Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
← catalyst calendarNCT06636383 · readout ≤ 3,787 d
Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.
Phase
—
Status
Recruiting
Study type
Observational
Enrollment
140estimated
Sites
19
Countries
Brazil, Canada, Denmark +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 | 2024-11-04 | actual | When the study began enrolling |
| Primary completion | Dec 2036 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Dec 2036 | estimated | The whole study's end, after follow-up |
| First posted | 2024-10-10 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-06 | 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 Ia
Intervention
- Other: No Intervention
Primary outcomes
what the primary-completion date above is the date OFIncidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator
measured 10 Years
Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies
measured 10 Years
Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
measured 10 Years
Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner
measured 10 Years
Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)
measured 1 Year
Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)
measured 1 Year
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