Cystic Fibrosis
Conditions
Keywords
Physical Fitness, Muscle Strength, Resistance Training, Lung funcion
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.
Interventions
OTHERExercise
16-week exercise intervention: At the beginning of the intervention, we will conduct a face-to-face familiarisation session with the exercises of the training programme. The intervention will consist of 2 sessions/week for 16 weeks. Each session consists of three stages: (i) Warm-up: 10 min of joint mobility and low intensity exercises involving the musculature to be worked in that session; (ii) Main part: circuit training mainly composed of strength exercises targeting the different muscle groups of the body and playing activities (iii) Cool down: 10 min with guided breathing work and stretching of the main muscle groups worked
Sponsors
Universidad Politecnica de Madrid
Carlos III Health Institute
Universidad Europea de Madrid
Hospital Universitario La Paz
Hospital Universitario Ramon y Cajal
Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 20 Years
Healthy volunteers
No
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;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in peripheral muscle strength | Baseline, pre-intervention and immediately after the intervention. | Upper and lower limbs muscle strength (kg) will be evaluated using dynamometers. |
| Changes in inspiratory/expiratory muscle strength (MIP/MEP) (cmH2O) | Baseline, pre-intervention and immediately after the intervention | Inspiratory and expiratory muscle strength: maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) will be measured. |
| Changes in functional capacity: lower limbs power capacity | Baseline, pre-intervention and immediately after the intervention | 30 seconds sit-to-stand test. Unit of measurement: number of repetitions. |
| Changes in functional capacity: walking capacity | Baseline, pre-intervention and immediately after the intervention | 10m Time (s) Up and Go tests. |
| Change in Cardiorespiratory fitness: maximal oxygen consumption | Baseline, pre-intervention and immediately after the intervention | To assess cardiorespiratory fitness, a treadmill and a gas analyser will be used. The test aims to determine maximal oxygen consumption (VO2peak in ml/kg/min). VO2peak will be recorded as the highest value obtained during a continuous 30 s period. |
| Change in Cardiorespiratory fitness: Ventilatory threshold VT1 | Baseline, pre-intervention and immediately after the intervention | To assess cardiorespiratory fitness, a treadmill and a gas analyser will be used. The test aims to determine the ventilatory threshold VT1 in response to maximal effort. VT1 will be determined using the criteria of an increase in both ventilatory equivalent for oxygen consumption (VE/VO2) and end-tidal oxygen pressure without an increase in ventilatory equivalent for carbon dioxide production (VE/VCO2). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Pulmonary Function: forced vital capacity (FVC) | Baseline, pre-intervention and immediately after the intervention | Spirometry will assess: forced vital capacity (FVC) in milliliters and percentage of predicted value |
| Changes in Pulmonary Function: Forced expiratory volume in the first second (FEV1) | Baseline, pre-intervention and immediately after the intervention | Spirometry will assess: forced expiratory volume in the first second (FEV1).Data will be expressed in absolute values and z-score based on the Global Lung Initiative (GLI) reference equation establishing as a limit of normality (LIN) a z-score value for FEV1 between -1.64 and + 1.64. |
| Changes in the anthropometric and body composition: Weight | Baseline, pre-intervention and immediately after the intervention | Weight (kg) |
| Changes in the anthropometric and body composition: Height | Baseline, pre-intervention and immediately after the intervention | Height (cm) |
| Changes in the anthropometric and body composition: BMI | Baseline, pre-intervention and immediately after the intervention | BMI (kg/m2) |
| Changes in body composition: Total fat mass | Baseline, pre-intervention and immediately after the intervention | Total fat mass (kg) |
| Changes in body composition: FMI | Baseline, pre-intervention and immediately after the intervention | fat mass index (FMI) (kg/m2), |
| Changes in body composition: lean mass kg | Baseline, pre-intervention and immediately after the intervention | Lean mass in kg |
| Changes in body composition: lean mass % | Baseline, pre-intervention and immediately after the intervention | Lean mass in % |
| Changes in quality of life using the Cystic Fibrosis Questionnaire | Baseline, pre-intervention and immediately after the intervention | Scores range from 0 to 100 with higher scores corresponding to better quality of life. |
| Changes in plasma levels muscle damage biomarkers | Baseline, pre-intervention and immediately after the intervention | Blood levels biomarkers of muscle damage as CK measured in micrograms per litre (mcg/L) |
| Changes in plasma levels of inflammation: hs-CRP | Baseline, pre-intervention and immediately after the intervention | High-sensitivity C-reactive protein (hs-CRP) assay in milligrams/litre |
| Changes in plasma levels of inflammation: Interleukins | Baseline, pre-intervention and immediately after the intervention | Analysis of interleukins such as IL6, IL-10 in picograms/millilitre |
Countries
Spain
Contacts
PRINCIPAL_INVESTIGATORMargarita Perez Ruiz, PhD
Universidad Politecnica de Madrid
Outcome results
None listed