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Completed

NCT Number: NCT05726994

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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Necker - Enfants malades

Paris, Île-de-France Region, 75015, France

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

exhaled breath collection

Biological

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

Data Collection

Other

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

Primary outcomes

  1. volatile organic compounds (VOC) profile

    Time frame: 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 outcomes

  1. Weight

    Time frame: At 0 day, 7 days, and 1 month of treatment

    Weight measured at visits

  2. Sweat test

    Time frame: At 0 day, 7 days, and 1 month of treatment

    Sweat test result

  3. Induced sputum - microbiology

    Time frame: At 0 day, 7 days, and 1 month of treatment

    Results of microbiological analysis of induced sputum

  4. Induced sputum - immunology

    Time frame: 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)

  5. Spirometry

    Time frame: At 0 day, 7 days, and 1 month of treatment

    Results of spirometry FVC measurements

  6. Spirometry

    Time frame: At 0 day, 7 days, and 1 month of treatment

    Results of spirometry FEV1 measurements

  7. Spirometry

    Time frame: At 0 day, 7 days, and 1 month of treatment

    Results of spirometry DEM25-75 measurements

  8. Urine

    Time frame: At 0 day and 1 month of treatment

    Biobanking for metabolic study

  9. Blood

    Time frame: At 0 day and 1 month of treatment

    Biobanking for metabolic study

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Collaborators

  • URC-CIC Paris Descartes Necker Cochin

Registry information

Official study title

Volatilomic Approaches for the Study of CFTR Modulators

Acronym: VOLATIL-CF

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Feb 14, 2023
Registry last updated
Jul 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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