Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Timeframe: From Baseline up to End of study (64 weeks)
Number of Participants With Infusion-Associated Reactions (IARs)
Timeframe: Within up to 24 hours after start of any infusion (during the treatment period i.e. from Baseline up to 64 weeks)
Number of Participants With Change in Physical Examination
Timeframe: Baseline, Week 52 (last complete assessment)
Number of Participants With Change in Neurological Examination
Timeframe: Baseline, Week 52 (last assessment)
Number of Participants With Abnormal Liver Function Laboratory Values at the End of Study
Timeframe: At End of Study (Week 64)
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Timeframe: From Baseline up to End of Study (64 weeks)
Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities
Timeframe: From Baseline up to End of Study (64 weeks)
Change From Baseline in Safety Biomarker Level: High Sensitivity C Reactive Protein (hsCRP) at Week 64
Timeframe: Baseline, Week 64 (pre-infusion)
Change From Baseline in Safety Biomarker: Ceramide Level at Week 64
Timeframe: Baseline, Week 64 (pre-infusion)
Change From Baseline in Safety Biomarker: Iron at Week 64
Timeframe: Baseline, Week 64 (pre-infusion)
Change From Baseline in Safety Biomarker: Cardiac Troponin I and Ferritin at Week 64
Timeframe: Baseline, Week 64 (pre-infusion)
Change From Baseline in Safety Biomarker: Interleukin (IL)-6 and IL-8 at Week 24
Timeframe: Baseline, Week 24 (pre-infusion, last assessment)
Change From Baseline in Safety Biomarker: Calcitonin at Week 64
Timeframe: Baseline, Week 64 (pre-infusion)
Doppler Echocardiogram: Absolute Change From Baseline in Left Ventricular Ejection Fraction at Week 52
Timeframe: Baseline, Week 52 (last assessment)
Number of Participants With Treatment-Emergent Antibody: Treatment-Induced/Treatment-Boosted Anti-drug Antibodies and Neutralizing Antibody (NAb)
Timeframe: From Baseline up to Week 64
Number of Participants With Abnormalities in Liver Ultrasound Doppler at Week 52
Timeframe: Week 52 (last assessment)