Replagal Enzyme Replacement Therapy for Children With Fabry Disease


Phase 2 Results

Summary of Purpose

Primary Objective(s): - To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease - To assess the effect of Replagal on heart rate variability in patients 7 to 17 years of age Secondary Objective(s): - To determine the pharmacokinetics of Replagal at baseline and after the initiation of enzyme...

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Trial Milestones

The following dates are available for this trial. Trial information last updated on 25 March 2014.

1 Jun 2004 5 Jun 2004 1 Jun 2011 1 Jun 2011 1 Mar 2014 1 Aug 2013
Start Date First Received 1st Completion Completion Verification Results

Trial Basics




Trial Design

  • Allocation: Non-Randomized
  • Masking: Open Label
  • Purpose: Treatment
  • Endpoint: Safety/Efficacy Study
  • Intervention: Parallel Assignment