Cystic FibrosisPHEAL-KAFTRIONCT05295524
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
A study testing Kaftrio utilization in real life in people with Cystic Fibrosis, run by Hopital Foch at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Kaftrio utilization in real life
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteFrance
- Study dates
- 14 Mar 2022to Mar 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Pregnant or breastfeeding women
- Patient who does not speak or understand French
- Patient deprived of liberty or under guardianship
Trial locations and contacts
1 site in 1 country.
About this study
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
- Being tested
- Kaftrio utilization in real life
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Kaftrio utilization in real lifeOther
daily utilization of PHEAL-CR-K application at home and VOC (volatile organic compound) measure of exhaled air for a group of the patients
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: Kaftrio utilization in real life |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
The patient performs the measurements at home with a connected spirometer allowing to inform the respiratory capacity through the measurement of FEV1
Measured: 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)
The patient has a connected balance at home to measure his weight. The body mass index is calculated by the application, with the height (in meters) indicated by the patient
Measured: 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
The patient wears a connected watch on day that measures the oxygen saturation in blood and reports the data in the application
Measured: 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
The heart rate is measured at home by a connected watch (on day). A connected mattress pad measures the heart rate and respiratory rate at rest (during the sleep).
Measured: 1 year
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
Respiratory exacerbations are declared at home by the patient in the application :
- Surveys allow to describe the cough (frequency, type, moment) and the expectorations (clinical perception)
- A feature is present to declare antibiotic prescription
Measured: 1 year
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)
A list of perceptions are reported in the application (cough, sputum, nasal secretions, dyspnea, fatigue, sleep dyspnea, sleep, appetite, other), with scales.
The patient declares the perceptions at home, daily or over the course of the study, by using the 3 scales for each indicator. The final score is the mean of the 3 scales. The minimum value is 0, the maximum value is 4. The highest score means a worse outcome.
Measured: 1 year
Collection of KAFTRIO® compliance (missed medication)
Measured: 1 year
Collection of anciliary antibiotic therapies and care provided (declaration in the application)
The patient can declare in the application the prescription of anciliary antibiotic therapies and care provided. This is not a measure but an complementary information of the patient health.
Measured: 1 year
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)
Measured: 1 year
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)
Measured: 1 year
Identification of VOCs (volatil organic compound) whose abundance is modified by the KAFTRIO® treatment
Measured: 1 year
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
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2022
- Study start14 Mar 2022
- Primary completionMar 2023 (estimated)
- Study completionMar 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- prospective, open-label, multicenter study
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hopital Foch
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dominique Grenet, Principal investigator, Foch Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05295524
- Sponsor's protocol ID
- 2021_0201
- Acronym
- PHEAL-KAFTRIO
- Status last verified
- Aug 2022
- First submitted
- 18 Feb 2022
- Last updated
- 4 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05295524
- Acronym / sponsor ID
- PHEAL-KAFTRIO / 2021_0201
- Conditions
- Cystic Fibrosis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Hopital FochUniversity, hospital or other
- Study start
- 14 Mar 2022 (actual)
- Primary completion
- Mar 2023 (estimated)
- Study completion
- Mar 2023 (estimated)
- First posted
- 25 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- prospective, open-label, multicenter study
| Arm | Type | Receives |
|---|---|---|
| Patients with Cystic Fibrosis, with KAFTRIO® treatment prescription | Experimental | Other: Kaftrio utilization in real lifePatients with Cystic Fibrosis, for whom treatment with KAFTRIO® is prescribed, regardless of previously prescribed CFTR modulator treatments. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- Pregnant or breastfeeding women
- Patient who does not speak or understand French
- Patient deprived of liberty or under guardianship
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Kaftrio utilization in real lifedaily utilization of PHEAL-CR-K application at home and VOC (volatile organic compound) measure of exhaled air for a group of the patients | Other | Patients with Cystic Fibrosis, with KAFTRIO® treatment prescription |
Primary outcome
Collection of the different remarkable events declared in the application (adverse events and favorable events measured by perception scales)
Time frame: 1 year
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
13 secondary outcomes
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
Time frame: 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
Time frame: 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)
Time frame: 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
Time frame: 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
Time frame: 1 year
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
Time frame: 1 year
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)
Time frame: 1 year
Collection of KAFTRIO® compliance (missed medication)
Time frame: 1 year
Collection of anciliary antibiotic therapies and care provided (declaration in the application)
Time frame: 1 year
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)
Time frame: 1 year
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)
Time frame: 1 year
Identification of VOCs (volatil organic compound) whose abundance is modified by the KAFTRIO® treatment
Time frame: 1 year
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
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dominique Grenet principal investigator
Foch Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Kaftrio utilization in real life in people with Cystic Fibrosis, run by Hopital Foch at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Elisabeth Hulier-Ammar (00 33 1 46 25 11 75, drci-promotion@hopital-foch.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Collection of the different remarkable events declared in the application (adverse events and favorable events measured by perception scales) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Aug 2022.