One-Shot gene therapy tested for rare copper disease
NCT ID NCT04537377
Summary
This is an early-stage study testing a single dose of an experimental gene therapy called VTX-801 in adults with Wilson's Disease. The main goal is to check if the treatment is safe and to see how it affects the body's copper levels over five years. Researchers will give the therapy by IV and then carefully monitor participants, including trying to reduce their standard medications.
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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.