Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI

Study Overview
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Brief Summary
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In this study, MRI of the lungs of healthy volunteers and participants with cystic fibrosis (stable and participants initiating CFTR modulator treatment) will be performed over a period of 6 months to determine if lung MRI is able detect structural and functional abnormalities/changes in early cystic fibrosis disease. During the 6 month period, 3 study visits will occur. 70 subjects aged 6 and older will participate in this study. Xenon MRI is a non-invasive imaging technique that does not involve x-rays or ionizing radiation. Rather, this imaging method utilizes the same hardware and software principles that are used for conventional proton MRI of patients in a hospital.

Official Title
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Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI

Conditions
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Cystic Fibrosis

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants must be greater than or equal to 6 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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 100 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Group 1<br>Healthy participants without lung disease | |
| Group 2<br>Participants with stable cystic fibrosis | |
| Group 3<br>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. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Reproducibility | Establish the interscan reproducibility of MRI over three visits in healthy volunteers and in patients with stable CF lung disease and compare to conventional breathing tests | 12 months | 
| Responsiveness | Assess responsiveness of MRI to treatment effects over three visits in CF patients receiving CFTR-modulator therapy and compare to conventional breathing tests | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CFTR modulator treatment, xenon MRI, Lung