Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis
NCT ID: NCT04415268
Last Updated: 2023-12-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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WITHDRAWN
NA
INTERVENTIONAL
2020-06-09
2021-05-15
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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).
Exercise
Surpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions.
Unsupervised exercise
Unsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home.
CFTR Modulators
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
Interventions
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Exercise
Surpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions.
Unsupervised exercise
Unsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home.
CFTR Modulators
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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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).
6 Years
18 Years
ALL
No
Sponsors
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Universidad Europea de Madrid
OTHER
Responsible Party
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Margarita Perez
Full Professor MD, PhD
Principal Investigators
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Margarita Perez-Ruiz, MD
Role: PRINCIPAL_INVESTIGATOR
Universidad Politecnica de Madrid
Locations
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Escuela de Doctorado e Investigacion, Universidad Europea
Villaviciosa de Odón, , Spain
Countries
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Other Identifiers
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CIPI/20/119
Identifier Type: -
Identifier Source: org_study_id