Gene therapy could free wilson disease patients from daily pills
NCT ID NCT04884815
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Centro Hospitalar Universitário Lisboa Norte
Lisbon, Lisbon District, 1649-035, Portugal
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Centro Hospitalar Universitário de São João
Porto, Porto District, 4200-319, Portugal
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Hospital Universitario Vall d'Hebron - PPDS
Barcelona, 08035, Spain
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Indiana University
Indianapolis, Indiana, 46202, United States
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Kings College NHS Foundation
London, Surrey, SE5 9RS, United Kingdom
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Northwestern University
Chicago, Illinois, 60611, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Stanford University
Redwood City, California, 94063, United States
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University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
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University of California Davis
Sacramento, California, 95817-1348, United States
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University of California Los Angeles
Los Angeles, California, 90095, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Utah
Salt Lake City, Utah, 84132, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37212-2700, United States