The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa. The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.
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Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs)
Timeframe: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With IARs
Timeframe: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first
For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam
Timeframe: Baseline (Day 1 of original study), Month 78
For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam
Timeframe: Baseline (Day 1 of original study), Month 84
For Adults: Number of Participants With Abnormality in Extended Neurological Examinations
Timeframe: Baseline (Day 1 of original study), Month 78
For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations
Timeframe: Baseline (Day 1 of original study), Month 84
Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Vital Signs
Timeframe: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Clinical Chemistry Parameters
Timeframe: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Liver Function Tests
Timeframe: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Hematology Parameters
Timeframe: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Electrocardiogram (ECG)
Timeframe: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
For Adults: Percent Change From Baseline in Pre-Infusion Plasma Ceramide at Month 90
Timeframe: Baseline (Day 1 of original study) and Month 90 (pre-infusion)
For Pediatrics: Percent Change From Baseline in Pre-Infusion Plasma Ceramide at Month 66
Timeframe: Baseline (Day 1 of original study) and Month 66 (pre-infusion)
Participants From Adult Study DFI13412: Number of Participants With Abnormal Liver Biopsy
Timeframe: Baseline (Day 1 of original study) and Month 36
Participants From Pediatric Study DFI13803: Number of Participants With Abnormalities in Liver Ultrasound Doppler
Timeframe: Baseline (Day 1 of original study), Week 2 and Months 12, 18 and 24
Number of Participants With Anti-Drug Antibodies (ADA) Against Olipudase Alfa
Timeframe: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first