NCT01458119

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

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 85 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Study AT1001-041 was an open-label, noncomparative, multicenter, long-term extension study for participants with Fabry disease who completed treatment in one of three previous trials of migalastat (AT1001-011 \[NCT00925301\], AT1001-012 \[NCT01218659\], or FAB-CL-205 \[NCT00526071\]). In these trials, migalastat was given as monotherapy. This was an extension study designed to evaluate the long-term safety and efficacy of migalastat for the treatment of Fabry disease. Study visits occurred every 6 months (m). Visit evaluations included physical examinations, clinical laboratory parameters, adverse events, and participant reported outcomes. The study consisted of a Baseline Visit, which was performed at the time of the final visit of the previous study, followed by clinic visits every 6 m for each year of the study. Study assessments included a physical examination, echocardiography, laboratory parameters, and participant-reported outcomes. Since participants enrolled in the study at varying time points based on the completion of the preceding migalastat study, treatment duration varied among participants. No maximum treatment duration was defined. There were no control groups in this study; all participants received migalastat as a 150-mg capsule taken orally once every other day (QOD) and inactive reminder capsules on alternate days. The sponsor (Amicus Therapeutics) discontinued Study AT1001-041 for logistical reasons and not due to either safety concerns or lack of efficacy. For participants who were ongoing in Study AT1001-041 at the time of discontinuation, the investigators were offered participation in a similar open-label, long-term migalastat treatment study (AT1001-042 \[NCT02194985\]) for participants ongoing at discontinuation.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat hydrochloride	Oral capsule QOD

Timeline

• Start date: 2011-10-14
• Primary completion: 2016-02-17
• Completion: 2016-02-17
• First posted: 2011-10-24
• Last updated: 2018-10-02
• Results posted: 2018-10-02

Locations

25 sites across 13 countries: United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Spain, Turkey (Türkiye), United Kingdom