A Phase 3, Randomized, Open-Label, Assessor-Blind, Noninferiority, Active-Comparator 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
- 128
- 试验地点
- 54
- 主要终点
- Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
研究概览
简要总结
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase. The purpose of the present study is to provide additional efficacy and safety data compared to approved, porcine-derived, enterically-coated and encapsulated pancreatic enzyme replacement therapy. The primary efficacy endpoint of the study will be comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).
Liprotamase is stable in stomach and digestive fluids allowing administration in a variety of convenient formulations and with a number of foods without enteric coating.
详细描述
Porcine derived enzymes are used for pancreatic enzyme replacement therapy in patients with cystic fibrosis (CF). Liprotamase is a biotechnology-derived enzyme replacement without enteric coating. This is an open-label, assessor blind, parallel group, multicenter, international trial to evaluate the noninferiority of liprotamase and pancrelipase in CF patients aged ≥7 years with pancreatic insufficiency. Subjects were randomized to liprotamase or pancrelipase, dose-matched to pre-study lipase doses. The lower bound of the 95% confidence interval (CI) for noninferiority was -15% for treatment difference in change from baseline coefficient of fat absorption (CFA).
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
- None
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Cystic Fibrosis based on presentation, genotype and/or sweat chloride
- •Fecal elastase <100 mcg/g stool
- •Minimum Coefficient of Fat (CFA) at screening while on stable PERT therapy
- •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
- •Forced expiratory volume in 1 second (FEV1) <30%
研究组 & 干预措施
Liprotamase
Individually-optimized dose to be administered orally
干预措施: Liprotamase (Drug)
porcine (pig) PERT
Individually-optimized dose to be administered orally
干预措施: porcine (pig) PERT (Drug)
结局指标
主要结局
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
时间窗: Baseline, 7 weeks
The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints
次要结局
- Coefficient of Nitrogen Absorption (CNA)(Baseline, 7 weeks)
- Coefficient of Fat Absorption (CFA)(Baseline, 7 weeks)