Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis. Issue 3 (31st August 2018)

• Record Type: Journal Article
• Authors: Ponsioen, Cyriel Y.; Lindor, Keith D.; Mehta, Ruby; Dimick‐Santos, Lara
• Is Part Of: Hepatology. Volume 68:Issue 3(2018)
• Page Start: 1174
• Page End: 1188
• Publication Date: 2018-08-31
• Subjects: Heart -- Diseases -- Nursing -- Periodicals; Lungs -- Diseases -- Nursing -- Periodicals; Intensive care nursing -- Periodicals; Foie -- Maladies -- Périodiques; 616.362
• DOI: 10.1002/hep.29882
• Languages: English
• ISSNs: 0270-9139

Abstract

Abstract : 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. This article reviews regulatory information, the available literature on natural history, as well as potential candidate clinical and surrogate endpoints for PSC. (Hepatology 2018; 00:000‐000).