Group 1: Healthy ControlsNCT04391322
Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI
An observational study of Group 1: Healthy Controls and Group 2: Stable CF, run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
This is a prospective, longitudinal observational study conducted at The Hospital for Sick Children (SickKids). The purpose of this study is to evaluate the ability of MRI to detect changes in lung structure and function in CF patients receiving CFTR-modulator therapy. Read more about this study
- Who is it for?
- Ages 4 to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Group 1: Healthy Controls, Group 2: Stable CF, Group 3: CF Participants Anticipated to Receive CFTR-modulator Therapy
- How many people
- 86 participants
- Where
- 1 siteCanada
- Study dates
- 20 Oct 2020to 30 Jun 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 4 to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: This study includes four populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease and Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods
Who may be able to join
- Participants must be greater than or equal to 4 years of age
- Informed consent by patient or parent/guardian consent and participant assent when appropriate.
- Able to perform reproducible spirometry and achieve a breath hold duration sufficient for MRI acquisition
Who cannot join
- Medical instability that would preclude the ability to undergo the required investigations
- FEV1 % predicted < 40%
- Severe claustrophobia
- Does not meet MRI screening criteria
- Cough within the past 3 days prior to study visit
- Usage of oral antibiotics within 3 weeks prior to study visit
Trial locations and contacts
1 site in 1 country.
About this study
This is a prospective, longitudinal observational study conducted at The Hospital for Sick Children (SickKids). The purpose of this study is to evaluate the ability of MRI to detect changes in lung structure and function in CF patients receiving CFTR-modulator therapy. Participants who meet eligibility criteria and consent to the study will undergo study procedures which include spirometry and body plethysmography (optional), multiple breath washout testing, MRI and measurement of quality of life. The study will consist of three to six study visits for Group 1, 2 and 3: baseline, one month (±1 week) and 6 month (±2 week) and if they re-consent then 3 additional visits of 1 year (±6 months). (2 years (±6 months) and 3 years (±6 months) follow-up. Some participants in Group 2 may transition to Group 3.
This study includes three populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease , Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods.
- Conditions
- Group 1: Healthy Controls, Group 2: Stable CF, Group 3: CF Participants Anticipated to Receive CFTR-modulator Therapy, Group 4: 4-8 yo CF Participants Starting Triple Combination Modulator Therapy
- Keywords
- CFTR modulator treatment, xenon MRI, Lung
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Healthy participants without lung disease | |
| Group 2 | Participants with stable cystic fibrosis | |
| Group 3 | Participants with cystic fibrosis, anticipated to receive treatment with CFTR-modulator therapy. Please note: treatment is determined by your physician as part of your normal therapy plan. | |
| Group 4 | Participants with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reproducibility
Establish the interscan reproducibility of fractional ventilation maps and 1H MRI over three and up to six visits in healthy volunteers and in patients with stable CF lung disease and compare to conventional spirometry and LCI.
Measured: 36 months
Correlation
Correlate changes in lung structure and function detected by MRI imaging with clinical outcomes including FEV1 rate of decline, frequency of pulmonary exacerbations, days of IV antibiotics and hospital days to treat pulmonary exacerbation and change in health-related quality of life measures (CFQ-R scores and CF-QUEST) in the setting of patients with stable CF lung disease and those receiving CFTR-modulator therapy.
Measured: 12 months
Responsiveness
Assess responsiveness of MRI to treatment effects over three and up to six visits visits in CF patients receiving CFTR-modulator therapy and compare to conventional breathing tests
Measured: 36 months
Younger cohort
Extension of above objectives originally only explored in children ages 8-18 years old, to younger cohort of CF participants ages 4-8 years old at baseline prior to initiation of CFTR modulator therapy, as well as at 12 and 24 months after therapy initiation.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2020
- Study start20 Oct 2020
- Primary completion30 Jun 2026 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Group 1: Healthy ControlsStudies of the same condition, open and under way.
- More trials run by The Hospital for Sick ChildrenThe sponsor's other registered studies.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 86 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Giles Santyr, The Hospital for Sick Children
- Investigators
- Giles Santyr, PhD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04391322
- Sponsor's protocol ID
- 1000063021
- Status last verified
- Feb 2026
- First submitted
- 12 May 2020
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Data will be shared between the two participating sites in the study. A data transfer agreement has been implemented to enable this transfer smoothly.
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI
- ClinicalTrials.gov ID
- NCT04391322
- Acronym / sponsor ID
- 1000063021
- Conditions
- Group 1: Healthy Controls, Group 2: Stable CF, Group 3: CF Participants Anticipated to Receive CFTR-modulator Therapy, Group 4: 4-8 yo CF Participants Starting Triple Combination Modulator Therapy
- Keywords
- CFTR modulator treatment, xenon MRI, Lung
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 86 participants (estimated)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- 20 Oct 2020 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 18 May 2020 (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.
This is a prospective, longitudinal observational study conducted at The Hospital for Sick Children (SickKids). The purpose of this study is to evaluate the ability of MRI to detect changes in lung structure and function in CF patients receiving CFTR-modulator therapy. Participants who meet eligibility criteria and consent to the study will undergo study procedures which include spirometry and body plethysmography (optional), multiple breath washout testing, MRI and measurement of quality of life. The study will consist of three to six study visits for Group 1, 2 and 3: baseline, one month (±1 week) and 6 month (±2 week) and if they re-consent then 3 additional visits of 1 year (±6 months). (2 years (±6 months) and 3 years (±6 months) follow-up. Some participants in Group 2 may transition to Group 3.
This study includes three populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease , Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | –Healthy participants without lung disease |
| Group 2 | – | –Participants with stable cystic fibrosis |
| Group 3 | – | –Participants with cystic fibrosis, anticipated to receive treatment with CFTR-modulator therapy. Please note: treatment is determined by your physician as part of your normal therapy plan. |
| Group 4 | – | –Participants with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods. |
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- This study includes four populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease and Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods
Key inclusion criteria (3 listed)
- Participants must be greater than or equal to 4 years of age
- Informed consent by patient or parent/guardian consent and participant assent when appropriate.
- Able to perform reproducible spirometry and achieve a breath hold duration sufficient for MRI acquisition
Key exclusion criteria (6 listed)
- Medical instability that would preclude the ability to undergo the required investigations
- FEV1 % predicted < 40%
- Severe claustrophobia
- Does not meet MRI screening criteria
- Cough within the past 3 days prior to study visit
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
Reproducibility
Time frame: 36 months
Establish the interscan reproducibility of fractional ventilation maps and 1H MRI over three and up to six visits in healthy volunteers and in patients with stable CF lung disease and compare to conventional spirometry and LCI.
Correlation
Time frame: 12 months
Correlate changes in lung structure and function detected by MRI imaging with clinical outcomes including FEV1 rate of decline, frequency of pulmonary exacerbations, days of IV antibiotics and hospital days to treat pulmonary exacerbation and change in health-related quality of life measures (CFQ-R scores and CF-QUEST) in the setting of patients with stable CF lung disease and those receiving CFTR-modulator therapy.
Responsiveness
Time frame: 36 months
Assess responsiveness of MRI to treatment effects over three and up to six visits visits in CF patients receiving CFTR-modulator therapy and compare to conventional breathing tests
Younger cohort
Time frame: 24 months
Extension of above objectives originally only explored in children ages 8-18 years old, to younger cohort of CF participants ages 4-8 years old at baseline prior to initiation of CFTR modulator therapy, as well as at 12 and 24 months after therapy initiation.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
- Individual participant dataSharing planned: Yes. Data will be shared between the two participating sites in the study. A data transfer agreement has been implemented to enable this transfer smoothly.
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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.
Giles Santyr, PhD principal investigator
The Hospital for Sick Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Group 1: Healthy Controls and Group 2: Stable CF, run by The Hospital for Sick Children at 1 site in Canada.
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 3 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
This study is still under way. Results are not available in the source record yet.
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?
Reproducibility (time frame: 36 months). 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 27 Feb 2026.