A Phase 2, Randomized, Double Blind, Parallel Dose Ranging Study of Oral TheraCLEC™ - Total in Cystic Fibrosis Subjects With Exocrine Pancreatic Insufficiency
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 129
- 试验地点
- 1
- 主要终点
- Change from baseline up to 19 days in Coefficient of fat absorption (CFA)
研究概览
简要总结
The purpose of this study is to determine the most effective dose of a new enzyme therapy on oral nutrient absorption in cystic fibrosis (CF) subjects with exocrine pancreatic insufficiency.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Pancreatic insufficiency (PI) documented by fecal elastase <100 mcg/gram measured at screening
- •Diagnosis of CF based upon the following criteria:
- •two clinical features consistent with CF AND
- •either genotype with two identifiable mutations consistent with CF OR
- •sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis
- •Forced Expiratory Volume Over One Second (FEV1) ≥30% predicted
- •Clinically stable with no evidence of acute upper or lower respiratory tract infection
排除标准
- •Pregnancy, breastfeeding or of childbearing potential and/or not willing to use accepted methods of birth control during enrollment in the study
- •History of fibrosing colonopathy
- •History of liver transplant or lung transplant
- •Unable to discontinue enteral tube feedings during the study
- •Subject weight <40 kg
- •Known hypersensitivity to food additives
- •Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening
- •Unable to discontinue the use of pancreatic enzymes for a 72-hour period or any other condition that the investigator believes would interfere with the intent of this study or would make study participation not in the best interest of the subject
研究组 & 干预措施
Low Liprotamase Dose
Liprotamase in a fixed combination of lipase (5,000 units), protease (5,000 units) and amylase (750 units) administered orally (one Size 5 capsule of liprotamase and five Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days
干预措施: Liprotamase (Drug)
Low Liprotamase Dose
Liprotamase in a fixed combination of lipase (5,000 units), protease (5,000 units) and amylase (750 units) administered orally (one Size 5 capsule of liprotamase and five Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days
干预措施: Placebo (Drug)
Mid Liprotamase Dose
Liprotamase in a fixed combination of lipase (25,000 units), protease (25,000 units) and amylase (3,750 units) administered orally (one Size 5 capsule of liprotamase, one Size 2 capsule of liprotamase, and four Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days
干预措施: Liprotamase (Drug)
Mid Liprotamase Dose
Liprotamase in a fixed combination of lipase (25,000 units), protease (25,000 units) and amylase (3,750 units) administered orally (one Size 5 capsule of liprotamase, one Size 2 capsule of liprotamase, and four Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days
干预措施: Placebo (Drug)
High Liprotamase Dose
Liprotamase in a fixed combination of lipase (100,000 units), protease (100,000 units) and amylase (15,000 units) administered orally (one Size 5 capsule of placebo and five Size 2 capsules of liprotamase) with each of three meals and two snacks daily for 28 days
干预措施: Liprotamase (Drug)
High Liprotamase Dose
Liprotamase in a fixed combination of lipase (100,000 units), protease (100,000 units) and amylase (15,000 units) administered orally (one Size 5 capsule of placebo and five Size 2 capsules of liprotamase) with each of three meals and two snacks daily for 28 days
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline up to 19 days in Coefficient of fat absorption (CFA)
时间窗: Baseline, up to 19 days
次要结局
- Change from baseline to 28 days in maximum change in glucose response(Baseline through 28 days)
- Change from baseline up to 19 day in Coefficient of nitrogen absorption (CNA)(Baseline, up to 19 days)
- Change from baseline up to 19 days in stool weight(Baseline, up to 19 days)
- Change from baseline up to 19 days in number of Stools(Baseline, up to 19 days)
- Change from baseline to 29 day endpoint in Cystic Fibrosis Questionnaire (CFQ) scale scores(Baseline, 29 days)