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.
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).
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:
- Male or female, aged 18 years or older
- Have elevated or normal levels of free copper in blood
- Treated with chelation or zinc therapy for 24 months or less
- Asymptomatic or symptomatic with neurological, psychiatric, or hepatic or combined symptoms
- In otherwise general good health
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: email@example.com phone: (734) 232-4182 or (844) 334-9253