~6 spots leftby Apr 2026

Open-Label Phase 3 Long-Term Safety Study of Migalastat

(AT1001-041 Trial)

Recruiting in Palo Alto (17 mi)
+24 other locations
Age: Any Age
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

Trial Summary

What is the purpose of this trial?

This was a long-term, open-label study of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) in participants with Fabry disease who completed treatment in a previous monotherapy trial with migalastat.

Research Team

MM

Medical Monitor, Clinical Research

Principal Investigator

Amicus Therapeutics

Eligibility Criteria

Inclusion Criteria

Completed migalastat treatment in a previous Fabry disease protocol
Both male and female participants were enrolled
Age 16 years or older
See 1 more

Treatment Details

Interventions

  • Migalastat Hydrochloride (Chaperone Therapy)
Participant Groups
1Treatment groups
Experimental Treatment
Group I: MigalastatExperimental Treatment1 Intervention
Migalastat 150-mg capsule taken orally QOD. The median duration of exposure was 23.5 m.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Amicus Therapeutics

Lead Sponsor

Trials
55
Recruited
2,700+