A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Wilson Disease
In brief
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.
Key facts
- Study ID
- NCT04884815
- Run by
- Ultragenyx Pharmaceutical Inc
- People needed
- 82
- Starts
- 2021-09-27
- Expected to finish
- 2034-03-01
- Last updated by the study team
- 2026-06-15
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California Los Angeles — Los Angeles, California, United States
- Stanford University — Redwood City, California, United States
- University of California Davis — Sacramento, California, United States
- Northwestern University — Chicago, Illinois, United States
- Indiana University — Indianapolis, Indiana, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- University of Michigan — Ann Arbor, Michigan, United States
- Duke University Medical Center — Durham, North Carolina, United States
- University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
- Vanderbilt University Medical Center — Nashville, Tennessee, United States
- University of Utah — Salt Lake City, Utah, United States
- Seattle Children's Hospital — Seattle, Washington, United States
- Centro Hospitalar Universitário Lisboa Norte — Lisbon, Lisbon District, Portugal
- Centro Hospitalar Universitário de São João — Porto, Porto District, Portugal
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona, Spain
- Kings College NHS Foundation — London, Surrey, United Kingdom
Full record on ClinicalTrials.gov
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