A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
← catalyst calendarNCT04884815 · readout ≤ 955 d
Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.
Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Single · Treatment
Enrollment
82estimated
Sites
16
Countries
Portugal, Spain, United Kingdom +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 | 2021-09-27 | actual | When the study began enrolling |
| Primary completion | Mar 2029 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Mar 2034 | estimated | The whole study's end, after follow-up |
| First posted | 2021-05-13 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-15 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Wilson Disease
Interventions
- Genetic: UX701 — also filed as rivunatpagene miziparvovec
- Drug: Standard of Care (SOC)
Primary outcomes
what the primary-completion date above is the date OFStage 1: Incidence of Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Adverse Events of Special Interest (AESIs), Treatment-Related TEAEs, and Treatment-Related TESAEs
measured Up to Week 52
Stage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Change in Total Copper from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Change in Ceruloplasmin-bound Copper from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Change in Ceruloplasmin from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Change in Non-Ceruloplasmin-bound Copper (NCC) from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Change in Free Copper from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Change in Ceruloplasmin Activity from Baseline at Week 52
measured Baseline, Week 52
Stage 1: Percent Reduction in Standard of Care (SOC) Medication by Week 52
measured Week 52
Stage 1: Number of Participants Who Discontinue SOC Medication by Week 52
measured Week 52
Stage 1: Number of Consecutive Weeks off SOC Medication at Week 52
measured Week 52
Stage 2: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52, Evaluated for Superiority
measured Baseline, Week 52
Stage 2: Percent Reduction in SOC Medication by Week 52, Evaluated for Superiority
measured Week 52
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