NCT04185363已完成3 期
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 84
- 试验地点
- 42
- 主要终点
- Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
研究概览
简要总结
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
详细描述
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
- •Completion of study MRX-502
排除标准
- •Any female who is pregnant or lactating or who is planning to become pregnant
- •Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
- •History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
- •Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
- •Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study
- •Cognitive impairment of the subject or caregiver that would, in the opinion of the investigator, preclude appropriate understanding of study information and compliance with study procedures
研究组 & 干预措施
Maralixibat
Experimental
All subjects will receive Maralixibat oral solution
干预措施: Maralixibat (Drug)
结局指标
主要结局
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
时间窗: From baseline through time of interim analysis, up to 120 weeks
次要结局
- Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time(From baseline through study completion, up to approximately 4 years)
- Mean change from baseline over time in serum bile acid (sBA) levels(From baseline through study completion, up to approximately 4 years)
- Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score(From baseline through study completion, up to approximately 4 years)
- Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score(From baseline through study completion, up to approximately 4 years)
- Mean change from baseline over time in height and weight z-scores(From baseline through study completion, up to approximately 4 years)
研究者
研究点 (42)
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