Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
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Phase
—
Status
Completed
Study type
Observational
Enrollment
12actual
Sites
6
Countries
Canada, Netherlands, Spain +1
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 | 2019-07-15 | actual | When the study began enrolling |
| Primary completion | 2025-02-25 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-02-25 | actual | The whole study's end, after follow-up |
| First posted | 2019-05-31 | actual | When this record first appeared on the registry |
| Results posted | 2026-03-18 | actual | When the sponsor posted results to the registry |
| Record updated | 2026-03-18 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Glycogen Storage Disease Type IA
- Von Gierke's Disease (GSD Type Ia)
Intervention
- Other: No intervention
Primary outcome
what the primary-completion date above is the date OFNumber of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs
measured From Baseline (Week 52 of 401GSDIA01/Visit 1 of 401GSDIA02) Up to Week 329
Posted documents
hosted by the registry — we link, never re-serveStudy Protocolposted 2023-04-03 · 775 KB · Prot_000.pdf
Statistical Analysis Planposted 2024-08-26 · 1.0 MB · SAP_001.pdf
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