A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Completed · Phase 2 · NCT02004704
Conditions: Sphingomyelin Lipidosis
At a glance
- Research payments reported, 2025
- $21,211
- U.S. investigators named
- 1
- Sites paid
- 1
- Planned or actual enrollment
- 25
About the trial
- Sponsor
- Genzyme, a Sanofi Company
- Tested
- GZ402665 (drug)
- Dates
- Started 2013-12-04; completion 2023-09-06
- Results posted
- Yes
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.
From ClinicalTrials.gov, last updated 2024-07-03. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $21,211 in research payments for this study in 2025 (9 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.
U.S. investigators named on this study
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Mount Sinai School of Medicine Psychiatry $16,211
- $5,000
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.