Wilson Disease


A phase 3, randomized, rater-blinded, multicenter study to evaluate the efficacy and safety of WTX101 administered for 48 weeks versus standard of care in Wilson disease subjects aged 18 and older, with an extension phase of up to 60 months

Sponsor: Wilson Therapeutics AB

Enrollment status: OPEN

Purpose

The purpose of the clinical study is to evaluate the safety and effectiveness of an investigational medication (WTX101) compared to standard of care treatment in patients with Wilson disease. This medication is considered "investigational" because it has not been approved by the Food and Drug Administration (FDA) to treat Wilson Disease. WTX101 has the potential to address the unmet needs in Wilson Disease by possibly reducing the amount of copper buildup in the body.

Eligibility

You may be eligible to participate in the clinical study if you meet the following criteria:

Study design

If you are eligible and agree to participate in the study, you will be randomly assigned to either receive treatment with WTX101 or regular standard of care treatment. Once the treatment period is completed, study participants may choose to receive WTX101 for up to an additional 60 months.

Principal investigator: Fred Askari, MD, PhD

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

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