- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04415268
Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis
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 Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Phase
- Not Applicable
Contacts and Locations
Study Locations
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Villaviciosa De Odón, Spain, 28670
- Escuela de Doctorado e Investigacion, Universidad Europea
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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). |
Surpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions.
Unsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home.
Standard of care: Lumacaftor with Ivacaftor or Tezacaftor combined with Ivacaftor. For children between 6 and 11 years of age, Lumacaftor 400 mg combined with Ivacaftor 500 mg in total per day, divided into 2 doses per day; for those over 11 years of age, Tezacaftor 100 mg combined with Ivacaftor 150 mg in the morning, adding a dose of Ivacaftor 150 mg at night. https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-000833-37/ES
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Strength
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in strength will be measured using a five repetition maximum test (5RM)
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Change in Cardiorespiratory Fitness
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in cardiorespiratory fitness will be measured using a cardiopulmonary exercise test (CPET)
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in Forced expiratory volume in 1 second (FEV1)
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in FEV1 will be measured using Spirometry (z-score based on Global Lung Function Initiative reference DOI: 10.1016/j.arbres.2017.07.019)
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in Forced vital capacity (FVC)
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in FVC will be measured using Spirometry (z-score based on Global Lung Function Initiative reference DOI: 10.1016/j.arbres.2017.07.019)
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in FEV1/FVC ratio (FEV1%)
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in FEV1/FVC ratio (FEV1%) will be measured using Spirometry (z-score based on Global Lung Function Initiative reference DOI: 10.1016/j.arbres.2017.07.019)
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in Forced expiratory flow (FEF)
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in FEF will be measured using Spirometry (z-score based on Global Lung Function Initiative reference DOI: 10.1016/j.arbres.2017.07.019)
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in Physical Activity Questionnaire (PAQ) for children and adolescents
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in physical activity levels will be measured using PAQ-C for children under 14 years of age and PAQ-A for adolescents over 14 years of age. Items 1 to 9 will be used in the physical activity composite score, and means will be calculated to obtain the final PAQ-C activity summary score. Items 1 to 8 will be used in the physical activity composite score, and means will be calculated to obtain the final PAQ-A activity summary score. A score of 1 indicates low physical activity, whereas a score of 5 indicates high physical activity. |
Four assessment points throughout the study: baseline and after each 8-week intervention
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Change in quality of life: Cystic Fibrosis-Questionnaire-Revised (CFQ-R)
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Changes in quality of life will be measures with the Cystic Fibrosis-Questionnaire-Revised (CFQ-R). Scores for each health related quality of life domain are calculated; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health. |
Four assessment points throughout the study: baseline and after each 8-week intervention
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Sweat chloride level
Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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Chloride concentration in sweat (mEq/L) will be measured in the laboratory using an MK II Chloride Analyzer 926S
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Four assessment points throughout the study: baseline and after each 8-week intervention
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Margarita Perez-Ruiz, MD, Universidad Politécnica de Madrid.
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CIPI/20/119
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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