Wilson Disease


Efficacy and safety study of WTX101 in adult Wilson disease patients

Enrollment status: CLOSED, Follow-up continuing

Purpose

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.

Principal investigator: Fred Askari, MD, PhD

Co-investigator: Matthew Lorincz, MD, PhD

Study coordinator: Elizabeth Wu, (734) 764-4048 or elizwu@med.umich.edu

Multicenter study for the assessment of copper parameters in Wilson disease subjects on standard of care treatment

Enrollment status: CLOSED, Follow-up continuing

Objective

To evaluate plasma and urine copper parameters in Wilson Disease subjects treated with standard of care medications.

Subject eligibility

Study design

The study lasts 24 months. Data will be collected during routinely scheduled Wilson disease clinic visits at approximately 6-month intervals. In addition, study blood samples will be collected for analysis at 8 time points.

Principal investigator: Fred Askari, MD, PhD

Co-investigator: Matthew Lorincz, MD, PhD

Study coordinator: Elizabeth Wu, (734) 764-4048 or elizwu@med.umich.edu