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Wilson Disease

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Efficacy and safety study of WTX101 in adult Wilson disease patients



Patients are being recruited for a phase 2, multi-centre, open-label, study to evaluate the efficacy and safety of WTX101 administered for 24 weeks in newly diagnosed Wilson Disease patients.

Study description

The study drug, WTX101 (bis-choline tetrathiomolybdate) is a de-coppering agent that is being investigated for the treatment of Wilson disease. The aim of this study is to confirm that the dosing regimen planned for use in late-phase studies with WTX101 is safe and effective in de-coppering newly diagnosed Wilson disease patients. The study will be conducted at nine Wilson disease expert centers (four in the US and five in Europe).

Subject eligibility

If you are a newly diagnosed Wilson disease patient you may be eligible to join the trial that is currently underway, if you meet the following criteria:

Study design

The study lasts for 7 months. There are eight study visits to the study site during this 7-month period and eight additional study visits with a potential for a nurse to visit you at home to reduce the amount of travel for you. You may also qualify for an optional extension study for an additional 12 months.

Primary investigator: Fred Askari, MD, PhD

Co-investigator: Matthew Lorincz, MD, PhD

Study coordinator: Elizabeth Kovan, email: phone: (734) 232-4182 or (844) 334-9253