Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity


Phase N/A Results N/A

Summary of Purpose

This study will collect data needed to design a treatment trial for patients with Fabry disease using the experimental drug AT-1001. Fabry disease is an inherited metabolic disorder in which an enzyme called alpha-galactosidase A, which normally breaks down fatty substances called glycolipids, is missing or does not function properly. As a result, glycolipids accumulate in various tissues, causing liver, kidney,...

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Trial Milestones

The following dates are available for this trial. Trial information last updated on 30 June 2017.

28 Mar 2005 31 Mar 2005 Unavailable 27 Mar 2008 27 Mar 2008 Unavailable
Start Date First Received 1st Completion Completion Verification Results

Trial Design

Not available


Not available