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 | Unknown

• 1 other identifier: 2021_0201
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Collection of the different remarkable events declared in the application (adverse events and favorable events measured by perception scales)

  Collection of the different remarkable events declared in the application (adverse events and favorable events) between Day 0 and Day 60 +/-30 days A great number of data will be collected from the indications declared by the patient at home and cannot be summarized in the title. The originality of the study is the collection in real life facilitated by the provision of connected objects and the functionnalities of the PHEAL-CR-K application

  1 year

Secondary Outcomes (13)

• 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

  1 year

• 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

  1 year

• 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)

  1 year

• Daily evolution or over the course of the measurements carried out during the follow-up period of oxygen saturation collected by a connected watch

  1 year

• 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

  1 year

Study Arms (1)

Patients with Cystic Fibrosis, with KAFTRIO® treatment prescription

EXPERIMENTAL

Patients with Cystic Fibrosis, for whom treatment with KAFTRIO® is prescribed, regardless of previously prescribed CFTR modulator treatments.

Other: Kaftrio utilization in real life

Interventions

Kaftrio utilization in real life OTHER

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

Patients with Cystic Fibrosis, with KAFTRIO® treatment prescription

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Hopital Foch: lead

Study Sites (1)

Grenet Dominique

Suresnes, France

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Dominique Grenet

  Foch Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Elisabeth Hulier-Ammar

CONTACT

00 33 1 46 25 11 75; drci-promotion@hopital-foch.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: prospective, open-label, multicenter study

Study Record Dates

• First Submitted: February 18, 2022
• First Posted: March 25, 2022
• Study Start: March 14, 2022
• Primary Completion: March 1, 2023
• Study Completion: March 1, 2023
• Last Updated: August 4, 2022

Record last verified: 2022-08

Locations

FR (1)