An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 180
- 试验地点
- 47
- 主要终点
- Proportion of patients who are alive and have not undergone liver transplant
研究概览
简要总结
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
详细描述
This is a Phase 3, multi-center, open-label extension study to evaluate the long-term efficacy and safety of odevixibat in patients with Biliary Atresia (BA). Patients who completed treatment in the A4250-011 BOLD study and meet eligibility criteria for Study A4250-016 (BOLD-EXT) can participate. The duration of the treatment period is 104 weeks, followed by a 4-week Safety Follow-up Period. Patients who wish to continue receiving odevixibat after 104 weeks can remain on treatment in the optional extension period (OEP).
Up to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the Middle East and Asia-Pacific.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of the 104-week Treatment Period of Study A4250-011
- •Signed informed consent by caregiver
排除标准
- •Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator's discretion
- •Any conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
- •Known hypersensitivity to any components of odevixibat
- •Patients who are scheduled for a liver transplant or are likely to require a liver transplant in the immediate future based on the investigator's judgment
研究组 & 干预措施
Odevixibat (A4250)
Capsules for oral administration once daily for 104 weeks.
干预措施: Odevixibat (Drug)
结局指标
主要结局
Proportion of patients who are alive and have not undergone liver transplant
时间窗: From baseline to Week 104
次要结局
- Change in Fibrosis-4 (Fib-4) score(From baseline to Week 104)
- Change in height(From Baseline to Weeks 26, 52, 78 and 104)
- Change in weight(From Baseline to Weeks 26, 52, 78 and 104)
- Change in serum bile acids(From Baseline to Weeks 26, 52, 78, and 104)
- Change in mid-arm circumference(From Baseline to Weeks 26, 52, 78 and 104)
- Change in pediatric end-stage liver disease (PELD) score(From Baseline to Weeks 26, 52, 78 and 104)
- Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score(From baseline to Week 104)
- Time to onset of any sentinel events(From Baseline to Week 104)