A Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, 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
- 入组人数
- 140
- 试验地点
- 50
- 主要终点
- Coefficient of Fat Absorption (CFA)
研究概览
简要总结
Liprotamase powder is a non-porcine, soluble and stable mixture of biotechnology-derived lipase, protease, and amylase digestive enzymes. The purpose of the present study is to to evaluate the non-inferiority of liprotamase compared with porcine-derived, enterically-coated pancreatic enzyme replacement therapy (PERT). The primary efficacy endpoint of the study will be comparative efficacy measured as the change from baseline in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).
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
盲法说明
unblinded to treatment randomization; blinded to primary efficacy variable
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
- •Good disease control with porcine PERT prior to enrollment
- •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 PERT
Individually-optimized dose to be administered orally
干预措施: porcine PERT (Drug)
结局指标
主要结局
Coefficient of Fat Absorption (CFA)
时间窗: 8 weeks
Non-inferiority of Liprotamase to approved porcine PERT
次要结局
- Coefficient of Nitrogen Absorption (CNA)(8 weeks)
- Safety, as measured by number of participants with adverse events or laboratory abnormalities(6 months)