Clinical Trials for Wilson Disease

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and other components of the National Institutes of Health (NIH) conduct and support research into many diseases and conditions, including liver diseases.

What are clinical trials for Wilson disease?

Clinical trials—and other types of clinical studies—are part of medical research and involve people like you. When you volunteer to take part in a clinical study, you help doctors and researchers learn more about disease and improve health care for people in the future.

Researchers are conducting clinical studies to better understand liver diseases, such as Wilson disease.

Find out if clinical studies are right for you.

Watch a video of NIDDK Director Dr. Griffin P. Rodgers explaining the importance of participating in clinical trials.

What clinical studies for Wilson disease are looking for participants?

You can view a filtered list of clinical studies on Wilson disease that are federally funded, open, and recruiting at www.ClinicalTrials.gov. You can expand or narrow the list to include clinical studies from industry, universities, and individuals; however, the NIH does not review these studies and cannot ensure they are safe. Always talk with your health care provider before you participate in a clinical study.

How is NIDDK- and NIH-funded research advancing the understanding of Wilson disease?

The NIDDK and other components of the NIH support basic research to increase our understanding of Wilson disease and lay the foundation for future advances in diagnosis and treatment. Research topics include

• understanding how copper is absorbed from the intestines, handled by tissues including brain and liver tissues, and then removed from the body
• understanding how the gene mutations in Wilson disease lead to copper not being properly removed and building up in the brain, liver, red blood cells, and kidneys
• developing better tests that doctors could use to check for Wilson disease in all infants at birth
• developing better treatments for Wilson disease and better ways to check copper levels in the blood and liver
• developing gene therapy for Wilson disease that might keep copper in the body at safe levels without the need for a special diet or lifelong treatment with chelating agents