Oral Budesonide Suspension (OBS) in Adolescent and Adult Subjects (11-40 Years of Age)With Eosinophilic Esophagitis: A Phase II, Randomized, Double-Blind, Placebo-Controlled Study With an Open Label Extension
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 93
- 试验地点
- 23
- 主要终点
- Percent of Participants Who Were Histologic Responders
研究概览
简要总结
This is a clinical trial to test an experimental drug for the treatment of Eosinophilic Esophagitis (EoE)
详细描述
Eosinophilic Esophagitis (EoE) is an inflammatory disorder of the esophagus and is a recognized clinical entity. Symptoms include feeding problems, heartburn, regurgitation, vomiting, abdominal pain and food impaction. The symptoms of EoE may be similar to gastroesophageal reflux disease (GERD) but do not resolve with gastric acid suppression. EoE is defined histologically as the presence of > 15 intraepithelial eosinophils per high power fields on one or more esophageal biopsy specimens.
This Phase II study is comparing oral budesonide (OBS) to placebo to demonstrate that OBS induces a histologic response and a symptom response using a Dysphagia Symptom Questionnaire over a 16 week course of therapy.
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 40 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and Females, age 11-40
- •Histologic evidence of EoE
- •History of clinical symptoms of EoE including dysphagia
- •Willing to continue with dietary, environmental or medical therapy
- •Ability to read and understand english
- •Written Consent
排除标准
- •Any Medical condition that may compromise the safety of the subjects or interfere with the signs and symptoms of EoE
- •Use of immunomodulatory therapy
- •Current use of swallowed corticosteroids
- •Esophageal strictures,varices or upper GI bleed
- •Other current diseases of the GI tract
- •Current viral infection or immunodeficiency condition
- •Pregnancy
- •Hypersensitivity to budesonide
- •History of non compliance
研究组 & 干预措施
Oral Budesonide Suspension
Taken once or twice daily for up to 40 weeks
干预措施: Oral Budesonide Suspension (MB-9) (Drug)
Matching Placebo
Taken once or twice daily for 20 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Percent of Participants Who Were Histologic Responders
时间窗: Week 16
Histologic response was defined as a peak eosinophil count \</= 6/high power field (light microscopy) (HPF) across all esophageal levels at the final treatment evaluation (Week 16). An independent, central pathologist determined the peak eosinophil count from the proximal, mid-, and distal levels and selected the maximum peak value. Histopathology data were collected in a blinded fashion.
Change From Baseline in The Dysphagia Symptom Questionnaire (DSQ) Score at The Final Treatment Period Evaluation
时间窗: Baseline, Week 16
Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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).The DSQ score was calculated based on responses to Questions 2 and 3 \[14 x (sum of points from Questions 2 and 3 in the daily DSQ)/number of diaries with non-missing data\]. Baseline was the DSQ score of the 14-day period before randomization. A negative change from baseline indicates that symptoms decreased.
次要结局
- Change From Baseline in The Peak Eosinophil Count at Each Available Esophageal Level at The Final Treatment Period Evaluation(Baseline, Week 16)
- Percent of Participants With Improved Symptoms on The Eosinophilic Esophagitis (EoE) Symptom Survey at The Final Treatment Period Evaluation(Week 16)
- Change From Baseline in The DSQ Score Over Time(Baseline, Weeks 8 and 12)
- Percent of Participants With a >/= 30% And >/= 50% Reduction In The DSQ Score From Baseline to The Final Treatment Period Evaluation(Baseline, Week 16)
- Change From Baseline in The Physician's Global Assessment (PGA) of Disease Activity at The Final Treatment Period Evaluation(Baseline, Week 16)
- Percent of Participants With New Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation(Week 16)
- Change From Baseline in The DSQ Score For The 50th Percentile of Participants at The Final Treatment Period Evaluation(Baseline, Week 16)
- Percent of Participants With Worsened Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation(Week 16)
- Change From Baseline in The Scores of DSQ Question 4 During The Treatment Period(Baseline, Weeks 8, 12, and 16)
- Percent of Participants With a Peak Eosinophil Count </= 15/High Power Field (Light Microscopy) (HPF) And </= 1/HPF at The Final Treatment Period Evaluation(Week 16)
- Percent of Participants Who Were Overall Responders at The Final Treatment Period Evaluation(Week 16)
- Change From Baseline in The Histopathologic Epithelial Features Combined Total Score at The Final Treatment Period Evaluation(Baseline, Week 16)
- Percent of Days That Participants Reported That They Avoided Solid Food During The Baseline And Treatment Periods(From 14 days prior to the baseline visit to the final treatment period evaluation)
- Change From Baseline in The Total Endoscopy Score at The Final Treatment Period Evaluation(Baseline, Week 16)
- Distribution of Responses For The Patient Global Impression of Change (PGIC) Survey at The Final Treatment Evaluation(Week 16)
- Percent of Participants Who Were Symptom Responders on The DSQ+Pain Scale at The Final Treatment Period Evaluation(Week 16)
- Percent of Participants With No Change in Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation(Week 16)
- Percent of Participants Without Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation(Week 16)
- Change From Baseline in The Scores of DSQ Question 1 During The Treatment Period(Baseline, Weeks 8, 12, and 16)