Long-term Extension of GTX-102 in Angelman Syndrome
← catalyst calendarNCT06415344 · readout ≤ 924 d
Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.
Phase
Phase 3
Status
Enrolling by invitation
Study type
Interventional
Design
Na · None · Treatment
Enrollment
255estimated
Sites
23
Countries
Australia, Canada, France +5
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 | 2024-07-31 | actual | When the study began enrolling |
| Primary completion | Feb 2029 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Feb 2029 | estimated | The whole study's end, after follow-up |
| First posted | 2024-05-16 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-27 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Angelman Syndrome
Intervention
- Drug: GTX-102 — also filed as apazunersen
Primary outcome
what the primary-completion date above is the date OFIncidence of Treatment Emergent Adverse Events (TEAEs)
measured Up to 5 Years
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