NCT00925301

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 67 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 16 Years – 74 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

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.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat hydrochloride	Oral capsule QOD
DRUG	Placebo	Oral capsule QOD

Timeline

• Start date: 2009-10-23
• Primary completion: 2012-06-12
• Completion: 2014-01-29
• First posted: 2009-06-22
• Last updated: 2018-10-30
• Results posted: 2018-10-30

Locations

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