Fabry Disease Clinical Trial
Official title:
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
This double-blind, randomized, placebo-controlled study was conducted in 67 participants at
46 sites worldwide. The study consisted of 2 stages and an optional open-label treatment
extension phase:
Stage 1 included a screening period of up to 2 months followed by a 6-month treatment period
which involved 4 visits to the clinic. Participants were randomized in equal proportions to
receive either migalastat or placebo.
After completing the 6-month double-blind phase, all participants entered Stage 2 of the
study and received migalastat in an open-label manner. Stage 2 treatment lasted for 6 months
and involved up to 4 visits to the clinic.
Participants who completed both Stage 1 and Stage 2 of the study as scheduled were offered
the opportunity to participate in an open-label treatment extension phase with migalastat.
The open-label treatment extension phase lasted 12 months and involved 2 visits to the
clinic. A follow-up visit was undertaken 1 month following completion or discontinuation from
the open-label treatment extension. Participants completing the 12-month open-label treatment
extension and providing consent to enter a separate long-term extension were not required to
complete this follow-up visit.
Study assessments included clinical laboratory tests, 12-lead electrocardiogram, kidney
biopsy, kidney function testing, echocardiography, and patient-reported outcomes.
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|---|---|---|---|
| Recruiting |
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Phase 2 | |
| Active, not recruiting |
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Phase 3 | |
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