Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor (G551D)
2017年12月28日 更新者：Tanja Gonska
Specific aims:
- To elucidate the biological mechanism that leads to pulmonary and nutritional improvement in CF patients following treatment with ivacaftor using advanced techniques to assess changes of the pulmonary and nutritional status
- To examine the relation between the individual response to ivacaftor and the presence of modifier genes associated with CF disease severity,
- To assess altered CFTR function using new available in vivo tests,
- To validate newly developed in vivo sweat tests with well established functional tests,
- To establish correlation between the CFTR response to Vx-770 measured in a new ex vivo method (organoids) and the actual clinical and/or functional response in individual patients,
- To examine response in other CF-specific features such as aqua wrinkling.
- To examine if sleep/activity level changes.
- To establish a biorepository to enable further investigations.
研究概览
地位
完全的
条件
研究类型
观察性的
注册 (实际的)
18
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
6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
取样方法
非概率样本
研究人群
CF patients with any of the following mutations G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and G970R approved by Health Canada to receive ivacaftor.
描述
Inclusion Criteria:
- any patient being prescribed ivacaftor
Exclusion Criteria:
- only relates to specific tests
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
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 |
|---|---|---|
|
Functional in vivo Cystic fibrosis transmembrane conductor regulator (CFTR) tests: Nasal Potential difference (NPD) measurements
大体时间：7 study visits over a 2 year period
|
NPD is measured to assess CFTR function as difference in mV between post and pre-drug start
|
7 study visits over a 2 year period
|
|
Functional in vivo test: Beta-adrenergic sweat secretion test
大体时间：7 study visits over a 2 year period
|
The beta-adrenergic sweat secretion test is measured as ratio of beta-adrenergic/cholinergic sweat secretion as difference between post and pre-drug start
|
7 study visits over a 2 year period
|
|
Functional in vitro test: Rectal tissue biopsies to measure CFTR function in vitro using Ussing chamber studies and organoids
大体时间：one study visit
|
Rectal tissue specimen is being used to measure the response to ivcaftor of individual CF patients in vitro
|
one study visit
|
|
Functional in vitro test: Collect nasal brushes for CFTR to generate nasal cell cultures for the assessment of ivacaftor response and CFTR function
大体时间：2 study visits in 6 months
|
Nasal cultures are used to measure the response to ivcaftor in vitro as difference of the forskolin-induced response of CFTR to ivacaftor
|
2 study visits in 6 months
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Measurement of aqua wrinkling
大体时间：2 study visits in 3 months
|
CF patients response to submerge in water for 5 min to develop a skin phenomenon known as aqua wrinkling is being measured subjectively pre and post-drug
|
2 study visits in 3 months
|
|
Measurement of exhaled fractionated nitrogen oxide (FeNo)
大体时间：5 study visits in 1 year
|
the fraction of the exhaled NO in % In the expiration of CF patients is being measured pre-and post-drug
|
5 study visits in 1 year
|
|
Measurements of the lung clearance index
大体时间：5 study visits in 1 year
|
Multiple breath wash-out technique is being used to measure the lung clearance index (no unit) pre and post-drug
|
5 study visits in 1 year
|
|
Measurements of the pancreas function using fecal elastase test
大体时间：4 study visits in 2 years
|
Fecal elastase in stool is determined in microg/g stool pre and post-drug
|
4 study visits in 2 years
|
|
Measurements of the pancreas function using serum trypsinogen
大体时间：4 study visits in 2 years
|
Serum trypsinogen is determined in nmol/L pre and post-drug
|
4 study visits in 2 years
|
|
Assessment of the nutritional status
大体时间：4 study visits in 2 years
|
Nutritional status is assessed using the BODPOD in kg fat or non-fat mass change or percentage fat or non-fat mass change pre and post-drug
|
4 study visits in 2 years
|
|
Measurements of the resting energy expenditure
大体时间：4 study visits in 2 years
|
The resting energy expenditure is measured using indirect calorimetry in kcal/d pre and post-post
|
4 study visits in 2 years
|
|
Assessment of pulmonary radiological changes using high resolution CT
大体时间：4 study visits in 2 years
|
Radiological pulmonary changes are being assessed using high resolution CT pre and post-drug (descriptive and Bhalla score)
|
4 study visits in 2 years
|
|
Assessment of glucose tolerance
大体时间：4 study visits in 2 years
|
Glucose tolerance is being assessed using the organ glucose tolerance test and glucose levels 2 hrs post in mmol/L will be compared pre and post-drug
|
4 study visits in 2 years
|
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。
赞助
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)
2013年4月23日
初级完成 (实际的)
2017年7月26日
研究完成 (实际的)
2017年7月26日
研究注册日期
首次提交
2017年12月18日
首先提交符合 QC 标准的
2017年12月28日
首次发布 (实际的)
2018年1月5日
研究记录更新
最后更新发布 (实际的)
2018年1月5日
上次提交的符合 QC 标准的更新
2017年12月28日
最后验证
2017年12月1日
更多信息
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