Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Completed · Phase 1, Phase 2 · NCT02292654
Conditions: Sphingomyelin Lipidosis
At a glance
- Research payments reported, 2025
- $2,500
- U.S. investigators named
- 0
- Sites paid
- 0
- Planned or actual enrollment
- 20
About the trial
- Sponsor
- Genzyme, a Sanofi Company
- Tested
- Olipudase alfa (drug)
- Dates
- Started 2015-05-01; completion 2019-12-09
- Results posted
- Yes
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.
From ClinicalTrials.gov, last updated 2022-03-23. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $2,500 in research payments for this study in 2025 (2 payments).
Products named: Xenpozyme
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
Sites paid to run it
- $2,500
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.