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ACTIVE NOT RECRUITING
NCT04537377
PHASE1/PHASE2

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

Sponsor: Vivet Therapeutics SAS

View on ClinicalTrials.gov

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

Interventions

GENETIC

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