Open Label Study of the Efficacy and Long Term Safety of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 33
- 试验地点
- 10
- 主要终点
- Change From Baseline to Endpoint (Week 13) in Fasting sBA Level
研究概览
简要总结
This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.
详细描述
The study is divided into 5 parts: a 4-week dose escalation period, a 4-week stable dosing period, a 5-week stable dosing period, a 59-week long-term exposure period, and an optional follow-up treatment period for eligible participants who continue treatment with LUM001. Participants in the optional follow-up treatment period will continue to receive study drug until they are eligible to enter another LUM001 study or until LUM001 is available commercially, whichever occurs first.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
LUM001 (Maralixibat)
Participants will receive LUM001, also known as Maralixibat (MRX) twice a day (BID).
干预措施: LUM001 (Maralixibat) (Drug)
结局指标
主要结局
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level
时间窗: Baseline (Day 0) to Week 13
次要结局
- Change From Baseline to Week 13/ET in Direct Bilirubin(Baseline (Day 0) to Week 13)
- Change From Baseline to Week 13/ET in ALT(Baseline (Day 0) to Week 13)
- Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)(Baseline (Day 0) to Week 13)
- Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)(Baseline (Day 0) to Week 13)
- Change From Baseline to Week 13/ET in Total Bilirubin(Baseline (Day 0) to Week 13)