A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
2019年3月18日 更新者：Vertex Pharmaceuticals Incorporated
A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise Tolerance in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
This study is designed to evaluate the effect of LUM/IVA on exercise tolerance in subjects with CF, homozygous for the F508del-CFTR mutation.
研究概览
研究类型
介入性
注册 (实际的)
70
阶段
- 第四阶段
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
12年 及以上 (孩子、成人、年长者)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Homozygous for the F508del-CFTR mutation
- Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
- Stable CF disease as judged by the investigator
- Forced expiratory volume in 1 second (FEV1) at least 40% and not greater than 90% of predicted
Exclusion Criteria:
- History of any comorbidity that might confound the results of the study, interfere with the use of cardiopulmonary exercise tests (CPETs) as an assessment, or pose an additional risk in administering study drug to the subject
- Any previous exposure to LUM or IVA
- History of cardiac arrhythmia, ischemic heart disease, congestive heart failure, or other clinically significant cardiac condition, or medical condition requiring chronic use of a beta blocker, non-dihydropyridine calcium channel blocker, or other cardiac medication known to affect exercise tolerance
- History of solid organ or hematological transplantation
- For subjects under 18 years of age at Screening, except those who have had bilateral lens removal, selected findings on a screening ophthalmologic examination will be exclusionary
- Using or expected to require any concomitant medication that is prohibited in this study
- History of alcohol or drug abuse, as deemed by the investigator, in the past year, including but not limited to cannabis, cocaine, and opiates
- Participation in an investigational drug study within 30 days before the Screening Visit
- Pregnant or nursing females; males with a female partner who is pregnant or nursing
- Colonization with organisms associated with a more rapid decline in pulmonary status
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：其他
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：三倍
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
安慰剂比较：Placebo
Placebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
|
|
|
实验性的：LUM/IVA
LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
其他名称：
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
大体时间：Baseline, Week 24
|
CPET was used to assess change in exercise tolerance, as measured by VO2max.
|
Baseline, Week 24
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
大体时间：Baseline, Week 24
|
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Exercise Duration During CPET at Week 24
大体时间：Baseline, Week 24
|
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in VO2max During CPET at Week 24
大体时间：Baseline, Week 24
|
CPET was used to assess change in exercise tolerance, as measured by VO2max.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
大体时间：Baseline, Week 24
|
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
|
Baseline, Week 24
|
|
Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
大体时间：Baseline, Week 24
|
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Functional VO2 Gain at Week 24
大体时间：Baseline, Week 24
|
Baseline, Week 24
|
|
|
Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24
大体时间：Baseline, Week 24
|
Baseline, Week 24
|
|
|
Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
大体时间：Baseline, Week 24
|
Baseline, Week 24
|
|
|
Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
大体时间：Baseline, Week 24
|
Baseline, Week 24
|
|
|
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
大体时间：Baseline, Week 24
|
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
|
Baseline, Week 24
|
|
Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
大体时间：Baseline, Week 24
|
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
大体时间：Baseline, Week 24
|
BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2).
|
Baseline, Week 24
|
|
Relative (Percent) Change From Baseline in BMI at Week 24
大体时间：Baseline, Week 24
|
BMI was defined as weight in kg divided by height in m^2.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
大体时间：Baseline, Week 24
|
The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
|
Baseline, Week 24
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
大体时间：Baseline, Week 24
|
The PHQ-8 is an eight item self-reported measure of depression.
Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day).
Total score is the sum of individual eight items and ranges from 0 to 24, with higher scores indicating more severe depression symptoms.
Total score of 0 to 5 indicates none to minimal depression, 6 to 10 indicates mild depression, 11 to 15 indicates moderate depression, 16 to 20 indicates moderately severe depression and 21 to 24 indicates severe depression.
|
Baseline, Week 24
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
大体时间：Baseline, Week 24
|
The GAD-7 is a seven item, self-reported measurement of GAD severity.
Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day).
Total score is the sum of individual seven items and ranges from 0 to 21, with higher scores indicating more severe anxiety symptoms.
Total score of 0 to 5 indicates none to minimal anxiety, 6 to 10 indicates mild anxiety, 11 to 15 indicates moderate anxiety, 16 to 21 indicates severe anxiety.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24
大体时间：Baseline, Week 24
|
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.
|
Baseline, Week 24
|
|
Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24
大体时间：Baseline, Week 24
|
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Duration of Sleep Time at Week 24
大体时间：Baseline, Week 24
|
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
|
Baseline, Week 24
|
|
Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24
大体时间：Baseline, Week 24
|
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
|
Baseline, Week 24
|
|
Absolute Change From Baseline in Time Above Sedentary Duration at Week 24
大体时间：Baseline, Week 24
|
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
|
Baseline, Week 24
|
|
Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24
大体时间：Baseline, Week 24
|
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
|
Baseline, Week 24
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
大体时间：Day 1 up to Week 28
|
Day 1 up to Week 28
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
出版物和有用的链接
负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2016年9月1日
初级完成 (实际的)
2017年9月1日
研究完成 (实际的)
2017年10月1日
研究注册日期
首次提交
2016年8月15日
首先提交符合 QC 标准的
2016年8月22日
首次发布 (估计)
2016年8月23日
研究记录更新
最后更新发布 (实际的)
2019年6月17日
上次提交的符合 QC 标准的更新
2019年3月18日
最后验证
2019年3月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.
安慰剂的临床试验
-
City of Hope Medical CenterNational Cancer Institute (NCI)完全的造血和淋巴细胞肿瘤 | 骨髓纤维化 | 慢性淋巴细胞白血病 | 缓解期成人急性髓性白血病 | 骨髓增生异常综合症 | 缓解期成人急性淋巴细胞白血病 | 骨髓增殖性肿瘤 | 慢性期慢性粒细胞白血病，BCR-ABL1 阳性 | 成人淋巴母细胞淋巴瘤 | 加速期慢性粒细胞白血病，BCR-ABL1 阳性 | HLA-A*0201 阳性细胞存在 | 巨细胞病毒感染 | 成人霍奇金淋巴瘤 | 成人非霍奇金淋巴瘤美国
-
Luye Pharma Group Ltd.尚未招聘
-
Mila (bMotion Technologies)完全的