Gene therapy hopes to tame rare copper disorder
NCT ID NCT04537377
First seen Jan 31, 2026 · Last updated Apr 25, 2026 · Updated 12 times
Summary
This study tests a new gene therapy called VTX-801 for adults with Wilson's disease, a rare condition where copper builds up in the body. Four participants receive a single dose to see if it is safe and can help control copper levels, possibly allowing them to stop other treatments. The trial lasts up to 5 years and is in early phases (1 and 2).
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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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Aarhus University Hospital
Aarhus, 8200, Denmark
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Advent Health
Orlando, Florida, 32803, United States
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Royal Surrey County Hospital
Guildford, Surrey, GU2 7XX, United Kingdom
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UC Davis Medical Center
Sacramento, California, 95817, United States
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University Hospital Essen
Essen, 45147, Germany
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University of Michigan Health System
Ann Arbor, Michigan, 48109, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75235, United States
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Universitätsklinikum Tübingen (UKT)
Tübingen, 72076, Germany
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Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157, United States
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Yale University School of Medecine
New Haven, Connecticut, 06510, United States
Conditions
Explore the condition pages connected to this study.