Clinical Trials Spotlight - Wilson's Disease

Image depicting Wilson's disease with an inflamed liverFirst Patient Enrolled for ACD CTSU Trial at U-M

The Ambulatory & Chronic Disease (ACD) CTSU is happy to announce that the first patient at the University of Michigan has been enrolled in the pivotal Phase 3 FOCuS clinical trial evaluating WTX101, an investigational first-in-class copper-protein-binding agent with a unique mechanism of action for the treatment of Wilson's Disease.

Wilson's Disease is a rare genetic disorder of impaired copper transport and excretion and affects approximately one in every 30,000 people worldwide and approximately 10,000 patients in the United States. Wilson's Disease is caused by loss of function of the ATP7B copper-binding protein. This leads to copper overload in the liver, release of free copper into the blood, and damaging accumulation of copper in the brain and other organs. Untreated Wilson's Disease inevitably leads to various combinations and severity of hepatic, neurologic and psychiatric symptoms, and is ultimately fatal.

The Phase 3 FOCuS clinical trial is a randomized, controlled, rater-blinded, multi-center study assessing the efficacy and safety of WTX101 monotherapy administered once daily for 48 weeks, compared to standard of care in patients with Wilson's Disease aged 18 years and older.

"WTX101 has shown great promise and I am excited to be part of the team advancing it through the clinic," said Frederick K. Askari, MD, Ph.D, associate professor and director of the Wilson Disease program and the study's PI. "The profile of this investigational drug is very encouraging, particularly the rapid control of clinical symptoms in combination with the simple once-daily dosing regimen and its promising side effect profile which could help improve compliance to therapy and treatment outcomes as a result I am confident WTX101 will have the potential to make a significant difference for the Wilson's Disease community."

Click here to learn more about FOCuS Trail supported by the ACD CTSO.



Contact us at

The Molecular & Behavioral Neuroscience Institute, 205 Zina Pitcher Place, Ann Arbor, MI 48105

A list of CTSO-CTSU contacts are available in the Personnel Directory.