Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 75
- 试验地点
- 22
- 主要终点
- Mean Change in Total Serum Bilirubin Levels
研究概览
简要总结
A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).
详细描述
This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.
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
- 21 Days 至 111 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
- •HPE or Kasai Procedure within 3 weeks prior to randomization
- •Clinical diagnosis of biliary atresia
排除标准
- •Subjects with intractable chronic diarrhea at randomization
- •Subjects not tolerating enteral feeds at randomization
- •History of ileal resection
- •Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
- •Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
- •Evidence of liver failure (e.g. significant ascites)
研究组 & 干预措施
Double Blind - Maralixibat
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
干预措施: Maralixibat (Drug)
Double Blind - Placebo
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
干预措施: Placebo (Other)
Open Label - Maralixibat
The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat.
干预措施: Maralixibat (Drug)
结局指标
主要结局
Mean Change in Total Serum Bilirubin Levels
时间窗: From baseline to Week 26
次要结局
- Mean Change in Total Serum Bile Acids(From baseline to Week 26)
- Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26(From baseline to Week 26)
- Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.(From Baseline to Week 26)
- Proportion of Participants Undergoing Liver Transplantation or Death(From Baseline to Week 26)
- Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices.(From Baseline to Week 26)
- Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL(From Baseline to Week 26)
- Proportion of Participants With Mean sBA Levels ≤40 mmol/L(From Baseline to Week 26)