A Phase 3, Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 21
- 主要终点
- Safety
研究概览
简要总结
Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice. The purpose of the present study is to provide efficacy and safety data for a new, soluble formulation of liprotamase, Liprotamase Powder for Oral Solution, in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).
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
- 28 Days 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For Part A: males or females ≥7 years of age
- •For Part B: males or females 28 days to <7 years
- •Diagnosis of cystic fibrosis based on presentation, genotype and/or sweat chloride
- •Low fecal elastase
- •Fair-to-good nutritional status
排除标准
- •History or diagnosis of fibrosing colonopathy
- •Distal intestinal obstruction syndrome in 6 months prior to screening
- •Receiving enteral tube feedings
- •Chronic diarrheal illness unrelated to pancreatic insufficiency
- •Liver abnormalities, or liver or lung transplant, or significant bowel resection
研究组 & 干预措施
Part A
Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age
干预措施: Liprotamase Powder for Oral Solution (Drug)
Part B
Liprotamase Powder for Oral Solution in Subjects aged 28 days to <7 years
干预措施: Liprotamase Powder for Oral Solution (Drug)
结局指标
主要结局
Safety
时间窗: 1 week
Number of subjects reporting 1 or more adverse events
次要结局
未报告次要终点