ATB200/AT2221 for Pompe Disease
Recruiting at 61 trial locations
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Amicus Therapeutics
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
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.Inclusion Criteria
Subject must have completed Study ATB200-03.
Exclusion Criteria
Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
Subject, if female, is pregnant or breastfeeding.
Subject, whether male or female, is planning to conceive a child during the study.
Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Open-label Extension
Participants receive ATB200 co-administered with AT2221 capsule (Miglustat) until regulatory approval or marketing authorization
Up to approximately 4 years
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Treatment Details
Interventions
- AT2221
- ATB200
Trial Overview 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.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: ATB200/AT2221Experimental Treatment2 Interventions
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Amicus Therapeutics
Lead Sponsor
Trials
55
Recruited
2,700+
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