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

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Biological: exhaled breath collection; Other: Data collection

• Registration Number: NCT05726994
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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.

Study Timeline

• Study First Submitted: 2023-01-12
• Study First Submitted QC: 2023-02-03
• Study Start: 2023-02-06
• Study First Posted: 2023-02-14
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-20
• Last Update Posted: 2023-03-22
• Primary Completion: 2024-02
• Study Completion: 2024-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
cystic fibrosis and Kaftrio®	Data collection	20 children with cystic fibrosis aged 6 to 12 who initiate Kaftrio®
cystic fibrosis and Kaftrio®	exhaled breath collection	20 children with cystic fibrosis aged 6 to 12 who initiate Kaftrio®

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Weight	At 0 day, 7 days, and 1 month of treatment	Weight measured at visits
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)
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
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

Trial Locations

Locations (1)

Hôpital Necker - Enfants malades; 🇫🇷 Paris, Ile-de-France, France