Clinical Research Directory
Browse clinical research sites, groups, and studies.
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
Sponsor: Vivet Therapeutics SAS
Summary
The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.
Official title: A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
4
Start Date
2021-09-03
Completion Date
2029-06-18
Last Updated
2026-01-30
Healthy Volunteers
No
Conditions
Interventions
VTX-801
The investigational medicinal product (VTX-801) is a replication-deficient recombinant adeno-associated viral vector (rAAV) consisting of an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the ATP7B gene (ATP7B-minigene). After reconstitution VTX-801 will be administered as a single dose intravenous (IV) administration per patient, at up to 3 different dose levels.
Locations (10)
UC Davis Medical Center
Sacramento, California, United States
Yale University School of Medecine
New Haven, Connecticut, United States
Advent Health
Orlando, Florida, United States
University of Michigan Health System
Ann Arbor, Michigan, United States
Wake Forest School of Medicine
Winston-Salem, North Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Aarhus University Hospital
Aarhus, Denmark
University Hospital Essen
Essen, Germany
Universitätsklinikum Tübingen (UKT)
Tübingen, Germany
Royal Surrey County Hospital
Guildford, Surrey, United Kingdom