A 12-Week Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects Aged 1 Year Through 17 Years, With Short Bowel Syndrome Who Are Dependent on Parenteral Support
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 42
- 试验地点
- 19
- 主要终点
- Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
研究概览
简要总结
This will be an open label, 4-cohort study. Non-randomized subjects will receive teduglutide in each of the 3 active cohorts. An attempt will be made to enroll additional subjects into an observational cohort who will receive standard of care. Three doses of teduglutide are to be investigated for 12 weeks. All subjects will be screened prior to the start of treatment (SOT) to establish baseline characteristics including safety, eligibility and nutritional support parameters.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) for at least 12 months prior to screening
- •Short bowel syndrome that requires parenteral nutrition/intravenous (PN/IV) support
- •Stable PN/IV support for at least 3 months prior to enrollment based upon the opinion of the investigator
排除标准
- •Serial transverse enteroplasty or any other bowel lengthening procedure performed within the past 3 months
- •Unstable absorption due to cystic fibrosis, untreated Hirschsprung's disease or known DNA abnormalities (ie, Familial Adenomatous Polyposis, Fanconi syndrome)
- •Evidence of obstruction on upper gastrointestinal (GI) series done within 6 months prior to screening
- •Major gastrointestinal surgical intervention within 3 months prior to screening (insertion of feeding tube or endoscopic procedure is allowed)
研究组 & 干预措施
teduglutide
Open label teduglutide, subcutaneously injected.
干预措施: teduglutide (Drug)
结局指标
主要结局
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
时间窗: Baseline, Week 12
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
时间窗: Baseline, Week 16
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
时间窗: Baseline, End of Treatment
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
时间窗: Baseline, Week 16
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
时间窗: Baseline, Week 12
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
时间窗: Baseline, End of Treatment
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
次要结局
未报告次要终点