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

NCT06251622 | Recruiting

• 1 other identifier: 69HCL23_1226
• Study Type: observational
• Target: 112
• Locations: 1 country
• Sites: 1

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

Keywords

Cystic fibrosis; CFTR modulators; Physical activity; Muscle function

Outcome Measures

Primary Outcomes (1)

• Measurement of isometric quadriceps strength (expressed in Newton), performed at René Sabran Hospital (as part of routine clinical practice for people with CF)

  The quadriceps strength will be measured on an isometric chair according to the last recommendations of the European Cystic Fibrosis Society (Saynor et al., 2023). Briefly, participants will perform at least three maximal voluntary contractions of the knee extensors, each lasting 4-6s, with a minimal recovery of 1-min between each attempt, with the aim to obtain three maximal quadriceps strength values varying less than 5% (that is usually obtained in less than 6 trials). The highest strength value among these three attempts will be kept for analysis.

  Day 0

Study Arms (2)

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).

Other: Volumetric ultrasound of the quadriceps and fitting of an accelerometer

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.

Interventions

Volumetric ultrasound of the quadriceps and fitting of an accelerometer 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).

People with Cystic fibrosis treated with CFTR modulators

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The population of patients with cystic fibrosis is selected from the patient cohort at Renée Sabran Hospital, a specialized center for the monitoring and treatment of patients with cystic fibrosis. The healthy individuals will be recruited in the general population from University and Hospital staff (including their children).

You may qualify if:

• 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
• 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

You may not qualify if:

• 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.
• Medical contraindication to engage in moderate-intensity physical activity.
• Knee joint pain incompatible with the measurement of quadriceps strength.
• Pregnancy.

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (1)

HCL - Hôpital Renée Sabran

Hyères, 83400, France

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis; Motor Activity

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Behavior

Central Study Contacts

Laurent MD MELY

CONTACT

04 94 38 17 52; laurent.mely@chu-lyon.fr

Antoine-Raphaël MD Bronstein

CONTACT

0662839256; antoine-raphael.bronstein@chu-lyon.fr

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2024
• First Posted: February 9, 2024
• Study Start: February 19, 2024
• Primary Completion: August 1, 2025
• Study Completion: October 1, 2025
• Last Updated: April 19, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)