One-Time gene therapy aims to free Wilson's patients from daily pills
NCT ID NCT04537377
First seen Jan 31, 2026 · Last updated May 24, 2026 · Updated 15 times
Summary
This early-stage trial tests a single dose of gene therapy (VTX-801) in 4 adults with Wilson's disease, a genetic condition causing copper buildup. The goal is to see if it's safe and can control copper levels well enough to let patients stop their daily medications. Participants will be followed for up to 5 years.
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This is a summary of
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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.