Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis

Cyriel Y. Ponsioen, Keith D. Lindor, Ruby Mehta, Lara Dimick‐Santos – 25 March 2018 – Primary sclerosing cholangitis (PSC) is a rare and chronic liver disease for which there is no effective therapy. Interest has grown in developing treatments for this condition, with several agents proposed as potential therapies. However, there is a lack of clarity about how to measure clinical benefit in trials involving patients with this complex and rare disease.

Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis

Cyriel Y. Ponsioen, Keith D. Lindor, Ruby Mehta, Lara Dimick‐Santos – 25 March 2018 – Primary sclerosing cholangitis (PSC) is a rare and chronic liver disease for which there is no effective therapy. Interest has grown in developing treatments for this condition, with several agents proposed as potential therapies. However, there is a lack of clarity about how to measure clinical benefit in trials involving patients with this complex and rare disease.

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