NCT02047318已完成2 期
A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001 Also Known as Maralixibat (MRX), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With 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
- 19
- 试验地点
- 3
- 主要终点
- Change From MRX Baseline to Week 48 in Fasting sBA Levels
研究概览
简要总结
The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.
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
- 12 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
LUM001 (Maralixibat)
Experimental
LUM001 also known as Maralixibat (MRX) administered orally up to twice each day
干预措施: LUM001 (Maralixibat) (Drug)
结局指标
主要结局
Change From MRX Baseline to Week 48 in Fasting sBA Levels
时间窗: MRX baseline to Week 48
The primary endpoint of this study was the mean change from MRX baseline to Week 48 in fasting sBA level.
次要结局
- Change From MRX Baseline Over Time in Fasting sBA Levels(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
- Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score(MRX baseline to Week 48)
- Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
- Change From MRX Baseline to Week 48 in Pruritus(MRX baseline to Week 48)
- Change From MRX Baseline Over Time in Pruritus(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
- Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase(MRX baseline to Week 48)
- Change From MRX Baseline Over Time in Alkaline Phosphatase(MRX baseline to end of treatment (maximum exposure was 336 weeks))
- Change From MRX Baseline Over Time in Alanine Aminotransferase(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
- Change From MRX Baseline to Week 48 in Aspartate Aminotransferase(MRX baseline to Week 48)
- Change From MRX Baseline Over Time in Aspartate Aminotransferase(MRX baseline to End of treatment (maximum exposure was 336 weeks))
- Change From MRX Baseline to Week 48 in Gamma Glutamyltransferase(MRX baseline to Week 48)
- Change From MRX Baseline Over Time in Gamma Glutamyltransferase(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
- Change From MRX Baseline to Week 48 in Alanine Aminotransferase(MRX baseline to Week 48)
- Change From MRX Baseline to Week 48 in Total and Direct Bilirubin(MRX baseline to Week 48)
- Change From MRX Baseline Over Time in Total and Direct Bilirubin(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
研究者
研究点 (3)
Loading locations...
相似试验
已完成
3 期
A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Neovascular Age-Related Macular DegenerationNeovascular Age-related Macular DegenerationNCT04777201Hoffmann-La Roche1,036
已完成
3 期
A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Diabetic Macular EdemaDiabetic Macular EdemaNCT04432831Hoffmann-La Roche1,479
已完成
3 期
Open-label Extension Study to Evaluate Long-term Safety of Sacubitril/Valsartan in Pediatric Patients With HFHeart FailureNCT03785405Novartis Pharmaceuticals216
已完成
2 期
A Long-Term Study of Rovalpituzumab TesirineCancerNCT03543358AbbVie3
已完成
2 期
Extension Study to Evaluate Safety and Efficacy of Natalizumab in Japanese Participants With Relapsing-Remitting Multiple SclerosisRelapsing-Remitting Multiple SclerosisMultiple SclerosisNCT01416155Biogen97