NCT00449878已完成3 期
A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 163
- 试验地点
- 1
- 主要终点
- Change from open label baseline to end of 6 day double blind treatment period in Coefficient of Fat Absorption (CFA)
研究概览
简要总结
This is a clinical trial which will evaluate the efficacy of Liprotamase treatment in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (PI).
详细描述
This trial is divided into four distinct periods:
- Baseline Period during which each patient is taken off pancreatic enzyme medications.
- An Open-Label Treatment Period during which all patients will receive ALTU-135 (liprotamase).
- Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo.
- Second Open-Label Treatment Period during which all patients will resume treatment with ALTU-135 (liprotamase).
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
- Triple (Participant, Care Provider, Investigator)
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
- •Females of childbearing potential must be willing to use birth control
- •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
- •Clinically stable with no evidence of acute upper or lower respiratory tract infection
- •PI determined by fecal elastase <100 µg/g stool measured at screening
- •Able to take pancreatic enzyme supplementation in the form of capsules
- •Able to perform the testing (e.g., stool collections) and inpatient stays required for this study, as judged by the Investigator
- •Baseline coefficient of fat absorption (CFA) less than or equal to 80%
排除标准
- •CFA >80% at Baseline
- •Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control during the study
- •History of fibrosing colonopathy
- •History of liver transplant, lung transplant or significant surgical resection of the bowel
- •Any acute or chronic diarrheal illness unrelated to PI
- •Unable to discontinue enteral tube feedings during the study
- •Known hypersensitivity to food additives
- •Inability to consume the diet required by the study, in the judgment of the Investigator
- •Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening
- •Abnormal liver function (except for patients with Gilbert Syndrome)
- •Signs and/or symptoms of liver cirrhosis, portal hypertension or documented liver disease unrelated to CF
- •Distal intestinal obstruction syndrome (DIOS) in the last six months prior to screening
- •Unable to discontinue the use of pancreatic enzymes
- •Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the patient
- •Patient is unlikely to complete the study, as determined by the Investigator
研究组 & 干预措施
Liprotamase
Experimental
Liprotamase is a fixed combination of lipase (32,500 units), protease (25,000 units) and amylase (3,750 units).
Open Label Period: Liprotamase administered orally with each of three meals and two snacks daily for 21 days.
Double Blind Treatment Period: Administered orally with each of three meals and two snacks daily for 6 days.
Second Open Label Period: Administered orally with each of three meals and two snacks daily for 7 days.
干预措施: Liprotamase (Drug)
Placebo
Placebo Comparator
Double Blind Treatment Period: Placebo (microcrystalline cellulose) administered orally with each of three meals and two snacks daily for 6 days.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from open label baseline to end of 6 day double blind treatment period in Coefficient of Fat Absorption (CFA)
时间窗: Open label baseline, end of 6 day double blind treatment period
次要结局
- Change from open label baseline to end of 6 day double blind treatment period in number of stools(Open label baseline, end of 6 day double blind treatment period)
- Change from open label baseline to end of 6 day double blind treatment period in Coefficient of nitrogen absorption (CNA)(Open label baseline, end of 6 day double blind treatment period)
- Change from open label baseline to end of 6 day double blind treatment period in stool weight(Open label baseline, end of 6 day double blind treatment period)
- Change from open label baseline to end of 6 day double blind treatment period in maximum blood glucose(Open label baseline, end of 6 day double blind treatment period)
研究者
研究点 (1)
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