First-in-human Study of UX016 in GNEM
← catalyst calendarNCT07511556 · readout ≤ 865 d
Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.
Phase
Phase 1/2
Status
Not yet recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
24estimated
Sites
2
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 | Oct 2026 | estimated | When the study began enrolling |
| Primary completion | Dec 2028 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Dec 2028 | estimated | The whole study's end, after follow-up |
| First posted | 2026-04-06 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-04-06 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- GNE Myopathy
Interventions
- Drug: UX016
- Other: Placebo
Primary outcomes
what the primary-completion date above is the date OFNumber of Participants with Treatment-Emergent Adverse Events (TEAEs)
measured Up to Week 104
Upper Extremity Composite (UEC) Score Change From Baseline
measured Baseline, 48 Weeks
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