Alexander G Miethke, Adib Moukarzel, Gilda Porta, Joshue Covarrubias Esquer, Piotr Czubkowski, Felipe Ordonez, Antonella Mosca, Amal A Aqul, Robert H Squires, Etienne Sokal, Daniel D'Agostino, Ulrich Baumann, Lorenzo D'Antiga, Nagraj Kasi, Nolwenn Laborde, Cigdem Arikan, Chuan-Hao Lin, Susan Gilmour, Naveen Mittal, Fang Kuan Chiou, Simon P Horslen, Wolf-Dietrich Huber, Thomas Jaecklin, Tiago Nunes, Anamaria Lascau, Lara Longpre, Douglas B Mogul, Will Garner, Pamela Vig, Vera F Hupertz, Regino P Gonzalez-Peralta, Udeme Ekong, Jane Hartley, Noemie Laverdure, Nadia Ovchinsky, Richard J Thompson
BACKGROUND: Progressive familial intrahepatic cholestasis (PFIC) is a group of autosomal recessive disorders, the most prevalent being BSEP deficiency, resulting in disrupted bile formation, cholestasis, and pruritus. Building on a previous phase 2 study, we aimed to evaluate the efficacy and safety of maralixibat-an ileal bile acid transporter inhibitor-in participants with all types of PFIC. METHODS: MARCH-PFIC was a multicentre, randomised, double-blind, placebo-controlled, phase 3 study conducted in 29 community and hospital centres across 16 countries in Europe, the Americas, and Asia...
May 6, 2024: Lancet. Gastroenterology & Hepatology