Gene therapy could free wilson disease patients from daily pills

NCT ID NCT04884815

Ongoing Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if it is safe and can help the body control copper levels, possibly allowing patients to take fewer or no daily medications. About 82 participants will receive either the gene therapy or standard care.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: UX701 (rivunatpagene miziparvovec) gene therapy
What this could lead to: If successful, this gene therapy could allow people with Wilson disease to reduce or stop taking daily copper-control medications, simplifying their treatment.
What could go wrong: This is an early-phase trial (Phase 1/2) with only 82 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or liver issues, and it may not work as hoped.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Wilson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

WILSON DISEASE

Contacts and locations

Locations

Centro Hospitalar Universitário Lisboa Norte

Lisbon, Lisbon District, 1649-035, Portugal
Centro Hospitalar Universitário de São João

Porto, Porto District, 4200-319, Portugal
Duke University Medical Center

Durham, North Carolina, 27710, United States
Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 08035, Spain
Indiana University

Indianapolis, Indiana, 46202, United States
Kings College NHS Foundation

London, Surrey, SE5 9RS, United Kingdom
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Northwestern University

Chicago, Illinois, 60611, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Stanford University

Redwood City, California, 94063, United States
University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States
University of California Davis

Sacramento, California, 95817-1348, United States
University of California Los Angeles

Los Angeles, California, 90095, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Utah

Salt Lake City, Utah, 84132, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37212-2700, United States

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