Replagal Enzyme Replacement Therapy for Children With Fabry Disease

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)

Timeline

Start

2004-06-10

Primary completion

2011-06-15

Completion

2011-06-15

Publications

• Schiffmann R, Pastores GM, Lien YH, Castaneda V, Chang P, Martin R, Wijatyk A. Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study. Orphanet J Rare Dis. 2014 Nov 26;9:169. doi: 10.1186/s13023-014-0169-6.

Drugs

Indications