One-Shot gene therapy aims to free patients from lifelong Copper-Control drugs
NCT ID NCT04884815
Summary
This study is testing a single-dose gene therapy called UX701 for adults with Wilson Disease, a condition where the body cannot properly process copper. The main goals are to see if the treatment is safe and if it can help the body regulate copper on its own, which could reduce or eliminate the need for daily medications. The study will follow participants for at least five years to check for long-term safety and benefits.
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.