A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once-daily Oral Avatrombopag in Japanese Subjects With Chronic Liver Disease and Thrombocytopenia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 39
- 主要终点
- Percentage of Participants With Platelet Count Greater Than or Equal to 50 x 10^9/L and at Least 20 x 10^9/L Increase From Baseline at Visit 4
研究概览
简要总结
This is a phase 2, randomized, double-blind, placebo-controlled, parallel group study using avatrombopag for Japanese subjects with thrombocytopenia associated with chronic liver disease. This study will assess the effect of avatrombopag on platelet counts in Japanese subjects. Subjects will be enrolled into 2 cohorts according to the mean platelet count measured at Screening and Baseline. Within the lower baseline platelet count cohort (less than 40 x 10^9/L), subjects will be randomized in a 1:1:1:3 ratio to receive placebo, 20 mg avatrombopag, 40 mg avatrombopag, or 60 mg avatrombopag for 5 days. Within the higher baseline platelet count cohort (from 40 to less than 50 x 10^9/L), subjects will be randomized in a 2:1:2 ratio to receive placebo, 20 mg avatrombopag, or 40 mg avatrombopag for 5 days.
详细描述
This study will consist of Prerandomization Phase and Randomization Phase. The Prerandomization Phase includes a Screening Period (Day -28 to Day -1) and a Baseline Period (Day 1). During the Prerandomization Phase, participants will be divided into two cohorts based on the platelet count: Low Platelet Count Cohort and High Platelet Count Cohort. Participants in Low Platelet Count Cohort will be randomized to receive one of the four treatments: Placebo, Avatrombopag 20 mg, Avatrombopag 40 mg, or Avatrombopag 60 mg. Participants in High Platelet Count Cohort will be randomized to receive one of the three treatments: Placebo, Avatrombopag 20 mg, or Avatrombopag 40 mg.
The Randomization Phase includes the Treatment Period and the Follow-up Period. The Follow-up Period comprises 3 visits: Visit 4 (5 to 8 days after last dose of study drug [Study Day 10 to 13]), Visit 5 (12 to 15 days after last dose of study drug [Study Day 17 to 20]), and 30 days after receiving the last dose of study drug.
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
- 20 Years 至 —（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
avatrombopag 20-mg
20 mg avatrombopag (1 x 20 mg tablet and 2 x 20 mg matching placebo tablets) once daily on Days 1 through 5
干预措施: avatrombopag (Drug)
avatrombopag 40-mg
40 mg avatrombopag (2 x 20 mg tablets and 1 x 20 mg matching placebo tablet) once daily on Days 1 through 5
干预措施: avatrombopag (Drug)
avatrombopag 60-mg
60 mg avatrombopag (3 x 20 mg tablets) once daily on Days 1 through 5
干预措施: avatrombopag (Drug)
placebo l
Placebo (3 x 20-mg matching placebo tablets) once daily on Days 1 through 5
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants With Platelet Count Greater Than or Equal to 50 x 10^9/L and at Least 20 x 10^9/L Increase From Baseline at Visit 4
时间窗: Baseline and Visit 4 (Day 10)
Responders were defined as participants whose platelet count was greater than or equal to 50×10\^9/liter (L) and change from baseline was at least 20×10\^9/L at Visit 4. Participants receiving a platelet transfusion prior to the platelet count assessment at Visit 4 were considered as non-responders in the analysis. Two-sided 95% confidence interval (CI) is calculated by Clopper and Pearson method.
次要结局
- Percentage of Participants With Platelet Count Greater Than or Equal to 50 x 10^9/L at Each Visit(Visit 3 (Day 4 or 5), Visit 4 (Day 10), Visit 5 (Day 17) and Visit 6 (Day 35))
- Percentage of Participants With Platelet Count Greater Than or Equal to 75 x 10^9/L at Each Visit(Visit 3 (Day 4 or 5), Visit 4 (Day 10), Visit 5 (Day 17) and Visit 6 (Day 35))
- Percentage of Participants With Platelet Count Greater Than or Equal to 150 x 10^9/L At Each Visit(Visit 3 (Day 4 or 5), Visit 4 (Day 10), Visit 5 (Day 17) and Visit 6 (Day 35))
- Percentage of Participants With Platelet Count Greater Than or Equal to 200 x 10^9/L At Each Visit(Visit 3 (Day 4 or 5), Visit 4 (Day 10), Visit 5 (Day 17) and Visit 6 (Day 35))
- Platelet Count and Change From Baseline in Platelet Count by Visit(Baseline, Visit 3 (Day 4 or 5), Visit 4 (Day 10), Visit 5 (Day 17) and Visit 6 (Day 35))