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)
← catalyst calendarNCT06192576 · readout in 867 d
Sponsored by Sanofi (industry) · SNY — their whole pipeline →. With Pulse Infoframe Ltd..
Phase
—
Status
Recruiting
Study type
Observational
Enrollment
10estimated
Sites
5
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-04-16 | actual | When the study began enrolling |
| Primary completion | 2029-01-02 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2029-01-15 | estimated | The whole study's end, after follow-up |
| First posted | 2024-01-05 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-10-31 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Niemann-Pick Diseases
- Acid Sphingomyelinase Deficiency
Intervention
- Drug: Olipudase alfa — also filed as Xenpozyme®
Primary outcomes
what the primary-completion date above is the date OFNumber of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions)
measured Baseline to 3 years
Presence and titers of serum anti-olipudase alfa IgG anti-drug antibodies (ADA) (and IgE ADA in cases of moderate/severe or recurrent reactions)
measured Baseline to 3 years
Number of cases in the exposed population with abnormal laboratory test results and vital signs
measured Baseline to 3 years
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