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

Cystic Fibrosis Clinical Trial

— VOLATIL-CF  

Official title:

Volatilomic Approaches for the Study of CFTR Modulators

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05726994
• Other study ID #: APHP221173
• Status: Recruiting
• Start date: February 6, 2023
• Est. completion date: August 2024

Study information

• Verified date: March 2023
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Charlotte ROY, Doctor
• Phone: +33 1 44 49 44 92
• Email: charlotte.roy[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: August 2024
• Est. primary completion date: February 2024
• Gender: All
• Age group: 2 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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.

Locations

Country	Name	City	State
France	Hôpital Necker - Enfants malades	Paris	Ile-de-France

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Weight	Weight measured at visits	At 0 day, 7 days, and 1 month of treatment
Secondary	Sweat test	Sweat test result	At 0 day, 7 days, and 1 month of treatment
Secondary	Induced sputum - microbiology	Results of microbiological analysis of induced sputum	At 0 day, 7 days, and 1 month of treatment
Secondary	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
Secondary	Spirometry	Results of spirometry FVC measurements	At 0 day, 7 days, and 1 month of treatment
Secondary	Spirometry	Results of spirometry FEV1 measurements	At 0 day, 7 days, and 1 month of treatment
Secondary	Spirometry	Results of spirometry DEM25-75 measurements	At 0 day, 7 days, and 1 month of treatment
Secondary	Urine	Biobanking for metabolic study	At 0 day and 1 month of treatment
Secondary	Blood	Biobanking for metabolic study	At 0 day and 1 month of treatment