A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

• Status: Active, not recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics
• Treatments: Miglustat

Criteria

Inclusion Criteria:

1. Subject must have completed Study ATB200-03.

Exclusion Criteria

1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.

2. Subject, if female, is pregnant or breastfeeding.

3. Subject, whether male or female, is planning to conceive a child during the study.