The GATEWAY trial is a multi-center, non-randomized, open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease, prior to and following background WD therapy withdrawal.
Six leading centers in the United States and Europe are expected to participate in the GATEWAY Phase 1/2 trial. The trial is expected to enroll up to sixteen adult patients with Wilson disease and will evaluate up to three doses of VTX-801. Patients will participate in a pre-dosing observational period and will be administered a prophylactic steroid regimen.
The primary endpoint of the GATEWAY trial is to assess the safety and tolerability of VTX-801 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including free serum copper and serum ceruloplasmin activity, as well as radiocopper-related parameters and VTX-801 responder status to allow standard-of-care withdrawal.
Vivet Therapeutics expects to enroll the first patient in early 2021.
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