A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi) in Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primary Biliary Cirrhosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 66
- 试验地点
- 24
- 主要终点
- Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)
研究概览
简要总结
The study is a randomized, double-blind, placebo-controlled, multicenter study. It is a 13-week Phase 2 study in adults with primary biliary cirrhosis designed to compare the effect of daily dosing with UDCA in combination with LUM001 or placebo.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Primary Biliary Cirrhosis
- •Moderate to severe pruritus
- •Taking ursodeoxycholic acid (UDCA) for at least 6 months, or unable to tolerate UDCA
- •Ability to understand and willingness to sign informed consent prior to initiation of any study procedures
排除标准
- •History or presence of other concomitant significant liver disease
- •Liver transplant
- •Known HIV infection
- •Women who are pregnant or lactating
研究组 & 干预措施
LUM001 and Ursodeoxycholic Acid (UDCA)
Administered orally once daily
干预措施: LUM001 (Drug)
LUM001 and Ursodeoxycholic Acid (UDCA)
Administered orally once daily
干预措施: Ursodeoxycholic Acid (Drug)
Placebo and Ursodeoxycholic Acid (UDCA)
Administered orally once daily
干预措施: Placebo (Drug)
Placebo and Ursodeoxycholic Acid (UDCA)
Administered orally once daily
干预措施: Ursodeoxycholic Acid (Drug)
结局指标
主要结局
Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)
时间窗: Baseline and Week 13/ET
Pruritus was assessed using ItchRO measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). (ItchRO) scores ranged from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit.
次要结局
- Change From Baseline in Fasting Serum Bile Acid Level at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET)(Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET))
- Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13(Baseline, Weeks 4, 8 and 13)
- Change From Baseline in Pruritus Using Adult ItchRO Average Daily Scores at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET)(Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET))
- Change From Baseline in Alkaline Phosphatase (ALP) at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET)(Baseline, Weeks 4, 8, 13 and Last Post-baseline (Week 13/ET))
- Change From Baseline in 5-D Itch Score at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET)(Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET))
- Change From Baseline in Bile Acid Synthesis as Measured by Serum 7 Alpha-Hydroxy-4-Cholesten-3-One C4 Level [7 Alpha C4]) at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET)(Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET))