NCT04168385已完成2 期
MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 17
- 主要终点
- Incidence of Treatment-Emergent Adverse Events
研究概览
简要总结
Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.
详细描述
This is a multicenter, open-label study of maralixibat in subjects diagnosed with cholestatic liver disease (including, but not limited to ALGS, PFIC or Biliary Atresia) who have previously participated in a maralixibat clinical study. All subjects received maralixibat in the previous studies and will continues to receive maralixibat in this study.
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 —（Child, Adult, Older 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
Maralixibat
Experimental
Participants will all receive Maralixibat oral solution
干预措施: Maralixibat (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events
时间窗: From informed consent through approximately 4.5 years, including 30 days after last dose.
TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..
次要结局
- Long-Term Effect on Pruritus(From Baseline through Week 160, including Change from Baseline values.)
- Long-Term Effect on Serum Bile Acid Levels(From Baseline through Week 160, including Change from Baseline values.)
研究者
研究点 (17)
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