participants received ATB200 co-administered with AT2221 (miglustat)
BIOLOGICAL	ATB200	Enzyme Replacement Therapy via intravenous infusion

Timeline

• Start date: 2019-12-18
• Primary completion: 2024-12-31
• Completion: 2024-12-31
• First posted: 2019-10-24
• Last updated: 2026-03-05
• Results posted: 2026-01-12

Locations

60 sites across 23 countries: United States, Argentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Canada, Denmark, France, Germany, Greece, Hungary, Italy, Japan, Netherlands, New Zealand, Poland, Slovenia, South Korea, Spain, Sweden, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study