A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Extended-release Tablets n Pediatric Subjects Aged 5 to 17 Years With Active, Mild to Moderate Ulcerative Colitis
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 70
- 试验地点
- 4
- 主要终点
- The primary efficacy endpoint is the proportion of subjects who achieve clinical remission at Day 56, defined as a total Mayo score of ≤ 1 with subscores of 0 for both rectal bleeding and stool frequency, and a subscore of ≤ 1 for endoscopy.
研究概览
简要总结
This is a randomized, double-blind, placebo-controlled, multicenter, study to evaluate the efficacy, safety, and pharmacokinetics (PK) of budesonide extended-release tablets for the induction of remission in pediatric subjects, with active, mild to moderate ulcerative colitis (UC). Subjects will be permitted to continue taking background oral or rectal 5-aminosalicylate (5-ASA) products.
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
- Double (Participant, Investigator)
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
- •Established diagnosis of UC is based on:
- •Clinical history
- •Characteristic endoscopic findings
- •Histopathology results from biopsies
- •Age 5 to 17 years, inclusive, at Screening. Subjects should weigh greater than 13.6 kg at Baseline.
- •Active UC of mild or moderate severity, defined as a total Mayo score between 4 and 10, inclusive, with rectal bleeding subscore of ≥ 1, stool frequency subscore of ≥1 and an endoscopy subscore of ≥
- •If on a background oral or rectal 5-ASA, the dose and formulation have remained unchanged for at least 6 weeks prior to Visit 2 (randomization), Baseline and the subject is willing to remain on the same formulation and dose for the duration of the study.
排除标准
- •Current or prior diagnosis of Crohn's disease or indeterminate colitis.
- •Limited distal proctitis (disease involving only the first 15 centimeters or less proximal to the anal verge.
- •Severe UC, defined as total Mayo score >
- •Not currently in an active phase or flare, defined as a total Mayo score <4, or Mayo score between 4 and 10, but Mayo subscore of 0 for rectal bleeding, stool frequency or endoscopy.
- •Infectious colitis (based on positive microbiologic tests at Screening) or any recent history of infectious colitis (within 30 days prior to Screening).
- •Prior gastrointestinal surgery, except appendectomy or hernia (e.g., inguinal, umbilical).
- •NOTE: Prior cholecystectomy is not exclusionary if more than 1 year prior to Screening.
- •Evidence or history of toxic megacolon or bowel resection.
研究组 & 干预措施
Low Dose Budesonide
干预措施: Low Dose Budesonide (Drug)
High Dose Budesonide
干预措施: High Dose Budesonide (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
The primary efficacy endpoint is the proportion of subjects who achieve clinical remission at Day 56, defined as a total Mayo score of ≤ 1 with subscores of 0 for both rectal bleeding and stool frequency, and a subscore of ≤ 1 for endoscopy.
时间窗: 56 days
The primary efficacy endpoint is the proportion of subjects who achieve clinical remission at Day 56, defined as a total Mayo score of ≤ 1 with subscores of 0 for both rectal bleeding and stool frequency, and a subscore of ≤ 1 for endoscopy. The Mayo score is a composite index score based on 4 components: stool frequency (subject reported), rectal bleeding (subject reported), findings of endoscopy, and the physician's rating of disease activity. Each component is scored from 0 to 3, with total scores range from 0 to 12 with higher scores indicating more severe disease.
次要结局
- Proportion of subjects who achieve a Mayo rectal bleeding subscale score of 0 at Day 56.(56 days)
- Proportion of subjects who achieve a Mayo endoscopy subscale score of 0 or 1 at Day 56.(56 days)
- Proportion of subjects who achieve a Mayo stool frequency subscale score of 0 at Day 56.(56 days)