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


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.


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 faskari@med.umich.edu

Study coordinator: Jenna Penterics, (734) 764-4911 or jepenter@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 ongoing


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 faskari@med.umich.edu

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