Muscle Function and Physical Activity in the Modern Era of Cystic Fibrosis

NCT ID: NCT06251622

Last Updated: 2024-04-19

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 112 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-02-19
• Study Completion Date: 2025-10-01

Brief 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.

Conditions

Cystic Fibrosis

Study Design

• Observational Model Type: CASE_CONTROL
• Study Time Perspective: PROSPECTIVE

Study Groups

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).

Volumetric ultrasound of the quadriceps and fitting of an accelerometer

Intervention Type: OTHER

The research procedure will simply involve a volumetric ultrasound of the quadriceps and the fitting of an accelerometer (watch worn on the wrist) for one week (collection of usual physical activities).

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.

No interventions assigned to this group

Interventions

Volumetric ultrasound of the quadriceps and fitting of an accelerometer

The research procedure will simply involve a volumetric ultrasound of the quadriceps and the fitting of an accelerometer (watch worn on the wrist) for one week (collection of usual physical activities).

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• Lack of a stable clinical condition (defined here as the presence of a pulmonary exacerbation and/or a significant change in treatments in the three weeks preceding inclusion in the study).
• Medical contraindication to engage in moderate-intensity physical activity.
• Knee joint pain incompatible with the measurement of quadriceps strength.
• Pregnancy.

• Known chronic respiratory, cardiovascular, metabolic, renal, or neuromuscular diseases.
• Presence of benign illnesses and/or acute infections requiring a doctor's visit and occasional treatment in the three weeks preceding inclusion in the study.
• Medical contraindication to engage in moderate-intensity physical activity.
• Knee joint pain incompatible with the measurement of quadriceps strength.
• Pregnancy.

• Minimum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Hospices Civils de Lyon

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

HCL - Hôpital Renée Sabran

Hyères, , France

Site Status: RECRUITING

Countries

France

Central Contacts

Laurent MD MELY

Role: CONTACT

laurent.mely@chu-lyon.fr

04 94 38 17 52 ext. +33

Antoine-Raphaël MD Bronstein

Role: CONTACT

antoine-raphael.bronstein@chu-lyon.fr

0662839256 ext. +33

Facility Contacts

Laurent MD MELY

Role: primary

laurent.mely@chu-lyon.fr

04 94 38 17 52 ext. +33

Other Identifiers

69HCL23_1226

Identifier Type: -

Identifier Source: org_study_id