Cystic FibrosisKAF-BIOTANCT05937815
Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor
A study testing Sample collection in people with Cystic Fibrosis, run by University Hospital, Bordeaux at 15 sites in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Sample collection
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 253 participants
- Where
- 15 sitesFrance
- Study dates
- 13 Sep 2021to 13 Sep 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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;
- Start treatment with elexacaftor/tezacaftor/ivacaftor in routine care, according to the indications in the Marketing Authorization at the time of inclusion;
- 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).
Who cannot join
- 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)
Trial locations and contacts
15 sites in 1 country. Contact the study team or a site near you to ask about taking part.
15 sites in 1 country
France15 sites, 15 recruiting
- Recruiting
CHU de Bordeaux - CRCM pédiatrique
Bordeaux
Raphaël ENAUD
- Recruiting
CHU de Grenoble Alpes CRCM pédiatrique
Grenoble
Catherine LLerena
- Recruiting
CHRU de Lille CRCM Pédiatrique
Lille
Nathalie Wizla
- Recruiting
CHU de Limoges CRCM Limousin
Limoges
Alexandra Masson-Rouchaud
- Recruiting
Hospices Civils de Lyon Service de pédiatrie, allergologie et mucoviscidose
Lyon
Philippe Reix
- Recruiting
AP-HM CRCM pédiatrique
Marseille
Jean-Christophe Dubus
- Recruiting
CHU de Montpellier
Montpellier
Raphael CHIRON
- Recruiting
CHU de Nancy
Nancy
Aurélie Tatopoulos
- Recruiting
CHU de Nice
Nice
Sylvie Leroy
- Recruiting
AP-HP CRCM Robert debré
Paris
Michèle GERARDIN
- Recruiting
AP-PH Hopital Cochin service de pédiatrie
Paris
Pierre-Régis BURGEL
- Recruiting
APHP Hopital Necker
Paris
Isabelle Sermet Gaudelus
- Recruiting
Fondation Ildys, Roscoff Centre Hélio Marin - Clinique "Mucoviscidose"
Roscoff
Sophie RAMEL
- Recruiting
CHU de Rouen
Rouen
Hélène Morisse Pradier
- Recruiting
CHU de Toulouse
Toulouse
Marie Mittaine
About this study
Official title: 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
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.
Read the detailed description
Cystic fibrosis is a systemic disease, which affects in particular the respiratory and digestive systems of patients, sites of chronic inflammation. It has also been shown that in these patients, the pulmonary and intestinal microbiota were distinct from those of healthy subjects and that the progression of the disease was associated with alterations in these microbiota. In addition, numerous data suggest the existence of an "intestinal-lung axis" and therefore encourage studying these two organs in parallel and not separately.
The management of cystic fibrosis has been marked in recent years by the appearance of CFTR modulators, in particular the combination lumacaftor/ivacaftor (LUM/IVA) (for patients homozygous F508del). The criteria for evaluating the efficacy of these treatments are based on the change in FEV (forced expiratory volume in 1 second), the number of exacerbations, body mass index or quality of life. However, it is essential to be able to document the effect of these treatments on the lung and digestive microbiota and inflammation. Since 2016, we have set up the national "Lum-Iva-Biota" cohort and have been able to show that the effect of LUM/IVA on the pulmonary microbiota was more marked in patients not previously colonized with P. aeruginosa.
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.
- Conditions
- Cystic Fibrosis
- Being tested
- Sample collection
- Keywords
- elexacaftor/tezacaftor/ivacaftor, lung and digestive microbiota and inflammation
- Medical subject headings
- Cystic Fibrosis, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sample collectionProcedure or surgery
collection of sputum, stool and blood samples at baseline, 6 months and 1 year after baseline
| Study group | Role | What they receive |
|---|---|---|
| patients with cystic fibrosis | Experimental | patients with cystic fibrosis before and one year after the start of treatment with elexacaftor/tezacaftor/ivacaftor Receives: Procedure: Sample collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
composition of the digestive bacterial microbiota
composition of the digestive, bacterial microbiota, at 12 months of treatment
Measured: 12 months after baseline (treatment initiation)
Secondary outcomes (7)
composition of the pulmonary bacterial microbiota
composition of the pulmonary bacterialmicrobiota, at 12 months of treatment
Measured: 12 months after baseline (treatment initiation)
composition of the pulmonary bacterial microbiota
composition of the pulmonary bacterialmicrobiota at baseline
Measured: at baseline (treatment initiation)
composition of the digestive fungal microbiota
composition of the digestive fungal microbiota, at 12 months of treatment
Measured: 12 months after baseline (treatment initiation)
composition of the digestive fungal microbiota
composition of the digestive fungal microbiota at baseline
Measured: at baseline (treatment initiation)
composition of the pulmonary fungal microbiota
composition of the pulmonary fungal microbiota, at 12 months of treatment
Measured: 12 months after baseline (treatment initiation)
composition of the pulmonary fungal microbiota
composition of the pulmonary fungal microbiota at baseline
Measured: at baseline (treatment initiation)
composition of the digestive, bacterial microbiota
composition of the digestive, bacterial microbiota at baseline
Measured: at baseline (treatment initiation)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2023
- Study start13 Sep 2021
- Primary completion13 Sep 2026 (estimated)
- Study completion13 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University Hospital, BordeauxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 253 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Raphaël Enaud, MDPhD, Principal investigator, University Hospital, Bordeaux
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05937815
- Sponsor's protocol ID
- CHUBX 2021/14
- Acronym
- KAF-BIOTA
- Status last verified
- Jan 2026
- First submitted
- 27 Jun 2023
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05937815
- Acronym / sponsor ID
- KAF-BIOTA / CHUBX 2021/14
- Conditions
- Cystic Fibrosis
- Keywords
- elexacaftor/tezacaftor/ivacaftor, lung and digestive microbiota and inflammation
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 253 participants (estimated)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Study start
- 13 Sep 2021 (actual)
- Primary completion
- 13 Sep 2026 (estimated)
- Study completion
- 13 Sep 2026 (estimated)
- First posted
- 10 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Cystic fibrosis is a systemic disease, which affects in particular the respiratory and digestive systems of patients, sites of chronic inflammation. It has also been shown that in these patients, the pulmonary and intestinal microbiota were distinct from those of healthy subjects and that the progression of the disease was associated with alterations in these microbiota. In addition, numerous data suggest the existence of an "intestinal-lung axis" and therefore encourage studying these two organs in parallel and not separately.
The management of cystic fibrosis has been marked in recent years by the appearance of CFTR modulators, in particular the combination lumacaftor/ivacaftor (LUM/IVA) (for patients homozygous F508del). The criteria for evaluating the efficacy of these treatments are based on the change in FEV (forced expiratory volume in 1 second), the number of exacerbations, body mass index or quality of life. However, it is essential to be able to document the effect of these treatments on the lung and digestive microbiota and inflammation. Since 2016, we have set up the national "Lum-Iva-Biota" cohort and have been able to show that the effect of LUM/IVA on the pulmonary microbiota was more marked in patients not previously colonized with P. aeruginosa.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with cystic fibrosis | Experimental | Procedure: Sample collectionpatients with cystic fibrosis before and one year after the start of treatment with elexacaftor/tezacaftor/ivacaftor |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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;
- Start treatment with elexacaftor/tezacaftor/ivacaftor in routine care, according to the indications in the Marketing Authorization at the time of inclusion;
- Be of the age specified in the marketing authorization in force;
Key exclusion criteria (3 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sample collectioncollection of sputum, stool and blood samples at baseline, 6 months and 1 year after baseline | Procedure or surgery | patients with cystic fibrosis |
Primary outcome
composition of the digestive bacterial microbiota
Time frame: 12 months after baseline (treatment initiation)
composition of the digestive, bacterial microbiota, at 12 months of treatment
7 secondary outcomes
composition of the pulmonary bacterial microbiota
Time frame: 12 months after baseline (treatment initiation)
composition of the pulmonary bacterial microbiota
Time frame: at baseline (treatment initiation)
composition of the digestive fungal microbiota
Time frame: 12 months after baseline (treatment initiation)
composition of the digestive fungal microbiota
Time frame: at baseline (treatment initiation)
composition of the pulmonary fungal microbiota
Time frame: 12 months after baseline (treatment initiation)
composition of the pulmonary fungal microbiota
Time frame: at baseline (treatment initiation)
composition of the digestive, bacterial microbiota
Time frame: at baseline (treatment initiation)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Raphaël Enaud, MDPhD principal investigator
University Hospital, Bordeaux
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Sample collection in people with Cystic Fibrosis, run by University Hospital, Bordeaux at 15 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Aurore Capelli, PhD (0557820877, aurore.capelli@chu-bordeaux.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
composition of the digestive bacterial microbiota (time frame: 12 months after baseline (treatment initiation)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.