At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 20 enrolled
Drug / intervention
Olipudase alfadrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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 Aged <18 Years With Acid Sphingomyelinase Deficiency
In Brief
A Phase 2 clinical trial evaluating Olipudase alfa for Sphingomyelin Lipidosis. Completed, enrolled 20 participants across 6 sites in 6 countries.
Detailed Summary
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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSphingomyelin Lipidosis
CountriesBrazil, France, Germany, Italy, United Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedNov 2014
Enrollment StartMay 2015
Primary CompletionDec 2019
TodayJul 2026
First PostedNov 17, 2014
Enrollment StartMay 1, 2015
Primary CompletionDec 9, 2019
TodayJul 2, 2026
Enrollment to primary: 4.6 yearsPosted 11.6 years ago
Interventions
Olipudase alfadrug
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusion