NCT02135432已完成早期 1 期
A Randomized, Double-Blind, Placebo Controlled Pilot Study to Determine the Safety and Efficacy of Ivacaftor (VX-770) for the Treatment of Chronic Obstructive Pulmonary Disease (The TOPIC Trial)
适应症
干预措施
相关药物
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Change in COPD as Measured by the Sweat Analysis in Each Group
研究概览
简要总结
The study is a randomized, double-blind, placebo-controlled, multiple-dose, pilot study of orally-administered ivacaftor in subjects with chronic obstructive pulmonary disease. Subjects will be administered the study drug ivacaftor 150 mg (or placebo) twice daily (BID).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 40 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or Female age 40-65
- •A clinical diagnosis of COPD as defined by GOLD
- •At Least a 10 pack year smoking history
- •Exhibit symptoms of chronic bronchitis defined by MRC
- •FEV1% predicted ≥ 35% and ≤70% Post Bronchodilator
- •Clinically stable in the last 4 weeks with no evidence of COPD exacerbation
- •Weight of 40 kg-120 kg
- •Willingness to use at least one form of acceptable birth control including abstinence, condom with spermicide, or hormonal contraceptives
- •Willing to monitor blood glucose if known history of diabetes mellitus requiring insulin or medical therapy
排除标准
- •Current Diagnosis of Asthma
- •Daytime use of Oxygen Therapy
- •Documented history of drug abuse within the last year
- •Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 28 days before receiving the first dose of study drug.
- •Cirrhosis or elevated liver transaminases > 3X ULN
- •GFR < 50 estimated by Cockcroft-Gault
- •Any illness of abnormal lab finding that, in the opinion of the investigator or the subject's general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- •Pregnant or Breastfeeding
- •Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice.
- •Uncontrolled Diabetes
- •Excluded medications and foods include the drugs and foods provided in the appendix document.
- •Clinically significant arrhythmias or conduction abnormalities that in the opinion of the investigator affect patient safety have been added as exclusion criteria and criteria for withdrawal.
- •Patients who have not been stable or have been hospitalized in the past 3 months with any clinically significant cardiac conditions.
- •Subjects with history of cancer (current or past, unless remote (>5years))except for localized non-melanomatous skin cancers History of Stroke/CVA History of myocardial infarction/acute coronary syndrome Cardiac Failure NYHC grade III-IV Diabetes Type I Uncontrolled Hypertension Primary or secondary pulmonary hypertension
研究组 & 干预措施
Ivacaftor (VX-770)
Experimental
twice a day administration of Ivacaftor: 150mg
干预措施: Ivacaftor (Drug)
Placebo
Placebo Comparator
matching placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change in COPD as Measured by the Sweat Analysis in Each Group
时间窗: baseline to 2 weeks
sweat analysis is measured by performing a sweat test in each participant. The primary analysis will compare the within group change in sweat chloride before (day 1) and after (day 14) ivacaftor or placebo administration and will be used to test the null hypothesis of no change in sweat chloride using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.
次要结局
- Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.(baseline to 2 weeks)
- Change in COPD as Measured by Nasal Potential Difference(baseline to 2 weeks)
- Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group(baseline to 2 weeks)
研究者
Dr. Steven M Rowe
MD
University of Alabama at Birmingham
研究点 (1)
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