Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis
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| ClinicalTrials.gov Identifier: NCT04415268 |
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Recruitment Status :
Recruiting
First Posted : June 4, 2020
Last Update Posted : June 4, 2020
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis in Children | Behavioral: Exercise Behavioral: Unsupervised exercise Drug: CFTR Modulators | Not Applicable |
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 : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 11 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Intervention Model Description: | Single-group non-randomized, prospective. Within-subject multitreatment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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 |
| Estimated Study Start Date : | June 9, 2020 |
| Estimated Primary Completion Date : | January 29, 2021 |
| Estimated Study Completion Date : | May 15, 2021 |
| Arm | Intervention/treatment |
|---|---|
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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). |
Behavioral: Exercise
Surpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions. Behavioral: Unsupervised exercise Unsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home. Drug: 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 Name: EudraCT Number 2019-000833-37 |
- Change in Strength [ Time Frame: 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 [ Time Frame: 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)
- Changes in Forced expiratory volume in 1 second (FEV1) [ Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention ]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)
- Changes in Forced vital capacity (FVC) [ Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention ]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)
- Changes in FEV1/FVC ratio (FEV1%) [ Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention ]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)
- Changes in Forced expiratory flow (FEF) [ Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention ]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)
- 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 ]
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.
- 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 ]
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.
- Sweat chloride level [ Time Frame: Four assessment points throughout the study: baseline and after each 8-week intervention ]Chloride concentration in sweat (mEq/L) will be measured in the laboratory using an MK II Chloride Analyzer 926S
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| Ages Eligible for Study: | 6 Years to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04415268
| Contact: Margarita Perez Ruiz, MD | +34912115200 ext 3010 | margarita.perez@universidadeuropea.es | |
| Contact: Veronica Sanz, MD | +34915035900 | veross81@yahoo.es |
| Spain | |
| Escuela de Doctorado e Investigacion, Universidad Europea | Recruiting |
| Villaviciosa De Odón, Spain, 28670 | |
| Contact: Margarita Perez Ruiz, MD +34912115200 ext 3010 margarita.perez@universidadeuropea.es | |
| Contact: Veronica Sanz, MD +34915035900 veross81@yahoo.es | |
| Principal Investigator: | Margarita Perez Ruiz, MD | Universidad Europea de Madrid |
| Responsible Party: | Margarita Perez, Full Professor in Exercise Physiology, MD, PhD, Universidad Europea de Madrid |
| ClinicalTrials.gov Identifier: | NCT04415268 |
| Other Study ID Numbers: |
CIPI/20/119 |
| First Posted: | June 4, 2020 Key Record Dates |
| Last Update Posted: | June 4, 2020 |
| Last Verified: | June 2020 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Cystic Fibrosis Electrostimulation Combined Training Physical Fitness |
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Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |