Clinical Trials for Alagille Syndrome

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 Alagille syndrome?

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 studying many aspects of Alagille syndrome, such as how the disease progresses over time. 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 Alagille syndrome are looking for participants?

You can view a filtered list of clinical studies on Alagille syndrome 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.

What have we learned about Alagille syndrome from NIDDK-funded research?

The NIDDK has supported many research projects to learn more about Alagille syndrome. For example, the NIDDK supports the Childhood Liver Disease Research Network (ChiLDReN), which studies rare liver diseases that affect children, including Alagille syndrome. ChiLDReN researchers have made discoveries about

• how Alagille syndrome affects children’s health and quality of life
• the gene mutations that cause Alagille syndrome
• gene mutations that might affect the severity of the disease