Primary Objective:
To evaluate the safety and tolerability of olipudase alfa administered intravenously in
pediatric patients every 2 weeks for 52 weeks.
Secondary Objective:
To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory
efficacy of olipudase alfa administered intravenously in pediatric patients every 2 weeks for
52 weeks.
Primary Outcomes
Measure: Number of adverse events Time: From screening through Week 64
Measure: Clinically significant changes in laboratory parameters (complete blood count (CBC), clinical chemistry, and urinalysis) Time: From screening through Week 64
Measure: Clinically significant changes in physical examinations (vital signs, electrocardiogram (ECG), doppler echocardiography, and liver ultrasound doppler) Time: From screening through Week 64
Secondary Outcomes
Measure: Maximum concentration (Cmax) Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52
Measure: Area under the curve until the last measurable concentration (AUClast) Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52
Measure: Area under the curve extrapolated to infinity (AUC) Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52
Measure: Half-life (t1/2) Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52
Measure: Clearance (CL) Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52
Measure: Volume of distribution (Vss) Time: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52
Measure: Change in sphingomyelin levels Time: From Day 1 through Week 64
Measure: Change in sphingomyelin metabolite levels Time: From Day 1 through Week 64