Dosing Study of Replagal in Patients With Fabry Disease


Phase 2 Results

Summary of Purpose

This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb3). In patients with Fabry disease, Alpha-galactosidase A does not function properly and, therefore, Gb3 builds up, causing...

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Trial Milestones

The following dates are available for this trial. Trial information last updated on 19 March 2015.

1 Sep 2003 6 Sep 2003 1 Dec 2013 1 Dec 2013 1 Mar 2015 12 Mar 2015
Start Date First Received 1st Completion Completion Verification Results

Trial Design

  • Masking: Open Label
  • Purpose: Treatment
  • Endpoint: Safety/Efficacy Study
  • Intervention: Single Group Assignment


Not available