A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
← catalyst calendarNCT04848779 · readout in 70 d
Sponsored by Sanofi (industry) · SNY — their whole pipeline →.
Phase
—
Status
Active, not recruiting
Study type
Observational
Enrollment
16estimated
Sites
15
Countries
Belgium, France, Germany +6
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 | 2021-06-10 | actual | When the study began enrolling |
| Primary completion | 2026-10-28 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2026-10-28 | estimated | The whole study's end, after follow-up |
| First posted | 2021-04-19 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-24 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Glycogen Storage Disease Type II
Intervention
- Drug: Alglucosidase alfa GZ419829 — also filed as Myozyme
Primary outcome
what the primary-completion date above is the date OFProportion of participants alive and free of invasive ventilation at Week 52 of treatment
measured Week 52
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