2022-501090-39-00招募中3 期
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
- 招募中
- 发起方
- Ipsen Pharma
- 入组人数
- 57
- 试验地点
- 17
- 主要终点
- Time from the date of first dose of study treatment to first occurrence of liver transplant, or death
研究概览
简要总结
To demonstrate a sustained effect of odevixibat on native liver survival (NLS) in children with biliary atresia (BA) who have completed study A4250-011 (BOLD)
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Completion of the 104-week Treatment Period of Study A4250-011
- •Signed informed consent by the patient’s parent(s) or legal guardian
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
结局指标
主要结局
Time from the date of first dose of study treatment to first occurrence of liver transplant, or death
Time from the date of first dose of study treatment to first occurrence of liver transplant, or death
次要结局
- • Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score from baseline to Weeks 4, 13, 26, 39, 52, 78, and 104 • Change in fibrosis-4 (Fib-4) score from baseline to Weeks 4, 13, 26, 39, 52, 78 and 104 • Change from baseline in serum bile acids at Weeks 26, 52, 78, and 104
- • Change in growth from baseline to Weeks 26, 52, 78, and 104 after initiation of odevixibat treatment, defined as the linear growth deficit (height for age, weight for age, and body mass index [BMI]) compared to a standard growth curve (Z-score, standard deviation [SD] from P50); mid-arm circumference
- • Time to onset of first sentinel event during the 104-week treatment period.
- • Change in pediatric end-stage liver disease (PELD) score from baseline to Weeks 26, 52, 78, and 104
- • Proportion of patients who are alive and have not undergone a liver transplant at Weeks 26, 52, 78 and 104
研究者
Clinical Operations Department
Scientific
Ipsen Pharma
研究点 (17)
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