Clinical trial NCT05726994: Volatilomic Approaches for the Study of CFTR Modulators (VOLATIL-CF)
Official title: Volatilomic Approaches for the Study of CFTR Modulators
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
volatile organic compounds (VOC)
exhaled breath
CFTR modulators
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2023-02-14
Enrollment: 20
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with cystic fibrosis aged 6 to 12 who will start Kaftrio® treatment.

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.

Eligibility 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.

Primary outcomes:
measure: volatile organic compounds (VOC) profile
timeFrame: At 0 day, 7 days, and 1 month of treatment
Secondary outcomes:
measure: Weight
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Sweat test
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Induced sputum - microbiology
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Induced sputum - immunology
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Spirometry
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Spirometry
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Spirometry
timeFrame: At 0 day, 7 days, and 1 month of treatment
measure: Urine
timeFrame: At 0 day and 1 month of treatment
measure: Blood
timeFrame: At 0 day and 1 month of treatment
Other outcomes:
Locations:
city: Paris
country: France
facility: Hôpital Necker - Enfants malades
state: Ile-de-France
Study officials:
affiliation: APHP
name: Isabelle SERMET-GAUDELUS, Professor
role: STUDY_DIRECTOR
References: