Clinical trial NCT04415268: Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis
Official title: Combined Effect of CFTR Protein Modulator Drugs and Exercise on Pulmonary Function, Fitness, Sweat Test and Quality of Life in Children With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Cystic Fibrosis in Children
Keywords: Cystic Fibrosis
Electrostimulation
Combined Training
Physical Fitness
Lead sponsor: Universidad Europea de Madrid
Sponsor class: OTHER
First posted: 2020-06-04
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single-group non-randomized, prospective. Within-subject multitreatment
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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).

Primary outcomes:
measure: Change in Strength
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Change in Cardiorespiratory Fitness
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
Secondary outcomes:
measure: Changes in Forced expiratory volume in 1 second (FEV1)
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Changes in Forced vital capacity (FVC)
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Changes in FEV1/FVC ratio (FEV1%)
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Changes in Forced expiratory flow (FEF)
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Changes in Physical Activity Questionnaire (PAQ) for children and adolescents
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Change in quality of life: Cystic Fibrosis-Questionnaire-Revised (CFQ-R)
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
measure: Sweat chloride level
timeFrame: Four assessment points throughout the study: baseline and after each 8-week intervention
Other outcomes:
Locations:
city: Villaviciosa De Odón
country: Spain
facility: Escuela de Doctorado e Investigacion, Universidad Europea
Study officials:
affiliation: Universidad Politecnica de Madrid
name: Margarita Perez-Ruiz, MD
role: PRINCIPAL_INVESTIGATOR
References: