TY - JOUR
T1 - Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis
AU - Ponsioen, Cyriel Y.
AU - Lindor, Keith D.
AU - Mehta, Ruby
AU - Dimick-Santos, Lara
PY - 2018
Y1 - 2018
N2 - 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).
AB - 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).
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85046949847&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/29574971
U2 - https://doi.org/10.1002/hep.29882
DO - https://doi.org/10.1002/hep.29882
M3 - Review article
C2 - 29574971
SN - 0270-9139
VL - 68
SP - 1174
EP - 1188
JO - Hepatology (Baltimore, Md.)
JF - Hepatology (Baltimore, Md.)
IS - 3
ER -