A Phase 2/3, Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Oral Ozanimod (RPC1063) in Pediatric Participants With Moderately to Severely Active Crohn's Disease With an Inadequate Response to Conventional Therapy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 5
- 试验地点
- 46
- 主要终点
- Percentage of Participants Who Achieve Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety, drug levels, and drug effects of ozanimod in pediatric participants with moderately to severely active Crohn's Disease.
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
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have a Pediatric Crohn's Disease Activity Index (PCDAI) score ≥ 30 and a Simple Endoscopic Score for Crohn's Disease (SES-CD) score ≥ 6 (or SES-CD ≥ 4 in participants with isolated ileal disease)
- •Participant has an inadequate response, intolerance, or loss of response to at least 1 of the following treatments for Crohn's Disease (CD):
- •i) corticosteroids ii) immunomodulators iii) biologic therapy iv) other systemic immunomodulatory therapies for CD
排除标准
- •Participant is likely to require, in the physician's judgment, bowel resection within 12 weeks of entry into the study
- •Participant has current stoma, ileal-anal pouch anastomosis, fistula that is likely to require, in the physician's judgment, surgical or medical intervention within 12 weeks of entry into the study or need for ileostomy or colostomy
- •Participant has extensive small bowel resection (> 100 cm) or known diagnosis of short bowel syndrome or participant requires total parenteral nutrition
- •Other protocol-defined inclusion/exclusion criteria apply.
研究组 & 干预措施
Ozanimod Dose Level 1
干预措施: Ozanimod (Drug)
Ozanimod Dose Level 2
干预措施: Ozanimod (Drug)
结局指标
主要结局
Percentage of Participants Who Achieve Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64
时间窗: Week 64
The PCDAI is an instrument that was developed and validated for numerical assessment of disease activity in children and adolescents with CD. The PCDAI consists of the following 11 variables: Abdominal pain, stools per day, wellbeing, weight, height velocity, abdominal tenderness, peri-rectal disease, extra-intestinal manifestation, hematocrit, erythrocyte sedimentation rate and albumin. The category of severity for each index item is assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score =5. For albumin level, the maximum score = 10. The PCDAI score is average of 11 variables scoring range of 0 to 100. Mild disease corresponds to scores of 11 to 30; moderate to severe disease corresponds to scores \> 30. Remission (no disease activity) is defined as a PCDAI score \< 10.
Percentage of Participants Achieving Simple Endoscopic Score for Crohn's Disease (SES-CD) ≤ 2 or SES-CD ≤ 4 Points With no SES-CD Subscore > 1 Point at Week 64
时间窗: Week 64
SES-CD assesses the degree of inflammation on the basis of 4 components: size of ulcers, ulcerated surface, affected surface, and presence of narrowing. Each of these 4 components are assessed in the five segments of the ileum and colon: ileum, right colon, transverse colon, left (descending and sigmoid) colon, and rectum, and is scored on a scale of severity 0 (Normal) to 3 (High). Sub-scores for each segment will be derived by adding component scores within segments. Total SES-CD score is the sum of the sub-scores across the five segments. The sum of each component across all segments ranges from 0 to 15, except for the presence of narrowing where it varies from 0 to 11. The range of the overall SES-CD score is 0-56, with larger scores indicating greater severity of disease.
次要结局
- Change From Baseline in Stool Frequency (SF) Score at Week 64(Baseline and Week 64)
- Absolute Lymphocyte Count at Week 64(Week 64)
- Percentage of Adolescents Who Achieve an Average Daily Abdominal Pain Score ≤ 1 Point and an Average Daily Stool Frequency ≤ 3 Points With Abdominal Pain and Stool Frequency no Worse Than Baseline at Week 64(Week 64)
- Percentage of Participants Who Achieve Pediatric Crohn's Disease Activity Index (PCDAI) < 10 at Week 12 Based on Data as Observed(Week 12)
- Percentage of Participants Who Achieve Simple Endoscopic Score for Crohn's Disease (SES-CD) Decrease From Baseline of ≥ 50% (ER-50) at Week 64(Week 64)
- Percentage of Participants Who Achieve Simple Endoscopic Score for Crohn's Disease (SES-CD) Decrease From Baseline of ≥ 50% (ER-50) at Week 12 Based on Data as Observed(Week 12)
- Percentage of Participants Who Achieve Reduction in Pediatric Crohn's Disease Activity Index (PCDAI) Score ≥ 12.5 and a Total PCDAI Score of < 30 Points at Week 64(Week 64)
- Percentage of Participants Who Achieve Reduction in Pediatric Crohn's Disease Activity Index (PCDAI) Score ≥ 12.5 and a Total PCDAI Score of < 30 Points at Week 12 Based on Data as Observed(Week 12)
- Percentage of Adolescents Who Achieve an Average Daily Abdominal Pain Score ≤ 1 Point and an Average Daily Stool Frequency ≤ 3 Points With Abdominal Pain and Stool Frequency no Worse Than Baseline at Week 12 Based on Data as Observed(Week 12)
- Change From Baseline in Abdominal Pain (AP) Score at Week 64(Baseline and Week 64)
- Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Score < 150 at Week 12 Based on Data as Observed(Week 12)
- Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Score < 150 at Week 64(Week 64)
- Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Reduction From Baseline of ≥ 100 Points or CDAI Score < 150 at Week 64(Week 64)
- Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Reduction From Baseline of ≥ 100 Points or CDAI Score < 150 at Week 12 Based on Data as Observed(Week 12)
- Percentage of Participants Who Achieve a Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64 While Remaining Corticosteroid Free in the Prior 12 Weeks(Week 64)
- Percentage of Adolescents Who Achieve a Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64 While Remaining Corticosteroid Free in the Prior 12 Weeks(Week 64)
- Percentage of Participants Who Achieve a Crohn's Disease Activity Index (CDAI) Score < 150 at Week 64 While Remaining Corticosteroid Free in the Prior 12 Weeks(Week 64)
- Percentage of Participants Achieving Simple Endoscopic Score for Crohn's Disease (SES-CD) ≤ 2 or SES-CD ≤ 4 Points With no SES-CD Subscore > 1 Point at Week 12 Based on Data as Observed(Week 12)
- Steady State Systemic Exposures of Ozanimod at Week 20 and Throughout the Study(Week 20 and up to end of study (Week 64))
- Absolute Lymphocyte Count at Week 12 Based on Data as Observed(Baseline (Day 1) and Week 12)
- Number of Participants With Adverse Events(From first dose (Day 1) and up to Week 12)