Examining muscle function and physical activity in cystic fibrosis patients

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

This study is trying to see how cystic fibrosis affects muscle strength and activity levels in patients, especially with new treatments, to understand how it impacts their exercise and quality of life.

Quick facts

• Study type: Observational
• Enrollment: 112 (estimated)
• Ages: 10 Years and up
• Sex: All
• Sponsor: Hospices Civils de Lyon (Academic / other)
• Locations: 1 site (Hyères)
• Trial ID: NCT06251622 on ClinicalTrials.gov

What this trial studies

This observational study investigates the impact of cystic fibrosis on muscle function and physical activity, particularly in the context of recent advancements in CFTR modulator therapies. It aims to assess both quantitative and qualitative muscle impairments in patients diagnosed with cystic fibrosis, focusing on how these factors influence exercise tolerance and overall quality of life. Participants will undergo volumetric ultrasound assessments of their quadriceps and wear accelerometers to monitor physical activity levels. The study seeks to provide insights into the relationship between modern treatments and muscle health in cystic fibrosis patients.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 10 years or older with a diagnosis of cystic fibrosis who are currently receiving CFTR modulator therapy.

Not a fit: Patients with unstable clinical conditions, such as those experiencing pulmonary exacerbations, may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of muscle function in cystic fibrosis, potentially leading to improved exercise recommendations and therapies for patients.

How similar studies have performed: While there have been studies on muscle function in cystic fibrosis, this study's focus on the effects of modern CFTR modulators on muscle health is relatively novel.

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:

* Exclusion criteria for CF patients:

  * 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.
* Exclusion criteria for healthy subjects:

  * 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.

Where this trial is running

Hyères

• HCL - Hôpital Renée Sabran — Hyères, France (Recruiting)

Study contacts

• Study coordinator: Laurent MD MELY

Conditions Cystic Fibrosis; Cystic fibrosis; CFTR modulators; Physical activity; Muscle function