An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease


Phase 1 Results N/A

Summary of Purpose

This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or fatty substance called ceramidetrihexoside, a building block in all cells of the body. The deficiency in breaking...

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Trial Milestones

The following dates are available for this trial. Trial information last updated on 16 August 2011.

1 Oct 2003 26 Jul 2006 1 Dec 2008 Unavailable 1 Dec 2008 Unavailable
Start Date First Received 1st Completion Completion Verification Results

Trial Design

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


Not available