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)

Universidad Politecnica de Madrid2 个研究点分布在 1 个国家目标入组 48 人开始时间: 2024年2月27日最近更新: 2026年5月1日

适应症

干预措施

试验速览

阶段: 不适用
状态: 进行中（未招募）
发起方: Universidad Politecnica de Madrid
入组人数: 48
试验地点: 2
主要终点: Changes in peripheral muscle strength

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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.

详细描述

Specific objectives

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.
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.
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.
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.
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.

研究设计

研究类型: Interventional
分配方式: Randomized
干预模型: Parallel
主要目的: Treatment
盲法: Single (Outcomes Assessor)

入排标准

年龄范围: 6 Years 至 20 Years（Child, Adult）
性别: All
接受健康志愿者: 否

入选标准

•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.

排除标准

•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;

研究组 & 干预措施

Intervention

Experimental

Telematic Exercise:

A remotely supervised resistance exercise program will be carried out for 16 weeks, with two weekly sessions lasting approximately 60 minutes each. Training will be performed in groups of four patients, according to their lung function/physical fitness.

The first training session will be on site (University) for familiarization, planning and adjustment of the exercises, and the following sessions will be performed online. Each session is divided into: (i) Warm-up and joint mobility; (ii) main part: strength exercises for different muscle groups; and (iii) cool down: stretching and breathing exercises.

干预措施: Exercise (Other)

Control

No Intervention

Control group will follow routine recommendations from the multidisciplinary CF team based on WHO´s guidelines

结局指标

主要结局

Changes in peripheral muscle strength

时间窗: 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)