An Open Label, Multicenter Study Evaluating Safety and Tolerability of Creon Micro in Pediatric Subjects From at Least One Month to Less Than Four Years of Age With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 40
- 试验地点
- 9
- 主要终点
- Body Weight
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of Creon micro in children younger than four years with pancreatic exocrine insufficiency.
详细描述
A prospective open-label, multi-center study evaluating safety and tolerability of Creon Micro in pediatric subjects from at least one month to less than four years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis (CF). Referring to the European Medicines Agency (EMEA) pediatric age criteria for the purpose of the study, subjects are categorized as infants and toddlers (< 2 years) and children, (2 years to < 4 years). In total, 40 subjects will be enrolled (20 subjects in each age subgroup). Subjects receive their prescribed pancreatic enzyme supplementation at the individual dose until start of treatment with Creon Micro, if the subjects were on pancreatic enzyme supplementation before start of the study. Subjects will be treated with Creon Micro during the study. The treatment duration is 3 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 4 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children younger than four years with confirmed cystic fibrosis diagnosis and a body weight of at least 2 kilograms
排除标准
- •Ileus or acute abdomen
- •history of fibrosing colonopathy
- •history of distal ileal obstruction without surgery
- •solid organ transplant or surgery affecting the large bowel, other than appendectomy
研究组 & 干预措施
Creon micro, minimicrospheres
干预措施: Pancreatin (Drug)
结局指标
主要结局
Body Weight
时间窗: 3 months
change from baseline at day 84
Height
时间窗: 3 months
change from baseline at day 84
Stool Frequency
时间窗: 3 months
Average daily stool frequency during treatment period: Number of bowel movements per day
Stool Consistency
时间窗: 3 months
Assessment of stool consistency by the caregiver on a daily basis: hard, formed/normal, soft, watery
Subject's Acceptance of Treatment
时间窗: 3 months
Acceptance to Creon Micro. The caregiver should give his/her opinion based on the following scale: very good, good, moderate, and unsatisfactory.
次要结局
- Number of Participants With Clinical Relevant Safety Laboratory Values(3 months)
- Pulse(3 months)
- Number of Participants With Findings During Physical Examination(3 months)
- Number of Subjects With Adverse Events(4 months)