The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 37
- 试验地点
- 13
- 主要终点
- Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus
研究概览
简要总结
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
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
LUM001 for oral administration
干预措施: LUM001 (Drug)
Placebo
Placebo administered orally once each day
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus
时间窗: Baseline, Week 13/Early Termination
Pruritus was assessed using Itch report outcome measure (ItchRO\[Obs\]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
次要结局
- Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level(Baseline, Week 13/Early Termination)
- Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels(Baseline, Week 13/Early Termination)
- Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations(Baseline, Week 13/Early Termination)