Cystic FibrosisG551DNCT03390985
Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor
An observational study of Cystic Fibrosis, run by Tanja Gonska.
Trial summary
Specific aims: 1. 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 2. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 18 participants
- Where
- No sites listed yet
- Study dates
- 23 Apr 2013to 26 Jul 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- any patient being prescribed ivacaftor
Who cannot join
- only relates to specific tests
About this study
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.
- Conditions
- Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional in vivo Cystic fibrosis transmembrane conductor regulator (CFTR) tests: Nasal Potential difference (NPD) measurements
NPD is measured to assess CFTR function as difference in mV between post and pre-drug start
Measured: 7 study visits over a 2 year period
Functional in vivo test: Beta-adrenergic sweat secretion test
The beta-adrenergic sweat secretion test is measured as ratio of beta-adrenergic/cholinergic sweat secretion as difference between post and pre-drug start
Measured: 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
Rectal tissue specimen is being used to measure the response to ivcaftor of individual CF patients in vitro
Measured: 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
Nasal cultures are used to measure the response to ivcaftor in vitro as difference of the forskolin-induced response of CFTR to ivacaftor
Measured: 2 study visits in 6 months
Secondary outcomes (9)
Measurement of aqua wrinkling
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
Measured: 2 study visits in 3 months
Measurement of exhaled fractionated nitrogen oxide (FeNo)
the fraction of the exhaled NO in % In the expiration of CF patients is being measured pre-and post-drug
Measured: 5 study visits in 1 year
Measurements of the lung clearance index
Multiple breath wash-out technique is being used to measure the lung clearance index (no unit) pre and post-drug
Measured: 5 study visits in 1 year
Measurements of the pancreas function using fecal elastase test
Fecal elastase in stool is determined in microg/g stool pre and post-drug
Measured: 4 study visits in 2 years
Measurements of the pancreas function using serum trypsinogen
Serum trypsinogen is determined in nmol/L pre and post-drug
Measured: 4 study visits in 2 years
Assessment of the nutritional status
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
Measured: 4 study visits in 2 years
Measurements of the resting energy expenditure
The resting energy expenditure is measured using indirect calorimetry in kcal/d pre and post-post
Measured: 4 study visits in 2 years
Assessment of pulmonary radiological changes using high resolution CT
Radiological pulmonary changes are being assessed using high resolution CT pre and post-drug (descriptive and Bhalla score)
Measured: 4 study visits in 2 years
Assessment of glucose tolerance
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
Measured: 4 study visits in 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2018
- Study start23 Apr 2013
- Primary completion26 Jul 2017
- Study completion26 Jul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tanja Gonska
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Tanja Gonska, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03390985
- Sponsor's protocol ID
- 1000036224
- Acronym
- G551D
- Status last verified
- Dec 2017
- First submitted
- 18 Dec 2017
- Last updated
- 5 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor
- ClinicalTrials.gov ID
- NCT03390985
- Acronym / sponsor ID
- G551D / 1000036224
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Tanja GonskaUniversity, hospital or other
- Study start
- 23 Apr 2013 (actual)
- Primary completion
- 26 Jul 2017 (actual)
- Study completion
- 26 Jul 2017 (actual)
- First posted
- 5 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (1 listed)
- any patient being prescribed ivacaftor
Key exclusion criteria (1 listed)
- only relates to specific tests
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
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
9 secondary outcomes
Measurement of aqua wrinkling
Time frame: 2 study visits in 3 months
Measurement of exhaled fractionated nitrogen oxide (FeNo)
Time frame: 5 study visits in 1 year
Measurements of the lung clearance index
Time frame: 5 study visits in 1 year
Measurements of the pancreas function using fecal elastase test
Time frame: 4 study visits in 2 years
Measurements of the pancreas function using serum trypsinogen
Time frame: 4 study visits in 2 years
Assessment of the nutritional status
Time frame: 4 study visits in 2 years
Measurements of the resting energy expenditure
Time frame: 4 study visits in 2 years
Assessment of pulmonary radiological changes using high resolution CT
Time frame: 4 study visits in 2 years
Assessment of glucose tolerance
Time frame: 4 study visits in 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Tanja Gonska.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2018.