Volatilomic Approaches for the Study of CFTR Modulators (VOLATIL-CF)

Study Overview
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Brief Summary
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This study relies on the hypotheses that (1) exhaled breath is intimately correlated to the patient's lung condition and that (2)the composition of exhaled breath , i.e. the VOCs profile, will be significantly modified from the first days of treatment by CFTR modulators in a or pauci/symptomatic patients such as young children under 12 years old. The non-invasive and longitudinal collection and analysis of exhaled breath may reveal modifications in signaling pathways impacted by these treatments on the very short term. This study is a single-center pilot study.

Detailed Description
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This is a single-center prospective cohort study that plans to include 20 children with cystic fibrosis aged 6 to 12 years old who will initiate Kaftrio® in early 2023. The children will be monitored for one month; three visits are planned as part of routine care (before initiation of treatment, in the course of the first week and after one month of treatment) during which exhaled breath collection and analysis will also be performed. Access to clinical data collected throughout routine follow-up of these children (analysis of induced sputum, urine and blood, sweat test, respiratory function tests) will be granted upon patient/parent authorization.

Official Title
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Volatilomic Approaches for the Study of CFTR Modulators

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Biological: exhaled breath collection
* Other: Data collection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with cystic fibrosis initiating Kaftrio® treatment. Patients and holders of parental authority not opposing participation in this research. Patients affiliated to a Health Insurance system or beneficiaries. Exclusion Criteria Patients deprived of liberty or under guardianship. Pregnant or breastfeeding patients. Lung transplanted patients.

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| cystic fibrosis and Kaftrio®<br>20 children with cystic fibrosis aged 6 to 12 who initiate Kaftrio® | Biological: exhaled breath collection<br>* Fasting children will be asked to breathe normally through a mouthpiece for the collection and analysis of exhaled breath.<br>Other: Data collection<br>* Clinical data will be collected in order to seek correlations with the exhaled breath profile.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| volatile organic compounds (VOC) profile | identification of VOCs in exhaled breath with a significant variation between 0 day, 7 days, and/or 1 month of treatment | At 0 day, 7 days, and 1 month of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Weight | Weight measured at visits | At 0 day, 7 days, and 1 month of treatment | 
| Sweat test | Sweat test result | At 0 day, 7 days, and 1 month of treatment | 
| Induced sputum - microbiology | Results of microbiological analysis of induced sputum | At 0 day, 7 days, and 1 month of treatment | 
| Induced sputum - immunology | Results of inflammatory markers analysis of induced sputum (neutrophil elastase, IL-8, IL-1b, IL-6) | At 0 day, 7 days, and 1 month of treatment | 
| Spirometry | Results of spirometry FVC measurements | At 0 day, 7 days, and 1 month of treatment | 
| Spirometry | Results of spirometry FEV1 measurements | At 0 day, 7 days, and 1 month of treatment | 
| Spirometry | Results of spirometry DEM25-75 measurements | At 0 day, 7 days, and 1 month of treatment | 
| Urine | Biobanking for metabolic study | At 0 day and 1 month of treatment | 
| Blood | Biobanking for metabolic study | At 0 day and 1 month of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, volatile organic compounds (VOC), exhaled breath, CFTR modulators