Exploring the effects of a new cystic fibrosis treatment on lung and gut health

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

This study is testing a new combination therapy for cystic fibrosis to see how it affects lung and gut health in people aged 12 and older with certain genetic mutations.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 253 (estimated)
• Ages: 6 Years to 17 Years
• Sex: All
• Sponsor: University Hospital, Bordeaux (Academic / other)
• Locations: 15 sites (Bordeaux and 14 other locations)
• Trial ID: NCT05937815 on ClinicalTrials.gov

What this trial studies

This study investigates the impact of the combination therapy elexacaftor/tezacaftor/ivacaftor on the microbiota and inflammation in cystic fibrosis patients. It focuses on individuals aged 12 and older who have specific genetic mutations associated with cystic fibrosis. By collecting and analyzing sputum and stool samples, the study aims to understand the relationship between lung and intestinal health in these patients. The research builds on previous findings from the Lum-Iva-Biota cohort, which highlighted the distinct microbiota in cystic fibrosis patients compared to healthy individuals.

Who should consider this trial

Good fit: Ideal candidates are cystic fibrosis patients aged 12 and older with at least one DeltaF508 mutation who are starting treatment with elexacaftor/tezacaftor/ivacaftor.

Not a fit: Patients who do not have cystic fibrosis or those not eligible for the specified treatment will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment strategies that enhance lung and digestive health in cystic fibrosis patients.

How similar studies have performed: Previous studies have shown promising results with similar approaches, particularly in understanding the microbiota's role in cystic fibrosis.

Eligibility criteria

Inclusion Criteria:

* 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).

Exclusion Criteria:

* 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)

Where this trial is running

Bordeaux and 14 other locations

• CHU de Bordeaux - CRCM pédiatrique — Bordeaux, France (Recruiting)
• CHU de Grenoble Alpes CRCM pédiatrique — Grenoble, France (Recruiting)
• CHRU de Lille CRCM Pédiatrique — Lille, France (Recruiting)
• CHU de Limoges CRCM Limousin — Limoges, France (Recruiting)
• Hospices Civils de Lyon Service de pédiatrie, allergologie et mucoviscidose — Lyon, France (Recruiting)
• AP-HM CRCM pédiatrique — Marseille, France (Recruiting)
• CHU de Montpellier — Montpellier, France (Recruiting)
• CHU de Nancy — Nancy, France (Recruiting)
• CHU de Nice — Nice, France (Recruiting)
• AP-HP CRCM Robert debré — Paris, France (Recruiting)
• AP-PH Hopital Cochin service de pédiatrie — Paris, France (Recruiting)
• APHP Hopital Necker — Paris, France (Recruiting)
• Fondation Ildys, Roscoff Centre Hélio Marin - Clinique "Mucoviscidose" — Roscoff, France (Recruiting)
• CHU de Rouen — Rouen, France (Recruiting)
• CHU de Toulouse — Toulouse, France (Recruiting)

Study contacts

• Principal investigator: Raphaël Enaud, MDPhD — University Hospital, Bordeaux
• Study coordinator: Aurore Capelli, PhD

Conditions Cystic Fibrosis; elexacaftor/tezacaftor/ivacaftor; lung and digestive microbiota and inflammation