Cystic Fibrosis (CF), Mucoviscidosis
Conditions
Keywords
Cystic fibrosis, physical activity, CFTR modulator treatment, exercise limitation, respiratory physiology, ventilatory limitation, reduction in ventilatory reserve, dynamic distension
Brief summary
Cystic fibrosis is a genetic disorder affecting the entire body and associated with respiratory exacerbations, impaired quality of life and reduced life expectancy. The therapeutic management of cystic fibrosis has been profoundly changed by the recent arrival of a combination of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Elexacaftor-Tezacaftor-Ivacaftor (ETI), which improve quality of life, respiratory function and reducing the number of exacerbations. The impact of these treatments on exercise adaptation has not been clearly identified. The main objective is to estimate the prevalence of ventilatory reserve amputation during submaximal exercise testing assessed by the 6-minute walk test (6MWT) in patients with cystic fibrosis treated with ETIs.
Detailed description
Cystic fibrosis is a genetic disorder affecting the entire body and associated with respiratory exacerbations, impaired quality of life and reduced life expectancy. The therapeutic management of cystic fibrosis has been profoundly changed by the recent arrival of a combination of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Elexacaftor-Tezacaftor-Ivacaftor (ETI), which improve quality of life, respiratory function and reducing the number of exacerbations. The impact of these treatments on exercise adaptation has not been clearly identified. The main objective is to estimate the prevalence of ventilatory reserve amputation during submaximal exercise testing assessed by the 6-minute walk test (6MWT) in patients with cystic fibrosis treated with ETIs.
Interventions
DEVICEMeasurement of resistance through forced oscillations and continuous measurement of ventilation and inspiratory capacity during TM6
Measurement of resistance through forced oscillations and continuous measurement of ventilation and inspiratory capacity during TM6
Sponsors
University Hospital, Lille
Vaincre la Mucoviscidose
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Intervention model description
No control group. Patients treated with ETI (Kaftrio-Kalydeco©) are treated as part of the management of their condition in accordance with the indications for marketing authorisation (MA), temporary authorisation for use (ATU) or French compassionate access.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female * Adult aged 18 or over * Suffering from cystic fibrosis * Treated at the CRCM in Lille and Créteil * Treated by ETI * Be covered by social security * Be able to understand the requirements of the study, provide written informed consent, and comply with the study's data collection procedures
Exclusion criteria
* Medical contraindication or inability to perform a stress test according to ERS recommendations * Absolute contraindications * Relative contraindications: * Exacerbation of the condition in the 4 weeks preceding the V1 visit (27). * Pregnant or breastfeeding women * Administrative reasons * Persons deprived of their liberty * Minors or protected adults * Persons who have refused or are unable to give informed consent * Persons in emergency situations
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Estimate the prevalence of ventilatory limitation measured by a portable spirometer during a submaximal exercise test (6-minute walk test) in patients with cystic fibrosis undergoing ETI. | duration of 56 weeks starting in December 2025 | The primary endpoint is ventilatory limitation defined as the difference between the estimated theoretical maximun minute ventilatory volume (VMM in L/min) estimated by multiplying FEV1 (L) x 35 and the measurement of external ventilation during exercise (VE, L/min) using a portable spirometer divided by the theoretical VM x100 less than 15%. Ventilatory limitation = (MMV (L/min) - VE (L/min) /MMV (L/min))\*100 \< 15% |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Estimate the prevalence of ventilatory limitation measured using a portable spirometer during a maximal incremental step test (A-STEP) with progressive steps in patients with cystic fibrosis undergoing ETI. | duration of 56 weeks starting in December 2025 | The assessment criterion is the difference between the estimated theoretical maximun minute ventilatory volume (VMM in L/min) estimated by multiplying FEV1 (L) x 35 and the measurement of external ventilation (VE in L/min) using a portable spirometer during exercise divided by the theoretical VM x100 less than 15%. |
| Assessment of the prevalence of dynamic distension during a submaximal (TM6) or maximal (incremental step test according to the A-Step protocol) exercise test by spirometry | duration of 56 weeks starting in December 2025 | The criterion for judgement is a decrease in inspiratory capacity (L) of more than 150 mL within 30 seconds after the end of the effort compared to the pre-effort inspiratory capacity (L) measured by spirometry. |
Countries
France
Contacts
CONTACTCamille AUDOUSSET, Doctor
Outcome results
None listed