Cystic Fibrosis in ChildrenNCT04415268
Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis
A study testing Exercise, Unsupervised exercise and CFTR Modulators in people with Cystic Fibrosis in Children, run by Universidad Europea de Madrid at 1 site in Spain.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Cystic Fibrosis in Children
- What is being tested?
- Exercise, Unsupervised exercise and CFTR Modulators
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteSpain
- Study dates
- 9 Jun 2020to 15 May 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
Spain1 site
Escuela de Doctorado e Investigacion, Universidad Europea
Villaviciosa de Odón, 28670
About this study
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
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.
Read the 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.
- Conditions
- Cystic Fibrosis in Children
- Being tested
- Exercise, Unsupervised exercise, CFTR Modulators
- Keywords
- Cystic Fibrosis, Electrostimulation, Combined Training, Physical Fitness
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExerciseBehavioural
Surpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions.
Unsupervised exerciseBehavioural
Unsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home.
CFTR ModulatorsDrug
Also called EudraCT Number 2019-000833-37
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
| Study group | Role | What they receive |
|---|---|---|
| Multitreatment | Experimental | 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). Receives: Behavioral: Exercise; Behavioral: Unsupervised exercise; Drug: CFTR Modulators |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Strength
Changes in strength will be measured using a five repetition maximum test (5RM)
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Change in Cardiorespiratory Fitness
Changes in cardiorespiratory fitness will be measured using a cardiopulmonary exercise test (CPET)
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Secondary outcomes (7)
Changes in Forced expiratory volume in 1 second (FEV1)
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)
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in Forced vital capacity (FVC)
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)
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in FEV1/FVC ratio (FEV1%)
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)
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in Forced expiratory flow (FEF)
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)
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in Physical Activity Questionnaire (PAQ) for children and adolescents
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.
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Change in quality of life: Cystic Fibrosis-Questionnaire-Revised (CFQ-R)
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.
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Sweat chloride level
Chloride concentration in sweat (mEq/L) will be measured in the laboratory using an MK II Chloride Analyzer 926S
Measured: Four assessment points throughout the study: baseline and after each 8-week intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2020
- Study start9 Jun 2020 (estimated)
- Primary completion29 Jan 2021 (estimated)
- Study completion15 May 2021 (estimated)
Why it stopped: we did not receive expected funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis in ChildrenStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single-group non-randomized, prospective. Within-subject multitreatment
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universidad Europea de Madrid
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Margarita Perez, Universidad Europea de Madrid
- Investigators
- Margarita Perez-Ruiz, MD, Principal investigator, Universidad Politecnica de Madrid
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04415268
- Sponsor's protocol ID
- CIPI/20/119
- Status last verified
- Dec 2023
- First submitted
- 28 May 2020
- Last updated
- 11 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04415268
- Acronym / sponsor ID
- CIPI/20/119
- Conditions
- Cystic Fibrosis in Children
- Keywords
- Cystic Fibrosis, Electrostimulation, Combined Training, Physical Fitness
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: we did not receive expected funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Universidad Europea de MadridUniversity, hospital or other
- Study start
- 9 Jun 2020 (estimated)
- Primary completion
- 29 Jan 2021 (estimated)
- Study completion
- 15 May 2021 (estimated)
- First posted
- 4 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-group non-randomized, prospective. Within-subject multitreatment
| Arm | Type | Receives |
|---|---|---|
| Multitreatment | Experimental | Behavioral: Exercise, Behavioral: Unsupervised exercise, Drug: CFTR ModulatorsPharmacological 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). |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ExerciseSurpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions. | Behavioural | Multitreatment |
| Unsupervised exerciseUnsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home. | Behavioural | Multitreatment |
| CFTR ModulatorsAlso: EudraCT Number 2019-000833-37Standard 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 | Drug | Multitreatment |
Primary outcome
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)
7 secondary outcomes
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 Forced vital capacity (FVC)
Time frame: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in FEV1/FVC ratio (FEV1%)
Time frame: Four assessment points throughout the study: baseline and after each 8-week intervention
Changes in Forced expiratory flow (FEF)
Time frame: Four assessment points throughout the study: baseline and after each 8-week intervention
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
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
Sweat chloride level
Time frame: Four assessment points throughout the study: baseline and after each 8-week intervention
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margarita Perez-Ruiz, MD principal investigator
Universidad Politecnica de Madrid
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Exercise, Unsupervised exercise and CFTR Modulators in people with Cystic Fibrosis in Children, run by Universidad Europea de Madrid at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 11 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Strength (time frame: Four assessment points throughout the study: baseline and after each 8-week intervention). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Dec 2023.