Oral Budesonide Suspension (OBS) in Adolescent and Adult Subjects (11 to 55 Years of Age, Inclusive) With Eosinophilic Esophagitis: A Phase 3 Randomized, Double-blind, Placebo-controlled Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 318
- 试验地点
- 75
- 主要终点
- Number of Participants With Histologic Response at the Final Treatment Period Evaluation (Week 16)
研究概览
简要总结
A study in adolescents and adults with eosinophilic esophagitis (EoE) to measure the histologic response and determine if any reduction in dysphagia is achieved.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 11 Years 至 55 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
Oral Budesonide Suspension (OBS)
Participants will receive Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks.
干预措施: Oral Budesonide Suspension (OBS) (Drug)
Placebo
Participants will receive oral dose of 10 ml of placebo matched with the experimental drug twice daily up to 16 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With Histologic Response at the Final Treatment Period Evaluation (Week 16)
时间窗: Week 16
Histologic response was defined as a peak eosinophil count of less than or equal to (\<=) 6/ high-powered field (HPF) across all available esophageal levels at final treatment period evaluation (Week 16). Histologic response after 12 weeks of double blind treatment at Week 16 was reported.
Number of Participants With Dysphagia Symptom Response at the Final Treatment Period Evaluation (Week 16)
时间窗: Week 16
Dysphagia symptom response was defined as greater than or equal to (\>=) 30 percent (%) reduction in the Dysphagia Symptom Questionnaire (DSQ) combined score (questions 2+3). DSQ contained 4 questions, all participants used a diary, and responded to Questions 1 (did you eat solid food) and 2 (did food pass slowly or get stuck). If the participant's answer to Question 2 was 'No', the diary ended for that day. If a participant answered 'Yes', he/she advanced to Questions 3 (did you have to do anything to make the food go down or get relief) and 4 (extent to which the participant experienced pain while swallowing). DSQ score= (\[sum of points from questions 2+3 in the daily DSQ\]×14)/ Number of diaries reported with non-missing data. Dysphagia symptom response after 12 weeks of double blind treatment at Week 16 was reported.
次要结局
- Number of Participants With Dysphagia Symptom Response (Binary Response) at the Final Treatment Period Evaluation (Week 16)(Week 16)
- Number of Participants With Overall Binary Response II at the Final Treatment Period Evaluation (Week 16)(Week 16)
- Terminal Rate Constant (Lambda Z) of Budesonide in Plasma(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)
- Number of Participants With Peak Eosinophil Count Less Than (<)15/High-Powered Field (HPF) or Less Than or Equal to (<=)1/High-Powered Field (HPF) at the Final Treatment Period Evaluation (Week 16)(Week 16)
- Change From Baseline in Total Endoscopy Score at the Final Treatment Period Evaluation (Week 16)(Baseline, Week 16)
- Change From Baseline in the Peak Eosinophil Count at the Final Treatment Period Evaluation (Week 16)(Baseline, Week 16)
- Change From Baseline in the Histopathologic Epithelial Features Combined Total Score Ratio (TSR) at the Final Treatment Period Evaluation (Week 16)(Baseline, Week 16)
- Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Combined Score at the Final Treatment Period Evaluation (Week 16)(Baseline, Week 16)
- Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) + Pain Score (Questions 2 +3+4) at the Final Treatment Period Evaluation (Week 16)(Baseline, Week 16)
- Maximum Observed Concentration (Cmax) of Budesonide in Plasma(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)
- Terminal Half-Life (t1/2) of Budesonide in Plasma(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)
- Apparent Oral Clearance (CL/F) of Budesonide in Plasma(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)
- Apparent Volume of Distribution (Vz/F) of Budesonide in Plasma(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)
- Number of Participants With Treatment-Emergent Adverse Events (AE)(From start of study drug administration up to follow-up (Week 20))
- Number of Participants With Overall Binary Response I at the Final Treatment Period Evaluation (Week 16)(Week 16)
- Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) Pain Score (Question 4) at the Final Treatment Period Evaluation (Week 16)(Baseline, Week 16)
- Time to Maximum Observed Plasma Concentration (Tmax) of Budesonide in Plasma(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)
- Area Under the Plasma Concentration-Time Curve (AUCtau) Between the Defined Interval of Budesonide Doses(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16)