Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Status:
Completed
Trial end date:
2011-06-15
Target enrollment:
Participant gender:
Summary
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 replacement therapy (ERT)
- To determine exploratory measurements of efficacy including renal function (ie,
estimated glomerular filtration rate [eGFR] and creatinine clearance), clinical outcomes
(in Cohorts 1 and 2), and sweating and left ventricular mass index (LVMI) (Cohort 1,
Phase 1 only)