A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease (NCT04884815)
An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults With Wilson Disease
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California Los Angeles — Los Angeles, California
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (16 Sites)
- University of California Los Angeles — Los Angeles, California
- Stanford University — Redwood City, California
- University of California Davis — Sacramento, California
- Northwestern University — Chicago, Illinois
- Indiana University — Indianapolis, Indiana
- Massachusetts General Hospital — Boston, Massachusetts
- University of Michigan — Ann Arbor, Michigan
- Duke University Medical Center — Durham, North Carolina
- University Hospitals Cleveland Medical Center — Cleveland, Ohio
- Vanderbilt University Medical Center — Nashville, Tennessee
- University of Utah — Salt Lake City, Utah
- Seattle Children's Hospital — Seattle, Washington
- Centro Hospitalar Universitário Lisboa Norte — Lisbon, Lisbon District
- Centro Hospitalar Universitário de São João — Porto, Porto District
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona,
- Kings College NHS Foundation — London, Surrey
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Wilson Disease
- Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial — Los Angeles, California
- Natural History of Wilson Disease — New Haven, Connecticut
- Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study — New Haven, Connecticut
Explore by condition: All Wilson Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.