A 4-Stage, Open-label, Multicenter Study Including Long-term Extension to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 11
- 试验地点
- 5
- 主要终点
- Stage 2: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of an investigational treatment (teduglutide) in Japanese patients with PN-dependent SBS. This study will also look at how teduglutide moves through the body (pharmacokinetics).
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
- 16 Years 至 —（Child, Adult, Older 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
Arm 1
0.05 mg/kg/day administered by subcutaneous injection over a 24-week period.
干预措施: Teduglutide (Drug)
结局指标
主要结局
Stage 2: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
时间窗: Baseline (stage 2), Week 24
Percent change from baseline in weekly PS volume at Week 24 was reported.
Stage 2: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Week 24
时间窗: Baseline (stage 2), Week 24
Absolute change from baseline in number of days per Week of PS usage at Week 24 was reported.
Stage 2: Number of Participants Who Achieved Enteral Autonomy at Week 24
时间窗: Week 24
Enteral autonomy was defined as no prescribed PS and no use of PS recorded in the participant diary at the end of stage 2. Number of participants who achieved enteral autonomy at Week 24 was reported.
Stage 2: Percent Change From Baseline in Plasma Citrulline Levels at Week 24
时间窗: Baseline (stage 2), Week 24
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at Week 24 was reported.
Stage 3: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
时间窗: Baseline (stage 3), Extension Month 24
Percent change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Extension Month 24
时间窗: Baseline (stage 3), Extension Month 24
Absolute change from baseline in number of days per Week of PS usage at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 2: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
时间窗: Baseline (stage 2), Week 24
Absolute change from baseline in weekly PS volume at Week 24 was reported.
Stage 2: Percentage of Participants Who Demonstrate Response to Teduglutide at End of Stage 2
时间窗: End of Stage 2 (up to Week 24)
Response was defined as the achievement of at least a 20% reduction from baseline (Visit 2) in weekly PS volume at Week 20 and again at Week 24.
Stage 2: Absolute Change From Baseline in Plasma Citrulline Levels at Week 24
时间窗: Baseline (stage 2), Week 24
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at Week 24 was reported.
Stage 3: Absolute Change From Baseline in Plasma Citrulline Levels at Extension Month 24
时间窗: Baseline (stage 3), Extension Month 24
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Percent Change From Baseline in Plasma Citrulline Levels at Extension Month 24
时间窗: Baseline (stage 3), Extension Month 24
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
时间窗: Baseline (stage 3), Extension Month 24
Absolute change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 4: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
时间窗: Baseline (stage 4), End of Treatment (up to 47 months)
Absolute change from baseline in weekly PS volume at end of treatment was reported.
Stage 4: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
时间窗: Baseline (stage 4), End of Treatment (up to 47 months)
Percent change from baseline in weekly PS volume at end of treatment was reported.
Stage 4: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at End of Treatment
时间窗: Baseline (stage 4), End of Treatment (up to 47 months)
Absolute change from baseline in number of days per week of PS usage at end of treatment was reported.
Stage 4: Absolute Change From Baseline in Plasma Citrulline Levels at End of Treatment
时间窗: Baseline (stage 4), End of Treatment (up to 47 months)
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at end of treatment was reported.
Stage 4: Percent Change From Baseline in Plasma Citrulline Levels at End of Treatment
时间窗: Baseline (stage 4), End of Treatment (up to 47 months)
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change in plasma citrulline from baseline at end of treatment was reported.
次要结局
- Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC0-Inf) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)
- Area Under the Concentration-Time Curve From Time Zero to the Last Time Point (AUC0-t) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)
- Maximum Concentration (Cmax) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)
- Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)
- Terminal Half-Life (t1/2) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)
- Apparent Clearance (CL/F) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)
- Apparent Volume of Distribution (Vz/F) of Teduglutide in Plasma(Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose)