Clinical trial NCT05295524: Description of the Short-term Effects of KAFTRIO® by Continuous Monitoring With the PHEAL-CR-K Application in Real Life in Patients With Cystic Fibrosis Eligible for KAFTRIO® Treatment
Official title: Description of the Short-term Effects of KAFTRIO® by Continuous Monitoring With the PHEAL-CR-K Application in Real Life in Patients With Cystic Fibrosis Eligible for KAFTRIO® Treatment
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Cystic Fibrosis
Lead sponsor: Hopital Foch
Sponsor class: OTHER
First posted: 2022-03-25
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: prospective, open-label, multicenter study
Primary purpose: OTHER

Brief summary: This is a prospective, open-label, multicenter study (1 year) with 50 patients with cystic fibrosis for whom treatment with KAFTRIO® is prescribed.Cystic fibrosis is a rare autosomal recessive hereditary disease linked to a mutation of the CFTR (Cystic Fibrosis Transmembrane Regulator) protein gene. For the majority of patients, no treatment with a CFTR protein modulator was available until the arrival of the KAFTRIO® triple therapy (ivacaftor/tezacaftor/elexacaftor). Clinical studies on this triple therapy demonstrate significant improvements in FEV (forced vital capacity) and also very rapid health improvement of patients. However, there is a lack of data recorded in real life at home by patients to trace the evolution curves of health parameters and patient perceptions from the first days after initiation of treatment. The PHEAL-CR-K application, specially developed for the study, makes it possible to collect physiological parameters and perceptions collected via connected objects or declared manually in the application. These data will reflect the evolution of the parameters from the start of the treatment and over a period of 3 months. In addition, the composition of volatile organic compounds (VOCs) of the air exhaled in the early phase of treatment with KAFTRIO® will be monitored for the group of patients followed at Foch Hospital. Exhaled air is an ideal biological fluid for clinical monitoring (non-invasive collection and real-time analysis). In cystic fibrosis, biomarkers in the exhaled air have been correlated with functional and clinical parameters. The objective is to collect the air exhaled before initiating treatment with KAFTRIO® and during treatment, to identify VOCs whose expression would be modified early. Changes in the composition of the exhaled air will be correlated with follow-up clinical data collected with the PHEAL-CR-K application and with functional data obtained during measurements of breath by spirometry (FEV) and sweat concentrations of chloride ions collected at the during a sweat test. The identified COVs could become early predictive biomarkers of clinical response.


Eligibility criteria: Inclusion Criteria:

* Patients aged 18 and over
* Patients with Cystic Fibrosis eligible for KAFTRIO®
* Patients for which KAFTRIO® treatment is prescribed, including patients previously treated with other CFTR modulators (ORKAMBI®, KALYDECO®, SYMKEVI®)
* Patients who signed the informed consent form
* Patients affiliated to a health insurance plan
* Patients equipped with a smartphone and having a Wifi connection at home

Exclusion Criteria:

* Pregnant or breastfeeding women
* Patient who does not speak or understand French
* Patient deprived of liberty or under guardianship

Primary outcomes:
measure: Collection of the different remarkable events declared in the application (adverse events and favorable events measured by perception scales)
timeFrame: 1 year
Secondary outcomes:
measure: FEV1 (Forced Expiratory Volume in one second) measurements by portable spirometer at Day 0 and Day 60 +/-30 days, between the consultation before introduction of the drug and the first follow-up visit
timeFrame: 1 year
measure: Daily evolution or over the course of the measurements carried out during the follow-up period of FEV1 (Forced Expiratory Volume in one second) with a spirometer
timeFrame: 1 year
measure: Daily evolution or over the course of the measurements carried out during the follow-up period of weight (in kilograms) and body mass index (in kg/m^2)
timeFrame: 1 year
measure: Daily evolution or over the course of the measurements carried out during the follow-up period of oxygen saturation collected by a connected watch
timeFrame: 1 year
measure: Daily evolution or over the course of the measurements carried out during the follow-up period of heart rate and respiratory rate measured by connected watch and mattress pad
timeFrame: 1 year
measure: Evolution of respiratory exacerbations by the declaration in the application of clinical perceptions (surveys to describe the cough and the expectoration) and/or declaration of antibiotic prescription
timeFrame: 1 year
measure: Collection of reported perceptions (cough, sputum, nasal secretions, dyspnea, fatigue, sleep dyspnea, sleep, appetite, other) via visual analog scales (from zero to 4, from the better to the worse outcome)
timeFrame: 1 year
measure: Collection of KAFTRIO® compliance (missed medication)
timeFrame: 1 year
measure: Collection of anciliary antibiotic therapies and care provided (declaration in the application)
timeFrame: 1 year
measure: Assessment of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) score (scale 0-100, with higher score indicating better health-related quality of life) before/after (at Day1 at inclusion and at Day 60+/-30 at the end of the study)
timeFrame: 1 year
measure: Evaluation of VOC (volatil organic compound) profiles in exhaled air at ay D0 and variation of these profiles after treatment (during the first week and Day 60 +/-30 days)
timeFrame: 1 year
measure: Identification of VOCs (volatil organic compound) whose abundance is modified by the KAFTRIO® treatment
timeFrame: 1 year
measure: Correlation of VOC (volatil organic compound) changes to clinical and paraclinical efficacy data: remarkable events, reported events, reported perceptions, FEV1 (Forced Expiratory Volume in one second), sweat chloride ion concentration
timeFrame: 1 year
Other outcomes:
Locations:
city: Suresnes
country: France
facility: Grenet Dominique
Study officials:
affiliation: Foch Hospital
name: Dominique Grenet
role: PRINCIPAL_INVESTIGATOR
References: