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

Sponsor

Assistance Publique - Hôpitaux de Paris (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05726994

Collaborator

(none)

Enrollment

Location

Anticipated Duration (Months)

1.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Biological: exhaled breath collection Other: Data collection

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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

20 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

Official Title:

Volatilomic Approaches for the Study of CFTR Modulators

Anticipated Study Start Date :

Feb 1, 2023

Anticipated Primary Completion Date :

Feb 1, 2024

Anticipated Study Completion Date :

Aug 1, 2024

Arms and Interventions

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

Arm

Intervention/Treatment

cystic fibrosis and Kaftrio®

20 children with cystic fibrosis aged 6 to 12 who initiate Kaftrio®

Biological: exhaled breath collection

Fasting children will be asked to breathe normally through a mouthpiece for the collection and analysis of exhaled breath.

Other: Data collection

Clinical data will be collected in order to seek correlations with the exhaled breath profile.

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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
Spirometry [At 0 day, 7 days, and 1 month of treatment]
Results of spirometry FEV1 measurements
Spirometry [At 0 day, 7 days, and 1 month of treatment]
Results of spirometry DEM25-75 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

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

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.