Two studies have reported positive phase III trials for ileal bile acid transporter inhibitors for pruritus and other cholestasis-associated clinical features. In the first study (ASSERT), the efficacy and safety of odevixibat was assessed for Alagille syndrome in a double-blind, randomized, placebo-controlled trial enrolling patients across ten countries. Patients (n = 52; median age 5.5 years) were randomly assigned to receive odevixibat (n = 35; 120 μg/kg per day) or placebo (n = 17) for 24 weeks. In the second study (MARCH-PFIC), a multicentre, randomized, double-blind, placebo-controlled study across 26 countries assessed the efficacy of maralixibat for progressive familial intrahepatic cholestasis (PFIC). Patients (n = 93; aged 1–17 years) were randomly assigned to take maralixibat (n = 47; 14 in the BSEP deficiency cohort, 33 in the all-PFIC cohort; starting dose 142.5 μg/kg, then escalated to 570 μg/kg) or a placebo (n = 46; 17 in the BSEP cohort, 31 in the all-PFIC cohort) twice-daily for 26 weeks. Both studies reported improvements in pruritus and reduced bile acid levels in the drug groups versus placebo. The most common adverse effect for both drugs was diarrhoea.

两项研究报道了回肠胆汁酸转运蛋白抑制剂治疗瘙痒和其他胆汁淤积相关临床特征的阳性 III 期试验。在第一项研究 （ASSERT） 中，在一项招募 10 个国家/地区患者的双盲、随机、安慰剂对照试验中评估了 odevixibat 对 Alagille 综合征的疗效和安全性。患者 （n = 52;中位年龄 5.5 岁） 被随机分配接受 odevixibat （n = 35;120 μg/kg 每天） 或安慰剂 （n = 17），持续 24 周。在第二项研究 （MARCH-PFIC） 中，一项跨越 26 个国家的多中心、随机、双盲、安慰剂对照研究评估了 maralixibat对进行性家族性肝内胆汁淤积症 （PFIC） 的疗效。患者（n = 93;年龄 1-17 岁）被随机分配服用 maralixibat（n = 47;BSEP 缺乏队列中有 14 名，全 PFIC 队列中有 33 名;起始剂量 142.5 μg/kg，然后增加至 570 μg/kg）或安慰剂（n = 46;BSEP 队列中有 17 名，全 PFIC 队列中有 31 名）每天两次，持续 26 周。两项研究都报告了与安慰剂相比，药物组的瘙痒改善和胆汁酸水平降低。两种药物最常见的不良反应是腹泻。