NCT04537377

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

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Vivet Therapeutics SAS · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Wilson's Disease

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2021-09-03
• Primary completion: 2024-12-17
• Completion: 2029-06-18
• First posted: 2020-09-03
• Last updated: 2026-01-30
• Results posted: 2026-01-28

Locations

10 sites across 4 countries: United States, Denmark, Germany, United Kingdom

Regulatory

• FDA-regulated drug study