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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

NCT05295524 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2022-08-04

No results posted yet for this study

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.

Conditions

Interventions

OTHER

Kaftrio utilization in real life

daily utilization of PHEAL-CR-K application at home and VOC (volatile organic compound) measure of exhaled air for a group of the patients

Sponsors & Collaborators

  • Hopital Foch

    lead OTHER

Principal Investigators

  • Dominique Grenet · Foch Hospital

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-14
Primary Completion
2023-03-31
Completion
2023-03-31

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05295524 on ClinicalTrials.gov