A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

About the trial

Sponsor

Sanofi (with Pulse Infoframe Ltd.)

Tested

Olipudase alfa (drug)

Dates

Started 2024-04-16; completion 2029-01-15

Results posted

Not yet

US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. The total overall study duration will be 5 years. The follow-up period will be a minimum of 1 year to a maximum of 3 years. The enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.

From ClinicalTrials.gov, last updated 2026-09-16. Full record, eligibility and contacts (opens in a new tab)

Industry funding

Sanofi US Services Inc. reported $26,123 in research payments for this study in 2025 (6 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.