Efficacy and Safety of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Treatment of Pruritus in Alagille Syndrome Patients

The ITCH study was a randomized, double-blind, placebo-controlled, parallel group, multi-center study in children with Alagille syndrome (ALGS). The study was designed to investigate the effects of LUM001 (Maralixibat), compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers associated with cholestatic liver disease, following daily dosing over a 13-week period.

ITCH研究是一项针对阿拉吉耶综合征（Alagille syndrome, ALGS）儿童患者的随机、双盲、安慰剂对照、平行组多中心临床试验。本研究旨在考察每日给药持续13周后，LUM001（马利昔班，Maralixibat）相较于安慰剂，对胆汁淤积性肝病相关瘙痒、血清胆汁酸、肝酶及其他生化标志物的影响。