Prevalence of Exercise-induced Ventilatory Limitation and Associated Factors in Patients With Cystic Fibrosis Receiving Elexacaftor-Tezacaftor-Ivacaftor (MUCOLIMEX)

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.

Study Overview

• Status: Recruiting
• Conditions: Cystic Fibrosis (CF); Mucoviscidosis
• Intervention / Treatment: Device: Measurement of resistance through forced oscillations and continuous measurement of ventilation and inspiratory capacity during TM6

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.

• Study Type: Interventional
• Enrollment (Estimated): 130
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Camille AUDOUSSET, Doctor; Phone Number: +33 +33 3.20.44.41.45; Email: DRC@chu-lille.fr

Study Locations

• France
  • Lille, France, 59037
    • Recruiting
    • University Hospital
    • Contact: Camille Audousset, Doctor; Phone Number: 0320445544; Email: camille.audousset@chu-lille.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: patients treated with ETI (Kaftrio-Kalydeco©)	Device: Measurement 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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%	duration of 56 weeks starting in December 2025

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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%.	duration of 56 weeks starting in December 2025
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	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.	duration of 56 weeks starting in December 2025

Collaborators and Investigators

• Sponsor: University Hospital, Lille
• Collaborators: Vaincre la Mucoviscidose

Study record dates

Study Major Dates

• Study Start (Actual): December 17, 2025
• Primary Completion (Estimated): February 2, 2027
• Study Completion (Estimated): April 4, 2027

Study Registration Dates

• First Submitted: November 24, 2025
• First Submitted That Met QC Criteria: December 16, 2025
• First Posted (Actual): January 2, 2026

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 21, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: physical activity; Cystic fibrosis; respiratory physiology; CFTR modulator treatment; exercise limitation; ventilatory limitation; reduction in ventilatory reserve; dynamic distension
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Behavior; Cystic Fibrosis; Motor Activity
• Other Study ID Numbers: 2024_0277; 2024-A02709-38 (Registry Identifier: ID-RCB)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No