NCT04336722已完成3 期
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy
Albireo, an Ipsen Company140 个研究点 分布在 19 个国家目标入组 254 人开始时间: 2020年7月8日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 254
- 试验地点
- 140
- 主要终点
- Time from randomization to first occurrence of liver transplant, or death
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 children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.
详细描述
Up to 70 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
- 年龄范围
- — 至 111 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A male or female patient with a clinical diagnosis of BA
- •Age at Kasai HPE ≤90 days
- •Eligible to start study treatment within 3 weeks post-Kasai HPE
排除标准
- •Patients with intractable ascites
- •Ileal resection surgery
- •ALT ≥10× upper limit of normal (ULN) at screening
- •Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization
- •Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
- •Choledochal cystic disease
- •INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
- •Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
- •Weight <3.5kg at randomization
研究组 & 干预措施
Placebo
Placebo Comparator
Capsules for oral administration (to match active) once daily for 104 weeks.
干预措施: Placebo (Drug)
Odevixibat (A4250)
Experimental
Capsules for oral administration once daily for 104 weeks.
干预措施: Odevixibat (Drug)
结局指标
主要结局
Time from randomization to first occurrence of liver transplant, or death
时间窗: From baseline to Week 104
次要结局
- Time to onset of any sentinel events(From baseline to Week 104)
- Percentage of participants with clinically significant changes in Physical Examination(From baseline to Week 104)
- Serum bile acid levels(From baseline to Weeks 13, 26, 52 and 104)
- Time to pediatric end-stage liver disease (PELD) score >15(From baseline to Week 104)
- Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)(From baseline to Week 104)
- Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)(From baseline to Week 104)
- Percentage of participants with clinically significant changes in Abdominal Ultrasound findings(From baseline to Week 26 and Week 104)
- Proportion of patients with liver transplant(From baseline to Week 104)
- Total bilirubin levels(From baseline to Weeks 13, 26, 52 and 104)
研究者
研究点 (140)
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