Gene therapy breakthrough aims to control copper in wilson disease
NCT ID NCT04884815
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 32 times
Summary
This study tests a new gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if a single dose can help the body control copper levels safely, possibly reducing the need for daily medications. About 82 participants will be monitored for side effects and changes in copper regulation over a year.
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This is a summary of
the original study
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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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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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Gordon and Leslie Diamond Health Care Centre
Vancouver, British Columbia, V5Z 1M9, Canada
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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 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
Conditions
Explore the condition pages connected to this study.