Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

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 maralixi...

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Veröffentlicht in:	The lancet. Gastroenterology & hepatology 2024-07, Vol.9 (7), p.620-631
Hauptverfasser:	Miethke, Alexander G, Moukarzel, Adib, Porta, Gilda, Covarrubias Esquer, Joshue, Czubkowski, Piotr, Ordonez, Felipe, Mosca, Antonella, Aqul, Amal A, Squires, Robert H, Sokal, Etienne, D'Agostino, Daniel, Baumann, Ulrich, D'Antiga, Lorenzo, Kasi, Nagraj, Laborde, Nolwenn, Arikan, Cigdem, Lin, Chuan-Hao, Gilmour, Susan, Mittal, Naveen, Chiou, Fang Kuan, Horslen, Simon P, Huber, Wolf-Dietrich, Jaecklin, Thomas, Nunes, Tiago, Lascau, Anamaria, Longpre, Lara, Mogul, Douglas B, Garner, Will, Vig, Pamela, Hupertz, Vera F, Gonzalez-Peralta, Regino P, Ekong, Udeme, Hartley, Jane, Laverdure, Noemie, Ovchinsky, Nadia, Thompson, Richard J
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent ATP Binding Cassette Transporter, Subfamily B - deficiency ATP Binding Cassette Transporter, Subfamily B, Member 11 - genetics Child Child, Preschool Cholestasis, Intrahepatic - blood Cholestasis, Intrahepatic - drug therapy Double-Blind Method Female Humans Infant Male Pruritus - drug therapy Pruritus - etiology Treatment Outcome
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