Clinical Trial

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Active, Not Recruiting Phase 1/2
View on ClinicalTrials.gov →
Summary
The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the effect of UX701 on copper regulation.
Protocol Amendment History 55 amendments
This ClinicalTrials.gov record has been amended 55 times since 2021-05-07; most recent amendment 2026-06-11.
Status change: Recruiting → Active, Not Recruiting 2025-09-15
Status change: Active, Not Recruiting → Recruiting 2025-05-06
Status change: Recruiting → Active, Not Recruiting 2024-01-29
Status change: Not Yet Recruiting → Recruiting 2021-08-16
Trial Details
NCT Number NCT04884815
Lead Sponsor Ultragenyx Pharmaceutical Inc
Conditions Wilson Disease
Enrollment 82 participants
Start Date 2021-09-27
Primary Completion 2029-03 (estimated)
Study Completion 2034-03 (estimated)
Updated on ClinicalTrials.gov 2026-06-15