NCT04729751已完成2 期
Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 23
- 主要终点
- Frequency of treatment-emergent adverse events [TEAEs]
研究概览
简要总结
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].
详细描述
This is an open label study where all participants will receive maralixibat treatment.
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
- 年龄范围
- 0 Days 至 364 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Body weight of ≥2.5 kg
- •<12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).
- •Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
- •Diagnosis of PFIC or ALGS
排除标准
- •Predicted complete absence of bile salt excretion pump (BSEP) function
- •History of surgical disruption of the enterohepatic circulation
- •History of liver transplant or imminent need for liver transplant
- •Decompensated cirrhosis
- •Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
- •Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study
研究组 & 干预措施
Maralixibat
Experimental
Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
干预措施: Maralixibat (Drug)
结局指标
主要结局
Frequency of treatment-emergent adverse events [TEAEs]
时间窗: From Baseline through to Week 13
次要结局
- To evaluate the effect on liver enzymes (ALT, AST) and bilirubin(From Baseline through to Week 13)
- To assess the plasma level of maralixibat in infant participants(At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit)
- Change in fasting serum bile acid (sBA) levels(From Baseline through to Week 13)
- To evaluate the effect on LSVs(From Baseline through to Week 13)
研究者
研究点 (23)
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