A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (NCT04537377)
A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease
🩺 Plain-English Summary (8th-Grade Level)
The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.
Who can join: Review the eligibility criteria below.
Location: UC Davis Medical Center — Sacramento, California
Age range: 18 Years to 65 Years
🧪 Interventions in This Study
- VTX-801 (GENETIC)
📋 "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 (10 Sites)
- UC Davis Medical Center — Sacramento, California
- Yale University School of Medecine — New Haven, Connecticut
- Advent Health — Orlando, Florida
- University of Michigan Health System — Ann Arbor, Michigan
- Wake Forest School of Medicine — Winston-Salem, North Carolina
- University of Texas Southwestern Medical Center — Dallas, Texas
- Aarhus University Hospital — Aarhus,
- University Hospital Essen — Essen,
- Universitätsklinikum Tübingen (UKT) — Tübingen,
- Royal Surrey County Hospital — Guildford, 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.
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💡 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.