A Multicenter, Open-label, Metabolic Balance Study to Evaluate the Effects of Apraglutide on Intestinal Absorption in Adult Subjects With Short Bowel Syndrome, Intestinal Failure (SBS-IF), and Colon-in-Continuity (CIC)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- VectivBio AG
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
研究概览
简要总结
The primary objective of the trial is to evaluate the safety of apraglutide in adult subjects with SBS-IF and CIC.
详细描述
This is an international, multicenter trial to evaluate the safety of apraglutide in adult participants with SBS-IF and CIC. The active pharmaceutical ingredient is apraglutide, an investigational glucagon-like peptide-2 (GLP-2) analogue. The trial consists of an evaluation period of 52 weeks.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent for this trial prior to any trial specific assessment.
- •Male and female subjects with SBS-IF and CIC, receiving parenteral support (PS), secondary to surgical resection of the small intestine with < 200 cm from duodenojejunal flexure.
- •Subject must require parenteral support (PS) at least 2 days per week and be considered stable.
- •No restorative surgery intended to change PS requirements in the trial period.
- •Age ≥ 18 years at screening.
排除标准
- •Pregnancy or lactation.
- •Body mass index equal or higher than 30 kg/m^2 at the time of screening.
- •Major abdominal surgery in the last 6 months prior to screening.
研究组 & 干预措施
Apraglutide
All participants received apraglutide administered subcutaneously (SC) once weekly for 52 weeks.
干预措施: Apraglutide (Drug)
结局指标
主要结局
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
时间窗: Day 1 up to approximately 55 weeks
A TEAE was any unfavorable and unintended sign, symptom, or disease temporally associated with apraglutide, whether or not related, that occurred or worsened after the dose of apraglutide. A serious TEAE was defined as any TEAE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. Clinically significant changes from baseline in clinical chemistry, hematology, hemostasis, anti-drug antibodies (ADAs), and urine analysis were reported as adverse events. Adverse events of special interest (AESI) included injection site reaction, gastrointestinal obstruction, gallbladder, biliary, and pancreatic disease, fluid overload, colorectal polyps, malignancies.
Absolute Change in Absorption of Energy Over Metabolic Balance (MB) Periods From Baseline at Week 48
时间窗: Baseline and Week 48
Applicable to Protocol V3.0 implemented in France (classed as secondary endpoint in Protocol V4.0 \[implemented in Belgium\]). The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis.
次要结局
- Relative Change From Baseline in Actual Weekly Parenteral Support (PS) Volume at Weeks 4, 24, and 52(Baseline, Week 4, Week 24, and Week 52)
- Absolute Change From Baseline in Actual Weekly PS Volume at Weeks 24 and 52(Baseline, Week 24 and Week 52)
- Number of Participants Who Achieved a Reduction of at Least 1 Day Per Week of PS From Baseline at Weeks 24 and 52(Baseline, Week 24 and Week 52)
- Number of Participants Considered Clinical Responders at Weeks 24 and 52(Baseline, Week 24 and 52)
- Number of Participants Who Achieved Enteral Autonomy at Weeks 24 and 52(Weeks 24 and 52)
- Absolute Change in Total Energy in PN From Baseline at Weeks 24 and 52(Baseline, Week 24 and Week 52)
- Relative Change in Absorption of Energy Over MB Periods From Baseline at Week 48(Baseline and Week 48)
- Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48(Baseline, Week 4 and Week 48)
- Absolute Change in Urine Volume Over MB Periods From Baseline at Week 4 and Week 48(Baseline, Week 4 and Week 48)
- Absolute Change in Absorption of Energy Over MB Periods From Baseline at Week 4(Baseline and Week 4)
- Absolute Change in Urinary Electrolytes Over MB Periods From Baseline at Week 4 and Week 48(Baseline, Week 4 and Week 48)
- Change From Baseline in Pittsburgh Sleep Quality Inventory (PSQI) Total Score at Week 24 and Week 52(Baseline, Week 24 and Week 52)
- Change From Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52(Baseline, Week 24 and Week 52)
- Change From Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52(Baseline, Week 24 and Week 52)
- Secondary: Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52(Week 24 and Week 52)
- Change From Baseline in Patient Global Impression of Satisfaction With Parenteral Support (PGI-SPS) at Week 24 and Week 52(Baseline, Week 24 and Week 52)
- Trough Apraglutide Plasma Concentration (Ctrough)(Pre-dose on Weeks 2, 4, 12, 24, 32, 40, 48, and 52)
- Mean Plasma Citrulline Level(Baseline and Weeks 2, 4, 12, 24, 32, 40, 48, and 52)