ATB200/AT2221 for Pompe Disease

Recruiting in Palo Alto (17 mi)

+60 other locations

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Amicus Therapeutics

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

This trial is for adults with late-onset Pompe disease who finished Study ATB200-03. Participants can't join if they're pregnant, breastfeeding, planning to have a child during the study, or if they plan to get gene therapy or join another Pompe disease study.

Participant Groups

The trial is testing the long-term safety and effectiveness of two drugs, AT2221 and ATB200, in treating adult patients with late-onset Pompe disease. It's an international study where everyone gets the same treatment.

1Treatment groups

Experimental Treatment

Group I: ATB200/AT2221Experimental Treatment2 Interventions

Participants received ATB200 co-administered with AT2221 capsule (Miglustat)