Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, volatile organic compounds (VOC), exhaled breath, CFTR modulators
Brief summary
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
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.
Interventions
BIOLOGICALexhaled breath collection
Fasting children will be asked to breathe normally through a mouthpiece for the collection and analysis of exhaled breath.
OTHERData collection
Clinical data will be collected in order to seek correlations with the exhaled breath profile.
Sponsors
Assistance Publique - Hôpitaux de Paris
URC-CIC Paris Descartes Necker Cochin
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| volatile organic compounds (VOC) profile | At 0 day, 7 days, and 1 month of treatment | identification of VOCs in exhaled breath with a significant variation between 0 day, 7 days, and/or 1 month of treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Weight | At 0 day, 7 days, and 1 month of treatment | Weight measured at visits |
| Sweat test | At 0 day, 7 days, and 1 month of treatment | Sweat test result |
| Induced sputum - microbiology | At 0 day, 7 days, and 1 month of treatment | Results of microbiological analysis of induced sputum |
| Induced sputum - immunology | At 0 day, 7 days, and 1 month of treatment | Results of inflammatory markers analysis of induced sputum (neutrophil elastase, IL-8, IL-1b, IL-6) |
| Spirometry | At 0 day, 7 days, and 1 month of treatment | Results of spirometry FVC measurements |
| Urine | At 0 day and 1 month of treatment | Biobanking for metabolic study |
| Blood | At 0 day and 1 month of treatment | Biobanking for metabolic study |
Countries
France
Contacts
STUDY_DIRECTORIsabelle SERMET-GAUDELUS, Professor
APHP
Outcome results
None listed