A Phase 3, Multicenter, Randomized, Double-blind Study to Determine the Safety and Efficacy of MMX Mesalamine/Mesalazine in Pediatric Subjects With Mild to Moderate Ulcerative Colitis, in Both Acute and Maintenance Phases
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 107
- 试验地点
- 33
- 主要终点
- Number of Participants With Clinical Response During Double-Blind Acute Phase at Week 8
研究概览
简要总结
To assess clinical response to MMX mesalamine/mesalazine between a low and high dose in children and adolescents aged 5-17 years with mild to moderate Ulcerative Colitis (UC) or who are in remission.
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
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative [LAR]) informed consent or assent as applicable to participate in the study.
- •Subject's parent/LAR demonstrates an understanding, ability, and willingness to fully comply with study procedures and restrictions.
- •Male and female children and adolescents aged 5-17 years, inclusive.
- •Body weight 18-90kg.
- •Male, or non-pregnant, non-lactating female who agrees to comply with any applicable contraceptive requirements of the protocol or females of non-childbearing potential.
- •Diagnosed with mild to moderate UC, established by sigmoidoscopy or colonoscopy with compatible histology. Screened subjects may also have an unconfirmed diagnosis of mild to moderate UC; however the diagnosis of mild to moderate UC must have been established by sigmoidoscopy or colonoscopy with compatible histology prior to baseline visit.
- •Subject is able to swallow the investigational product whole.
- •Double-blind Acute Phase:
- •Partial UC-DAI score ≥2 (a combined rectal bleeding and stool frequency score ≥1 and PGA=1 or 2) at the Baseline Visit, for which 5-ASA would be used as part of normal treatment.
- •If the subject is on 5-ASA treatment prior to study entry, then the dose must be stable. Stable therapy is defined as no change in dose, or no initiation of 5-ASA, from the onset of the current acute flare through discontinuation of therapy (required at the Baseline Visit).
- •Double-blind Maintenance Phase:
- •Partial UC-DAI ≤1 (rectal bleeding=0, stool frequency ≤1, and PGA=0) at the Baseline Visit.
排除标准
- •Severe UC (defined by PGA=3).
- •Crohn's disease, bleeding disorders, active peptic ulcer disease, or UC known to be confined to the rectum (isolated rectal proctitis).
- •Asthma, only if known to be 5 ASA sensitive.
- •Positive stool culture for enteric pathogens (including Salmonella, Shigella, Yersinia, Aeromonas, Plesiomonas, or Campylobacter). Clostridium difficile toxin, ova, or parasites present.
- •Systemic or rectal corticosteroid use within 4 weeks prior to the Screening Visit. Topical, intranasal, or inhaled use is not exclusionary.
- •Immunomodulator (6-mercaptopurine, azathioprine) use within 6 weeks prior to the Screening Visit.
- •History of biologic (eg, anti-tumor necrosis factor agents, integrin receptor antagonists) use at any time.
- •Antibiotic use within 7 days prior to the Screening Visit.
- •Any anti-inflammatory drugs, not including 5-ASA treatment but including non-steroidal anti-inflammatory drugs such as aspirin, COX-2 inhibitors or ibuprofen, within 7 days prior to the Screening Visit unless used at over-the-counter levels for <3 days. However, prophylactic use of a stable dose of aspirin up to 325mg/day for cardiac disease is permitted.
- •Prebiotic/probiotic use within 7 days prior to the Screening Visit. Yogurt products are permitted.
研究组 & 干预措施
MMX Mesalamine/Mesalazine (Low Dose)
Once daily, tablets - the amount depends on the participants weight; 900 milligram per day (mg/day) for participants weighing 18 kg to less than or equal to (<=) 23 kilograms (kg); 1200 mg/day for participants weighing greater than (>) 23 kg to <= 35 kg; 1800 mg/day for participants weighing > 35 kg to <= 50 kg; 2400 mg/day for participants weighing > 50 kg to <= 90 kg.
干预措施: MMX Mesalamine/Mesalazine (Low Dose) (Drug)
MMX Mesalamine/Mesalazine (High Dose)
Once daily, tablets - the amount depends on the participants weight;1800 mg/day for participants weighing 18 kg to <= 23 kg; 2400 mg/day for participants weighing > 23 kg to <= 35 kg; 3600 mg/day for participants weighing > 35 kg to <= 50 kg; 4800 mg/day for participants weighing > 50 kg to <= 90 kg.
干预措施: MMX Mesalamine/Mesalazine (High Dose) (Drug)
结局指标
主要结局
Number of Participants With Clinical Response During Double-Blind Acute Phase at Week 8
时间窗: Week 8
Clinical response was defined as partial ulcerative colitis disease activity index (UC-DAI) score \< or =1 with rectal bleeding = 0, stool frequency \< or =1, and physician's global assessment (PGA = 0). Number of participants with clinical response were reported.
Number of Participants With Clinical Response During Double-blind Maintenance Phase at Week 26
时间窗: Week 26
Clinical response was defined as partial UC-DAI \<=1 with (rectal bleeding = 0, stool frequency \< or =1, and PGA = 0). Number of participants who had maintained clinical response were reported.
次要结局
- Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Central Reading(Week 8)
- Number of Participants With Remission at Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-Blind Maintenance Phase at Week 26(Week 26)
- Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Local Reading(Week 26)
- Number of Participants With Improvement in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-blind Acute Phase at Week 8(Week 8)
- Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Central Reading(Week 26)
- Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Local Reading(Week 8)
- Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase(Baseline to Week 8)
- Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase(Baseline, Week 13, and Week 26)