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

Assistance Publique - Hôpitaux de Paris

Status

Enrolling

Conditions

Cystic Fibrosis

Treatments

Other: Data collection

Biological: exhaled breath collection

Study type

Observational

Funder types

Other

Identifiers

NCT05726994

APHP221173

Details and patient eligibility

About

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.

Full 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.

Enrollment

20 estimated patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.