Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis.

Primary sclerosing cholangitis (PSC) is a rare and chronic liver disease for which there is no effective therapy. There has been growing interest in developing treatments for this condition, with several agents being proposed as potential therapies. However, there is a lack of clarity about how to measure clinical benefit in trials in patients with this complex and rare disease. This article reviews regulatory information, the available literature on natural history, as well as potential candidate clinical and surrogate endpoints. (Hepatology 2018; 00:000-000).