Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Cystic Fibrosis
- Sponsor
- Tanja Gonska
- Enrollment
- 18
- Primary Endpoint
- Functional in vivo test: Beta-adrenergic sweat secretion test
- Status
- Completed
- Last Updated
- 8 years ago
Overview
Brief Summary
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.
Investigators
Tanja Gonska
Principal Investigator
The Hospital for Sick Children
Eligibility Criteria
Inclusion Criteria
- •any patient being prescribed ivacaftor
Exclusion Criteria
- •only relates to specific tests
Outcomes
Primary Outcomes
Functional in vivo test: Beta-adrenergic sweat secretion test
Time Frame: 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
Functional in vitro test: Rectal tissue biopsies to measure CFTR function in vitro using Ussing chamber studies and organoids
Time Frame: one study visit
Rectal tissue specimen is being used to measure the response to ivcaftor of individual CF patients in vitro
Functional in vitro test: Collect nasal brushes for CFTR to generate nasal cell cultures for the assessment of ivacaftor response and CFTR function
Time Frame: 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
Functional in vivo Cystic fibrosis transmembrane conductor regulator (CFTR) tests: Nasal Potential difference (NPD) measurements
Time Frame: 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
Secondary Outcomes
- Measurements of the pancreas function using fecal elastase test(4 study visits in 2 years)
- Measurements of the lung clearance index(5 study visits in 1 year)
- Assessment of pulmonary radiological changes using high resolution CT(4 study visits in 2 years)
- Measurements of the pancreas function using serum trypsinogen(4 study visits in 2 years)
- Measurements of the resting energy expenditure(4 study visits in 2 years)
- Measurement of aqua wrinkling(2 study visits in 3 months)
- Assessment of the nutritional status(4 study visits in 2 years)
- Measurement of exhaled fractionated nitrogen oxide (FeNo)(5 study visits in 1 year)
- Assessment of glucose tolerance(4 study visits in 2 years)