Combined Effect of CFTR Protein Modulator Drugs and Exercise on Pulmonary Function, Fitness, Sweat Test and Quality of Life in Children With Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- Change in Strength
研究概览
简要总结
This study aims to assess the effects of programmed exercise combined with CFTR protein modulator drugs in the cardiorespiratory fitness, strength, functional capacity and agility in a group of young patients with Cystic Fibrosis.
详细描述
The present study is a new clinical trial that extends our previous work intended to assess the effect of programmed exercise with or without electrical stimulation on cardiorespiratory fitness, strength, functional capacity and agility in a group of young patients with Cystic Fibrosis (ECOMIRIN) registered at ClinicalTrials.gov (Identifier: NCT04153669).
After finishing the study, a subset of the participants was treated with either TEZ/IVA or LUM/IVA per standard of care, as they were considered candidates according to the AEMPS (https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-000833-37/ES). Those patients are expected to complete five months of pharmacological treatment without exercise by June 2020.
Primary Objective To evaluate the effect of an exercise program on physical fitness in cystic fibrosis patients after 5 months of administration of LUM/IVA or TEZ/IVA in comparison to the effect produced by the same exercise program before administration of the drug.
Secondary Objectives Other specific objectives include comparing the beneficial effects of exercise and LUM/IVA or TEZ/IVA on: 1) cardiorespiratory function and muscle strength; 2) the concentration of chlorine in sweat; and 3) quality of life.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •In order to be included, patients must have participated in the ECOMIRIN clinical trial (ClinicalTrials.gov Identifier: NCT04153669).
- •They must have a previous CF diagnosis, defined as: sweat chlorine ≥ 60 mEq/L and/or carry two variants of the CFTR gene characterized as pathogenic.
- •Age 6-18 years; eligible to receive treatment with TEZ/IVA or LUM/IVA according to the AEMPS (LUM/IVA for 6-11 year-old children homozygous for the F508del mutation; TEZ/IVA for ≥ 12 year-old children homozygous for the Phe508del mutation, as well as heterozygous for the F508 mutation plus one of the following mutations: P67L, R117C, L206W, R352Q, A455E, D579G, 711 + 3A G, S945L, S977F, R1070W, D1152H, 2789 + 5G A, 3272-26A G, and 3849 + 10kbC T);
- •Agreement to collaborate in performing static and dynamic pulmonary function tests.
- •Consent of children and/or parents or legal guardians to participate in the study after having read and understood the informed consent form.
排除标准
- •CF patients not consenting to participate in the study at any time after reading and understanding the informed consent form will be excluded.
- •CF patients who are not eligible to receive treatment with TEZ/IVA or LUM/IVA according to the AEMPS during the course of the study will be excluded.
- •Study participants will be excluded if they test positive for any contagious viral infection that may affect the outcomes (e.g. SARS-CoV-2).
研究组 & 干预措施
Multitreatment
Pharmacological treatment per standard of care (whole study length, starting on week 1).
Supervised exercise protocol (phase 1, 8 weeks starting on week 9). Unsupervised exercise protocol (phase 2, 8 weeks starting on week 17).
干预措施: Exercise (Behavioral)
Multitreatment
Pharmacological treatment per standard of care (whole study length, starting on week 1).
Supervised exercise protocol (phase 1, 8 weeks starting on week 9). Unsupervised exercise protocol (phase 2, 8 weeks starting on week 17).
干预措施: Unsupervised exercise (Behavioral)
Multitreatment
Pharmacological treatment per standard of care (whole study length, starting on week 1).
Supervised exercise protocol (phase 1, 8 weeks starting on week 9). Unsupervised exercise protocol (phase 2, 8 weeks starting on week 17).
干预措施: CFTR Modulators (Drug)
结局指标
主要结局
Change in Strength
时间窗: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in strength will be measured using a five repetition maximum test (5RM)
Change in Cardiorespiratory Fitness
时间窗: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in cardiorespiratory fitness will be measured using a cardiopulmonary exercise test (CPET)
次要结局
- Change in quality of life: Cystic Fibrosis-Questionnaire-Revised (CFQ-R)(Four assessment points throughout the study: baseline and after each 8-week intervention)
- Changes in Forced vital capacity (FVC)(Four assessment points throughout the study: baseline and after each 8-week intervention)
- Changes in Forced expiratory flow (FEF)(Four assessment points throughout the study: baseline and after each 8-week intervention)
- Sweat chloride level(Four assessment points throughout the study: baseline and after each 8-week intervention)
- Changes in Forced expiratory volume in 1 second (FEV1)(Four assessment points throughout the study: baseline and after each 8-week intervention)
- Changes in FEV1/FVC ratio (FEV1%)(Four assessment points throughout the study: baseline and after each 8-week intervention)
- Changes in Physical Activity Questionnaire (PAQ) for children and adolescents(Four assessment points throughout the study: baseline and after each 8-week intervention)
研究者
Margarita Perez
Full Professor MD, PhD
Universidad Europea de Madrid