Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Ag
Brief Summary:
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.