Phase 2 Study in Newly Diagnosed Wilson Disease Patients with WTX101 (Tetrathiomolybdate)… Now Recruiting! – ELPA – European Liver Patients Association


2015-10-22 10:49

Patients are being recruited for a Phase 2, multi-center, open-label, study to evaluate the efficacy and safety of WTX101 administered for 24 weeks in newly diagnosed Wilson Disease patients with an extension phase of 12 months. This study is being sponsored by Wilson Therapeutics.

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 future studies with WTX101 is safe and effective in de-coppering newly diagnosed Wilson Disease patients. The study will be conducted at 9 Wilson Disease expert centers (University of Michigan Hospital, Ann Arbor, MI; Yale University Medical Center, New Haven, CT; UCLA Ronald Reagan Medical Center, Los Angeles, CA; Northwestern University, Chicago, IL; Medical University of Vienna, Vienna, Austria; University Hospital, Heidelberg, Germany; Institute of Psychiatry and Neurology, Warsaw, Poland, Queen Elizabeth Hospital, Birmingham, UK; Royal Surrey County Hospital, Surrey, UK).

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 blood free copper levels
  • Treated with chelation or zinc therapy for 28 days or less
  • Have hepatic or neurological symptoms or both
  • In otherwise general good health   

The study lasts for 7 months.  There are 8 study visits to the study site during this 7 month period and 8 additional study visits with a potential for a nurse to visit you at home to reduce the amount of travel for you. After completing the 6 months of the primary study, you may qualify to continue in the extension study for an additional 12 months.  If you choose to participate in the Extension Study, you will have 5 additional study visits to the study site during those 12 months. Wilson Therapeutics will pay for your travel expenses.  

For further information about this study, please go to or

To discuss possible participation at any of the United States study centers, please contact the University of Michigan Hospital Wilson Disease Clinic by calling +1-800-333-9013. Contact details for all the individual sites are listed in the clinical trials links above.