Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor
KAF-BIOTA
Monitoring of the Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor: Study of the Pulmonary and Gut Microbiota and Inflammation
1 other identifier
interventional
253
1 country
15
Brief Summary
Cystic fibrosis is a systemic disease, which affects in particular the respiratory and digestive systems of patients, sites of chronic inflammation. A new combination of elexacaftor/tezacaftor/ivacaftor has proven its efficacy for the treatment of patients aged 12 years and over with two F508del mutations or a so-called "minimal function" mutation associated with one F508del mutation. European marketing authorization was obtained in August 2020 and access in France should therefore arrive soon. Given that this treatment targets new mutations and that the efficacy seems greater than with LUM/IVA, it is important to assess its impact on the microbiota and the pulmonary and digestive inflammation of patients. It is therefore a question of taking advantage of the experience of the Lum-Iva-Biota cohort, and the validated and operational sample circuit established in the various participating centers to set up a biological collection for the collection and storage of sputum and stools of patients during the first year of treatment with elexacaftor/tezacaftor/ivacaftor, in order to study the effect of treatment on the lung and digestive microbiota/mycobiota and inflammation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for not_applicable
Started Sep 2021
Typical duration for not_applicable
15 active sites
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
Study Start
First participant enrolled
September 13, 2021
CompletedFirst Submitted
Initial submission to the registry
June 27, 2023
CompletedFirst Posted
Study publicly available on registry
July 10, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 13, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
September 13, 2024
CompletedJuly 10, 2023
July 1, 2023
2 years
June 27, 2023
July 7, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
composition of the digestive bacterial microbiota
composition of the digestive, bacterial microbiota, at 12 months of treatment
12 months after baseline (treatment initiation)
Secondary Outcomes (7)
composition of the pulmonary bacterial microbiota
12 months after baseline (treatment initiation)
composition of the pulmonary bacterial microbiota
at baseline (treatment initiation)
composition of the digestive fungal microbiota
12 months after baseline (treatment initiation)
composition of the digestive fungal microbiota
at baseline (treatment initiation)
composition of the pulmonary fungal microbiota
12 months after baseline (treatment initiation)
- +2 more secondary outcomes
Study Arms (1)
patients with cystic fibrosis
EXPERIMENTALpatients with cystic fibrosis before and one year after the start of treatment with elexacaftor/tezacaftor/ivacaftor
Interventions
collection of sputum, stool and blood samples at baseline, 6 months and 1 year after baseline
Eligibility Criteria
You may qualify if:
- To have cystic fibrosis (sweat test \> 60 mmol/l);
- Carrier of at least one DeltaF508 mutation;
- Be followed in the current care by a participant in the CRCM study;
- Be of the age specified in the marketing authorization in force;
- Person affiliated or beneficiary of a social security scheme;
- Consent obtained by the patient (for adult patients) or the holders of parental authority (for minor patients) before any examination required by the research and oral and/or written consent by the participant (depending on his or her age) .
- Patient agreeing to take part in cohort follow-up studies of patients treated with elexacaftor/tezacaftor/ivacaftor, included in the French cystic fibrosis register (cf. Study by Pr BURGEL and/or MODUL CF).
You may not qualify if:
- Start of treatment with elexacaftor/tezacaftor/ivacaftor as part of a therapeutic trial.
- Patient already on CFTR modulator (including lumacaftor/ivacaftor)
- Vulnerable people (pregnant woman, person under guardianship/curators)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
CHU de Bordeaux - CRCM pédiatrique
Bordeaux, France
CHU de Grenoble Alpes CRCM pédiatrique
Grenoble, France
CHRU de Lille CRCM Pédiatrique
Lille, France
CHU de Limoges CRCM Limousin
Limoges, France
Hospices Civils de Lyon Service de pédiatrie, allergologie et mucoviscidose
Lyon, France
AP-HM CRCM pédiatrique
Marseille, France
CHU de Montpellier
Montpellier, France
CHU de Nancy
Nancy, France
CHU de Nice
Nice, France
AP-HP CRCM Robert debré
Paris, France
AP-PH Hopital Cochin service de pédiatrie
Paris, France
APHP Hopital Necker
Paris, France
Fondation Ildys, Roscoff Centre Hélio Marin - Clinique "Mucoviscidose"
Roscoff, France
CHU de Rouen
Rouen, France
CHU de Toulouse
Toulouse, France
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Raphaël Enaud, MDPhD
University Hospital, Bordeaux
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- DIAGNOSTIC
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 27, 2023
First Posted
July 10, 2023
Study Start
September 13, 2021
Primary Completion
September 13, 2023
Study Completion
September 13, 2024
Last Updated
July 10, 2023
Record last verified: 2023-07
Data Sharing
- IPD Sharing
- Will not share