A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Investigate the Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Subjects With Active Crohn's Disease
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Celgene
- 入组人数
- 701
- 试验地点
- 535
- 主要终点
- The Percentage of Participants Who Achieved a Clinical Remission at Week 12
研究概览
简要总结
The purpose of study is to test the effects of an experimental medication GED-0301 (mongersen) in patients who have active Crohn's disease. The study will test GED-0301 compared to placebo for 52 weeks. The study treatment is blinded which means that patients and the study doctor will not know which treatment has been assigned. Patients in this study will be allowed treatment with stable doses of oral aminosalicylates, oral corticosteroids, immunosupressants and antibiotics for the treatment of Crohn's disease.
After 12 weeks in the study until the end of the study, patients who do not have an improvement in their Crohns disease symptoms will have the option to enter a long term active treatment study. Participants who discontinued the study anytime or completed the study at Week 52 were then observed for an additional 4 weeks.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must satisfy the following criteria to be enrolled in the study:
- •Male or female ≥ 18 years
- •Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD)
- •Must meet a determined average minimum number of daily stools or rating of abdominal pain over a 7 day period
- •Subject must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine, 6-mercaptopurine, or methotrexate); or biologics for the treatment of CD.
排除标准
- •The presence of any of the following will exclude a subject from enrollment:
- •Diagnosis of ulcerative colitis (UC), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis
- •Local manifestations of Crohn's Disease (CD) such as symptomatic/severe strictures, abscesses, short bowel syndrome; or other disease complications for which surgery might be indicated or could confound the evaluation of efficacy
- •Intestinal resection within 6 months or any intra-abdominal surgery within 3 months prior to the Screening Visit
- •Ileostomy or a colostomy
- •Subject has a history of any clinically significant medical condition that, in the investigator's opinion, would prevent the subject from participating in the study.
研究组 & 干预措施
GED0301 160mg x 12 weeks followed by periodic 160 mg GED0301
GED-0301 160 mg once daily (QD) for 12 weeks; followed by placebo QD for 4 weeks; followed by alternating GED-0301 160 mg QD for 4 weeks and placebo QD for 4 weeks, until the the Week 52 Visit
干预措施: GED-0301 (Drug)
GED0301 160mg x 12 weeks followed by periodic 160 mg GED0301
GED-0301 160 mg once daily (QD) for 12 weeks; followed by placebo QD for 4 weeks; followed by alternating GED-0301 160 mg QD for 4 weeks and placebo QD for 4 weeks, until the the Week 52 Visit
干预措施: Placebo (Drug)
GED0301 160mg x 12 weeks followed by periodic GED0301 40mg
GED-0301 160 mg once daily (QD) for 12 weeks; followed by placebo QD for 4 weeks; followed by alternating GED-0301 40 mg QD for 4 weeks and placebo QD for 4 weeks, until the Week 52 Visit
干预措施: GED-0301 (Drug)
GED0301 160mg x 12 weeks followed by periodic GED0301 40mg
GED-0301 160 mg once daily (QD) for 12 weeks; followed by placebo QD for 4 weeks; followed by alternating GED-0301 40 mg QD for 4 weeks and placebo QD for 4 weeks, until the Week 52 Visit
干预措施: Placebo (Drug)
GED0301 160mg x 12 weeks followed by continuous GED0301 40mg
GED-0301 160 mg once daily (QD) for 12 weeks; followed by continuous GED-0301 40 mg QD, until the Week 52 Visit
干预措施: GED-0301 (Drug)
Placebo
Placebo once daily (QD) until the Week 52 Visit
干预措施: Placebo (Drug)
结局指标
主要结局
The Percentage of Participants Who Achieved a Clinical Remission at Week 12
时间窗: Week 12
Clinical remission is defined as a Crohn's Disease Activity Index (CDAI) score \< 150. The Crohn's Disease Activity Index is used to quantify the signs and symptoms of Crohn's disease and the affect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
次要结局
- Percentage of Participants With Endoscopic Response-25 Centrally Read at Week 12(Week 0, Week 12)
- Percentage of Participants With Endoscopic Response-50 Centrally Read at Week 52(Week 52)
- The Percentage of Participants Who Achieved a Clinical Response at Week 12(Week 12)
- The Number of Participants Who Discontinued IP Due to an Treatment Emergent Adverse Events(From the first day of GED-0301 until 28 days after the last dose of IP; maximum treatment duration was 52.6 weeks)
- Percentage of Participants Who Achieved Clinical Remission at Week 52(Week 52)
- The Percentage of Participants Who Achieved a Clinical Response at Week 4(Week 4)
- The Percentage of Participants Who Achieved a Clinical Remission at Week 4(Week 4)
- The Percentage of Participants Who Achieved a Corticosteroid-Free Clinical Remission at Week 52(Week 52)
- Percentage of Participants Who Achieved a Sustained Clinical Remission at Both Week 12 and 52(Weeks 12 and 52)
- Percentage of Participants With Endoscopic Remission Centrally Read at Week 52(Week 52)
- The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) From Week 0 to Week 52(From the first day of GED-0301 until 28 days after the last dose of investigational product (IP); maximum treatment duration was 52.6 weeks)