A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Wilson's Disease

In brief

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.

Key facts

Study ID

NCT04537377

Run by

Vivet Therapeutics SAS

People needed

4

Starts

2021-09-03

Expected to finish

2029-06-18

Last updated by the study team

2026-01-30

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

Where it is running

• 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

Full record on ClinicalTrials.gov

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