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Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor (KAF-BIOTA)

Primary Purpose

Cystic Fibrosis

Status
Recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Sample collection
Sponsored by
University Hospital, Bordeaux
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Cystic Fibrosis focused on measuring elexacaftor/tezacaftor/ivacaftor, lung and digestive microbiota and inflammation

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • CHU de Bordeaux - CRCM pédiatriqueRecruiting
  • CHU de Grenoble Alpes CRCM pédiatriqueRecruiting
  • CHRU de Lille CRCM PédiatriqueRecruiting
  • CHU de Limoges CRCM LimousinRecruiting
  • Hospices Civils de Lyon Service de pédiatrie, allergologie et mucoviscidoseRecruiting
  • AP-HM CRCM pédiatriqueRecruiting
  • CHU de MontpellierRecruiting
  • CHU de NancyRecruiting
  • CHU de NiceRecruiting
  • AP-HP CRCM Robert debréRecruiting
  • AP-PH Hopital Cochin service de pédiatrieRecruiting
  • APHP Hopital NeckerRecruiting
  • Fondation Ildys, Roscoff Centre Hélio Marin - Clinique "Mucoviscidose"Recruiting
  • CHU de RouenRecruiting
  • CHU de ToulouseRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

patients with cystic fibrosis

Arm Description

patients with cystic fibrosis before and one year after the start of treatment with elexacaftor/tezacaftor/ivacaftor

Outcomes

Primary Outcome Measures

composition of the digestive bacterial microbiota
composition of the digestive, bacterial microbiota, at 12 months of treatment

Secondary Outcome Measures

composition of the pulmonary bacterial microbiota
composition of the pulmonary bacterialmicrobiota, at 12 months of treatment
composition of the pulmonary bacterial microbiota
composition of the pulmonary bacterialmicrobiota at baseline
composition of the digestive fungal microbiota
composition of the digestive fungal microbiota, at 12 months of treatment
composition of the digestive fungal microbiota
composition of the digestive fungal microbiota at baseline
composition of the pulmonary fungal microbiota
composition of the pulmonary fungal microbiota, at 12 months of treatment
composition of the pulmonary fungal microbiota
composition of the pulmonary fungal microbiota at baseline
composition of the digestive, bacterial microbiota
composition of the digestive, bacterial microbiota at baseline

Full Information

First Posted
June 27, 2023
Last Updated
July 7, 2023
Sponsor
University Hospital, Bordeaux
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1. Study Identification

Unique Protocol Identification Number
NCT05937815
Brief Title
Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor
Acronym
KAF-BIOTA
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 13, 2021 (Actual)
Primary Completion Date
September 13, 2023 (Anticipated)
Study Completion Date
September 13, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Bordeaux

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
elexacaftor/tezacaftor/ivacaftor, lung and digestive microbiota and inflammation

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
253 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
patients with cystic fibrosis
Arm Type
Experimental
Arm Description
patients with cystic fibrosis before and one year after the start of treatment with elexacaftor/tezacaftor/ivacaftor
Intervention Type
Procedure
Intervention Name(s)
Sample collection
Intervention Description
collection of sputum, stool and blood samples at baseline, 6 months and 1 year after baseline
Primary Outcome Measure Information:
Title
composition of the digestive bacterial microbiota
Description
composition of the digestive, bacterial microbiota, at 12 months of treatment
Time Frame
12 months after baseline (treatment initiation)
Secondary Outcome Measure Information:
Title
composition of the pulmonary bacterial microbiota
Description
composition of the pulmonary bacterialmicrobiota, at 12 months of treatment
Time Frame
12 months after baseline (treatment initiation)
Title
composition of the pulmonary bacterial microbiota
Description
composition of the pulmonary bacterialmicrobiota at baseline
Time Frame
at baseline (treatment initiation)
Title
composition of the digestive fungal microbiota
Description
composition of the digestive fungal microbiota, at 12 months of treatment
Time Frame
12 months after baseline (treatment initiation)
Title
composition of the digestive fungal microbiota
Description
composition of the digestive fungal microbiota at baseline
Time Frame
at baseline (treatment initiation)
Title
composition of the pulmonary fungal microbiota
Description
composition of the pulmonary fungal microbiota, at 12 months of treatment
Time Frame
12 months after baseline (treatment initiation)
Title
composition of the pulmonary fungal microbiota
Description
composition of the pulmonary fungal microbiota at baseline
Time Frame
at baseline (treatment initiation)
Title
composition of the digestive, bacterial microbiota
Description
composition of the digestive, bacterial microbiota at baseline
Time Frame
at baseline (treatment initiation)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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)
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Aurore Capelli, PhD
Phone
0557820877
Email
aurore.capelli@chu-bordeaux.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Raphaël Enaud, MDPhD
Phone
05 56 79 98 24
Email
raphael.enaud@chu-bordeaux.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Raphaël Enaud, MDPhD
Organizational Affiliation
University Hospital, Bordeaux
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHU de Bordeaux - CRCM pédiatrique
City
Bordeaux
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Raphaël ENAUD
Facility Name
CHU de Grenoble Alpes CRCM pédiatrique
City
Grenoble
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Catherine LLerena
Facility Name
CHRU de Lille CRCM Pédiatrique
City
Lille
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nathalie Wizla
Facility Name
CHU de Limoges CRCM Limousin
City
Limoges
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alexandra Masson-Rouchaud
Facility Name
Hospices Civils de Lyon Service de pédiatrie, allergologie et mucoviscidose
City
Lyon
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philippe Reix
Facility Name
AP-HM CRCM pédiatrique
City
Marseille
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jean-Christophe Dubus
Facility Name
CHU de Montpellier
City
Montpellier
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Raphael CHIRON
Facility Name
CHU de Nancy
City
Nancy
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aurélie Tatopoulos
Facility Name
CHU de Nice
City
Nice
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sylvie Leroy
Facility Name
AP-HP CRCM Robert debré
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michèle GERARDIN
Facility Name
AP-PH Hopital Cochin service de pédiatrie
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pierre-Régis BURGEL
Facility Name
APHP Hopital Necker
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Isabelle Sermet Gaudelus
Facility Name
Fondation Ildys, Roscoff Centre Hélio Marin - Clinique "Mucoviscidose"
City
Roscoff
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sophie RAMEL
Facility Name
CHU de Rouen
City
Rouen
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hélène Morisse Pradier
Facility Name
CHU de Toulouse
City
Toulouse
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marie Mittaine

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor

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