NCT01903460已完成2 期
A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Paediatric Patients With Alagille Syndrome
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
- 20
- 试验地点
- 3
- 主要终点
- Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level
研究概览
简要总结
The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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
- •Diagnosis of Alagille Syndrome
- •Evidence of cholestasis
- •Moderate to severe pruritus
- •Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures
排除标准
- •Surgical disruption of the enterohepatic circulation
- •Liver transplant
- •History or presence of other concomitant liver disease
- •Females who are pregnant or lactating
- •Known HIV infection
研究组 & 干预措施
LUM001
Experimental
LUM001 administered orally once each day
干预措施: LUM001 (Drug)
Placebo
Placebo Comparator
Placebo administered orally once each day
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level
时间窗: Baseline to 13 weeks or end of treatment
Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.
次要结局
- Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores(Baseline to 13 weeks or end of treatment)
- Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes(Baseline to 13 weeks or end of treatment)
研究者
研究点 (3)
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