Fabry Disease Clinical Trial
Official title:
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
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.
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.
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