Volatilomic Approaches for the Study of CFTR Modulators (VOLATIL-CF)
VOLATIL-CF
1 other identifier
observational
20
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Feb 2023
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 12, 2023
CompletedStudy Start
First participant enrolled
February 6, 2023
CompletedFirst Posted
Study publicly available on registry
February 14, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2024
CompletedMarch 22, 2023
March 1, 2023
12 months
January 12, 2023
March 20, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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 Outcomes (9)
Weight
At 0 day, 7 days, and 1 month of treatment
Sweat test
At 0 day, 7 days, and 1 month of treatment
Induced sputum - microbiology
At 0 day, 7 days, and 1 month of treatment
Induced sputum - immunology
At 0 day, 7 days, and 1 month of treatment
Spirometry
At 0 day, 7 days, and 1 month of treatment
- +4 more secondary outcomes
Study Arms (1)
cystic fibrosis and Kaftrio®
20 children with cystic fibrosis aged 6 to 12 who initiate Kaftrio®
Interventions
Fasting children will be asked to breathe normally through a mouthpiece for the collection and analysis of exhaled breath.
Clinical data will be collected in order to seek correlations with the exhaled breath profile.
Eligibility Criteria
Children with cystic fibrosis aged 6 to 12 who will start Kaftrio® treatment.
You may qualify if:
- Patients with cystic fibrosis initiating Kaftrio® treatment.
- Patients and holders of parental authority not opposing participation in this research.
You may not qualify if:
- Patients deprived of liberty or under guardianship.
- Pregnant or breastfeeding patients.
- Lung transplanted patients.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Hôpital Necker - Enfants malades
Paris, Île-de-France Region, 75015, France
Biospecimen
Induced sputum, Plasma, Urine
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Isabelle SERMET-GAUDELUS, Professor
APHP
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 12, 2023
First Posted
February 14, 2023
Study Start
February 6, 2023
Primary Completion
February 1, 2024
Study Completion
August 1, 2024
Last Updated
March 22, 2023
Record last verified: 2023-03
Data Sharing
- IPD Sharing
- Will not share