Gene therapy trial aims to free patients from lifelong medication for rare copper disorder
NCT ID NCT04537377
Summary
This early-stage study is testing a single dose of an experimental gene therapy called VTX-801 in adults with Wilson's Disease. The main goal is to see if the treatment is safe and if it can help the body manage copper levels, potentially reducing the need for lifelong standard medications. The study will follow a small group of participants for up to five years to monitor their health and copper levels.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for WILSON'S DISEASE are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Aarhus University Hospital
Aarhus, 8200, Denmark
-
Advent Health
Orlando, Florida, 32803, United States
-
Royal Surrey County Hospital
Guildford, Surrey, GU2 7XX, United Kingdom
-
UC Davis Medical Center
Sacramento, California, 95817, United States
-
University Hospital Essen
Essen, 45147, Germany
-
University of Michigan Health System
Ann Arbor, Michigan, 48109, United States
-
University of Texas Southwestern Medical Center
Dallas, Texas, 75235, United States
-
Universitätsklinikum Tübingen (UKT)
Tübingen, 72076, Germany
-
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157, United States
-
Yale University School of Medecine
New Haven, Connecticut, 06510, United States
Conditions
Explore the condition pages connected to this study.