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

Volatilomic Approaches for the Study of CFTR Modulators

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.

Study Overview

• Status: Recruiting
• Conditions: Cystic Fibrosis
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Anticipated): 20

Contacts and Locations

• Study Contact: Name: Charlotte ROY, Doctor; Phone Number: +33 1 44 49 44 92; Email: charlotte.roy@aphp.fr

Study Locations

• France
  • Ile-de-France
    • Paris, Ile-de-France, France, 75015
      • Recruiting
      • Hopital Necker - Enfants Malades

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Children with cystic fibrosis aged 6 to 12 who will start Kaftrio® treatment.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
volatile organic compounds (VOC) profile	identification of VOCs in exhaled breath with a significant variation between 0 day, 7 days, and/or 1 month of treatment	At 0 day, 7 days, and 1 month of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Weight	Weight measured at visits	At 0 day, 7 days, and 1 month of treatment
Sweat test	Sweat test result	At 0 day, 7 days, and 1 month of treatment
Induced sputum - microbiology	Results of microbiological analysis of induced sputum	At 0 day, 7 days, and 1 month of treatment
Induced sputum - immunology	Results of inflammatory markers analysis of induced sputum (neutrophil elastase, IL-8, IL-1b, IL-6)	At 0 day, 7 days, and 1 month of treatment
Spirometry	Results of spirometry FVC measurements	At 0 day, 7 days, and 1 month of treatment
Spirometry	Results of spirometry FEV1 measurements	At 0 day, 7 days, and 1 month of treatment
Spirometry	Results of spirometry DEM25-75 measurements	At 0 day, 7 days, and 1 month of treatment
Urine	Biobanking for metabolic study	At 0 day and 1 month of treatment
Blood	Biobanking for metabolic study	At 0 day and 1 month of treatment

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start (Actual): February 6, 2023
• Primary Completion (Anticipated): February 1, 2024
• Study Completion (Anticipated): August 1, 2024

Study Registration Dates

• First Submitted: January 12, 2023
• First Submitted That Met QC Criteria: February 3, 2023
• First Posted (Actual): February 14, 2023

Study Record Updates

• Last Update Posted (Actual): March 22, 2023
• Last Update Submitted That Met QC Criteria: March 20, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; exhaled breath; CFTR modulators; volatile organic compounds (VOC)
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: APHP221173

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No