Lum-Iva-biota: Exploring the Respiratory Mycobiota and Microbiota Profile in French CF Patients Taking Lumacaftor-Ivacaftor (Lum-Iva-Biota)
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|ClinicalTrials.gov Identifier: NCT03565692|
Recruitment Status : Recruiting
First Posted : June 21, 2018
Last Update Posted : April 10, 2020
n2015, VERTEX company - producing already KALYDECO (IVACAFTOR, VX-770) potentiator molecule that is recommended for the treatment of CF patients aged ≥ 6 y, with CFTR mutation altering the channel regulation (class III mutations) as G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549Nou S549R) -was allowed by the Federal Drug Administration (FDA) and European Medicines Agency (EMEA) for producing and using ivacaftor combination (such as lumacaftor/ ivacaftor initially, and more recently tezacaftor/ivacaftor, tezacaftor/ivacaftor/VX-659, tezacaftor/ivacaftor/VX-445 and tezacaftor/ivacaftor/VX-152) in clinical trials for patient with cystic fibrosis, according to age and mutation eligibility criteria.
Since 2016, the French patients homozygous for the p.Phe508del mutation and older than 12 years are able to be treated with the association LUMACAFTOR-IVACAFTOR and this French authorization is being extended for 6-11 years old children (while the European Commission has already granted an extension of the Marketing Authorization for lumacaftor/ivacaftor to include 6-11 years old children with cystic fibrosis since January 2018). Patients treated by lumacaftor/ivacaftor (or other ivacaftor new combinations) are closely monitored according to criteria established by the working group "New Therapeutic Approaches" of the French Society Cystic fibrosis.
This study is a phase IV observational trial for a period of 1 year. In this context, the team aims at initiating a comprehensive monitoring of the lung and gut mycobiota and microbiota evolution under LUMACAFTOR-IVACAFTOR (or other ivacaftor combinations) treatment. This project is directly linked to the monitoring of cystic fibrosis patients who begin treatment with LUMACAFTOR-IVACAFTOR (or other ivacaftor combinations) in France. The pro- and eukaryotic microbiota analysis is based on the secondary use of sputum and stool samples associated with several clinical data of CF patients under ivacaftor combinations and follow-up during the 1st year of therapy. According to the French law, Lum-Iva-Biota project is a non-interventional study. It aims at demonstrating that changes in the hydration of secretions at the pulmonary and intestinal levels related to LUMACAFTOR-IVACAFTOR therapy (or other new generation of ivacaftor combinations) promote a change in the lung and gut mycobiota and microbiota profiles which may achieve the characteristics of the "healthy type" (in terms of composition, richness and diversity).
|Condition or disease||Intervention/treatment|
|Cystic Fibrosis||Biological: the lung mycobiota and microbiota profile Biological: the gut mycobiota and microbiota profile|
|Study Type :||Observational|
|Estimated Enrollment :||250 participants|
|Official Title:||Lum-Iva-biota: Exploring the Respiratory Mycobiota and Microbiota Profile in French CF Patients Taking Lumacaftor-Ivacaftor|
|Actual Study Start Date :||July 1, 2018|
|Estimated Primary Completion Date :||February 28, 2023|
|Estimated Study Completion Date :||February 28, 2023|
Patients treated by LUMACAFTOR-IVACAFTOR
Patients treated by LUMACAFTOR-IVACAFTOR (or other ivacaftor combinations) Patients over 6 years who are currently able to benefit from LUMACAFTOR-IVACAFTOR (or other ivacaftor combinations) according the mutation eligibility criteria and for whom conventional microbiological analysis of sputum samples and stool will be collected during their follow-up after the treatment onset.
Biological: the lung mycobiota and microbiota profile
All sputum samples will follow regarding mycological and metagenomic analyses as summarized :
Biological: the gut mycobiota and microbiota profile
All stool samples will follow regarding mycological and metagenomic analyses as summarized : - Separation of the pellet and supernatant, stored at -20 ° C until testing - Targeted metagenomics of bacterial communities (based on 16S amplification) and of fungal community (based on ITS2 amplification) - Bioinformatic analysis of metagenomics raw data, correlation with bioclinical data of each patient, statistical analysis, and characterization of phenomena of co-evolution/co-exclusion according to evolutionary ecology concepts. - Measurement of inflammation.
- Change of specific bacterial and/or fungal pathogens [ Time Frame: 18 months ]Measure by conventional methods (history of microbial culture and GM assay) and particularly by metagenomic analysis of pulmonary pro- and eukaryotic microbiota.
- Forced expiratory volume in 1 second (FEV1) [ Time Frame: Day 1 ]Difference between the amount of air exhaled may be measured during the first second
- Forced expiratory volume (FEV1) [ Time Frame: 6 Months ]Difference between the amount of air exhaled may be measured during the first second
- Forced expiratory volume (FEV1) [ Time Frame: 12 Months ]Difference between the amount of air exhaled may be measured during the first second
- Change of specific bacterial and/or fungal pathogens [ Time Frame: 12 months ]Measure by conventional methods (history of microbial culture and GM assay) and particularly by metagenomic analysis of lung pro- and eukaryotic microbiota.
- Change of specific bacterial and/or fungal pathogens [ Time Frame: 12 months ]Measure by metagenomic analysis of gut pro- and eukaryotic microbiota.
Biospecimen Retention: Samples Without DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03565692
|Contact: Laurence DELHAES, MD, PhD||+335 56 79 56 firstname.lastname@example.org|
|CHU de Bordeaux - CRCM||Recruiting|
|Bordeaux, France, 33000|
|Contact: Mickael FAYON, MD,PhD email@example.com|
|Principal Investigator: Mickael FAYON, MD,PhD|
|Sub-Investigator: Patrick BERGER, MD,PhD|
|Sub-Investigator: Raphael ENAUD, MD|
|Centre Hospitalier Universitaire Grenoble Alpes||Recruiting|
|Grenoble, France, 38000|
|Contact: Catherine Llerena, MD CLlerena@chu-grenoble.fr|
|Principal Investigator: Catherine Llerena, MD|
|Sub-Investigator: Boubou Camara, MD|
|CHRU de Lille||Not yet recruiting|
|Lille, France, 69000|
|Contact: Nathalie Wizla, MD firstname.lastname@example.org|
|Principal Investigator: Nathalie Wizla, MD|
|Sub-Investigator: Anne Prévotat, MD|
|Hospices Civils de Lyon||Not yet recruiting|
|Lyon, France, 69000|
|Contact: Philippe Reix, MD email@example.com|
|Principal Investigator: Philippe Reix, MD|
|Assistance publique Hôpitaux Marseille||Recruiting|
|Marseille, France, 13000|
|Contact: Jean Christophe Dubus, MD,PhD JeanChristophe.DUBUS@ap-hm.fr|
|Principal Investigator: Jean Christophe Dubus, MD,PhD|
|Assistance Publique Hôpitaux de paris||Recruiting|
|Paris, France, 75000|
|Contact: Harriet Corvol, MD,PhD firstname.lastname@example.org|
|Principal Investigator: Harriet Corvol, MD,PhD|
|Sub-Investigator: Pierre-Regis BURGEL, MD|
|Sub-Investigator: Isabelle Sermet-Gaudelus, MD,PhD|
|Sub-Investigator: Michèle Gérardin Gérardin, MD|
|Suresnes, France, 92150|
|Contact: Dominique Grenet, MD email@example.com|
|Principal Investigator: Dominique Grenet, MD|
|CHU de Toulouse||Recruiting|
|Toulouse, France, 31000|
|Contact: Marlène Murris-Espin, MD firstname.lastname@example.org|
|Principal Investigator: Marlène Murris-Espin, MD|
|Principal Investigator:||Laurence DELHAES, MD,PhD||University Hospital, Bordeaux|