Clinical trial NCT06322446: Exercise in People With Cystic Fibrosis on CFTR Modulator Therapy
Official title: Effects of a Strength Exercise Program on Muscular Health in People With CF Treated With Modulators of the Cystic Fibrosis Transmembrane Conductance Regulator Receptor (CFTR)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Cystic Fibrosis
Keywords: Physical Fitness
Muscle Strength
Resistance Training
Lung funcion
Lead sponsor: Universidad Politecnica de Madrid
Sponsor class: OTHER
Collaborators: name: Carlos III Health Institute
name: Universidad Europea de Madrid
name: Hospital Universitario La Paz
name: Hospital Universitario Ramon y Cajal
name: Hospital Infantil Universitario Niño Jesús, Madrid, Spain
First posted: 2024-03-21
Enrollment: 48
Sex: ALL
Minimum age (years): 6
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Recently, the treatment of Cystic Fibrosis (CF) incorporated new modulators/enhancers of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). It is thus increasingly important to study the side effects of these drugs, their extrapulmonary effects and possible interaction with other drugs and with exercise. For this purpose, a randomized controlled trial is proposed to determine the effects of a telematic exercise intervention on muscle health, in a group of 48 children and adolescents with CF treated with these new generation CFTR modulators. They will be randomly assigned to two groups (exercise and control group). The effect of the intervention will be analysed measuring the variables of muscle health, cardiorespiratory fitness, lung function, body composition, inflammatory biomarkers and miRNAs. After completion of the intervention program, adherence to exercise and clinical evolution after one year will be analysed.

Detailed description: Specific objectives

1. To determine the effects of a strength exercise intervention in a group of children and adolescents with cystic fibrosis treated with new generation CFTR modulators on: (1.i) peripheral muscle strength; (1.ii) respiratory muscle strength; (1.iii) muscle mass and (1.iv) biomarkers of muscle damage.
2. To determine the effects of a strength exercise intervention in a group of children and adolescents with cystic fibrosis being treated with new generation CFTR modulators on: (2.i) cardiorespiratory fitness; (2.ii) body composition and (2.iii) lung function.
3. To determine the effects of a strength-based exercise intervention in a group of children and adolescents with cystic fibrosis treated with new-generation CFTR modulators on: (3.i) biomarkers of inflammatory status and (3.ii) expression of associated miRNAs.
4. To determine the effects of a strength-based exercise intervention in a group of children and adolescents with cystic fibrosis treated with the new generation CFTR modulators on the levels of Elexaxcaftor/Ivacaftor/Tezacaftor and their metabolites.
5. To assess the clinical evolution and adherence to exercise after 6 months of the programme in children and adolescents with cystic fibrosis treated with the new generation of CFTR modulators.

Eligibility criteria: Inclusion Criteria:

* patients diagnosed with CF.
* patients between 6-20 years of age.
* patients receiving treatment with new CFTR protein modulating medication
* reading, acceptance and signing of the informed consent form.

Exclusion Criteria:

* CF patients with symptoms of pulmonary exacerbation during the last four weeks.
* with a diagnosis of other cardiorespiratory lung diseases progressing to a symptom of persistent respiratory dysfunction.
* CF patients with musculoskeletal alterations that influence assessments.
* CF patient who is pregnant during the time of the study
* CF patient with cognitive impairment;
* CF patient with incomplete dosing of modulator therapy;

Primary outcomes:
measure: Changes in peripheral muscle strength
timeFrame: Baseline, pre-intervention and immediately after the intervention.
measure: Changes in inspiratory/expiratory muscle strength (MIP/MEP) (cmH2O)
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in functional capacity: lower limbs power capacity
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in functional capacity: walking capacity
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Change in Cardiorespiratory fitness: maximal oxygen consumption
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Change in Cardiorespiratory fitness: Ventilatory threshold VT1
timeFrame: Baseline, pre-intervention and immediately after the intervention
Secondary outcomes:
measure: Changes in Pulmonary Function: forced vital capacity (FVC)
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in Pulmonary Function: Forced expiratory volume in the first second (FEV1)
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in the anthropometric and body composition: Weight
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in the anthropometric and body composition: Height
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in the anthropometric and body composition: BMI
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in body composition: Total fat mass
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in body composition: FMI
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in body composition: lean mass kg
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in body composition: lean mass %
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in quality of life using the Cystic Fibrosis Questionnaire
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in plasma levels muscle damage biomarkers
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in plasma levels of inflammation: hs-CRP
timeFrame: Baseline, pre-intervention and immediately after the intervention
measure: Changes in plasma levels of inflammation: Interleukins
timeFrame: Baseline, pre-intervention and immediately after the intervention
Other outcomes:
Locations:
city: Madrid
country: Spain
facility: Facultad de Ciencias de la Actividad Física y Deporte - INEF UPM
Study officials:
affiliation: Universidad Politecnica de Madrid
name: Margarita Perez Ruiz, PhD
role: PRINCIPAL_INVESTIGATOR
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