Comparison of Muscle Function and Physical Activity Levels Between People With Cystic Fibrosis and Healthy Individuals in the Modern Era of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators: A Case-Control Study

Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Observational
SUMMARY

Cystic fibrosis (CF) is characterized by various extrapulmonary manifestations, including altered skeletal muscle function, with both quantitative (e.g. reduced muscle mass) and qualitative (e.g. impaired oxidative function) impairments that may have a negative impact on exercise tolerance and quality of life. These abnormalities have traditionally been related to disease (e.g. systemic inflammation) or behavioral factors (e.g. increased physical inactivity). However, most of the studies that observed these abnormalities and tried to shed light on the underlying factors were either small or conducted before the widespread of CFTR (Cystic fibrosis transmembrane conductance regulator) modulators that have profound impact on the trajectory of the disease. While several studies suggested that the major recent improvements in therapeutics, including highly effective CFTR modulators, may have positive effects on skeletal muscle function, either directly (e.g. improved mitochondrial function) or indirectly (e.g. reduction in physical inactivity), no studies to date have thoroughly investigated this issue in a representative sample of people with CF. The absence of recent data on muscle function and physical activity levels casts doubt on the relevance of recommendations on exercise training in this population that were published before the widespread use of highly effective CFTR modulators. This study aims to compare muscle function, measured according to the latest recommendations of the European Cystic Fibrosis Society (Saynor et al., 2023), and physical activity of children and adults with CF under CFTR modulators, compared to age- and sex-matched healthy individuals. We hypothesize that the strength, endurance, muscle power, and physical activity levels of individuals with cystic fibrosis, treated with CFTR modulators, remain reduced compared to healthy individuals.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 10
Healthy Volunteers: t
View:

• Criteria for inclusion of CF patients:

‣ Diagnosis of cystic fibrosis

⁃ Treated with CFTR modulator therapy

⁃ Men and women (children or adults) aged 10 years or older

⁃ Affiliated with or entitled to social security coverage

⁃ For people ≥ 18 years old: participant expressing its oral non-opposition

⁃ For minor child (\<18 years old): participant and its legal representative expressing their oral non-opposition

• Criteria for inclusion of healthy subjects:

‣ Men and women (children or adults) aged 10 years or older

⁃ Affiliated with or entitled to social security coverage

⁃ For people ≥ 18 years old: participant expressing its oral non-opposition

⁃ For minor child (\<18 years old): participant and its legal representative expressing their oral non-opposition

Locations
Other Locations
France
HCL - Hôpital Renée Sabran
RECRUITING
Hyères
Contact Information
Primary
Laurent MD MELY
laurent.mely@chu-lyon.fr
04 94 38 17 52
Backup
Antoine-Raphaël MD Bronstein
antoine-raphael.bronstein@chu-lyon.fr
0662839256
Time Frame
Start Date: 2024-02-19
Estimated Completion Date: 2025-10-01
Participants
Target number of participants: 112
Treatments
People with Cystic fibrosis treated with CFTR modulators
Children (over 10 years old) and adults with cystic fibrosis, with a stable clinical condition and no contraindications to engaging in moderate-intensity physical activities (PA).
Healthy individuals
Healthy children (over 10 years old) and adults without known diseases (chronic respiratory, cardiovascular, metabolic, renal, or neuromuscular diseases) that may affect their peripheral muscle strength.
Related Therapeutic Areas
Sponsors
Leads: Hospices Civils de Lyon

This content was sourced from clinicaltrials.gov