A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Albireo
- 入组人数
- 52
- 试验地点
- 32
- 主要终点
- Change From Baseline in Scratching Score
研究概览
简要总结
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
详细描述
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of Alagille syndrome
- •History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
- •Elevated serum bile acid level
排除标准
- •History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
- •History of liver transplant, or a liver transplant is planned within 6 months of randomization
- •ALT >10× upper limit of normal (ULN) at screening
- •Total bilirubin >15 × ULN at screening
- •Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome
研究组 & 干预措施
Odevixibat (A4250)
Capsules for oral administration once daily for 24 weeks.
干预措施: Odevixibat (Drug)
Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Scratching Score
时间窗: Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.
Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.
次要结局
- Serum Bile Acid Levels(Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.)