Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Completed · Phase 2, Phase 3 · NCT02004691
Conditions: Sphingomyelin Lipidosis
At a glance
- Research payments reported, 2025
- $3,000
- U.S. investigators named
- 0
- Sites paid
- 0
- Planned or actual enrollment
- 36
About the trial
- Sponsor
- Genzyme, a Sanofi Company
- Tested
- placebo (saline) (drug), Olipudase alfa (drug)
- Dates
- Started 2015-12-18; completion 2023-10-19
- Results posted
- Yes
Primary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult participants with acid sphingomyelinase deficiency (ASMD) by assessing changes in: 1) spleen volume as measured by abdominal magnetic resonance imaging (MRI) (and, for the United States \[US\] only, in association with participant perception related to spleen volume as measured by splenomegaly-related score \[SRS\]); and 2) infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide (DLCO). Secondary Objectives…
From ClinicalTrials.gov, last updated 2024-11-07. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $3,000 in research payments for this study in 2025 (8 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
- $3,000
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.