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Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis

Primary Purpose

Cystic Fibrosis in Children

Status

Unknown status

Phase

Not Applicable

Locations

Spain

Study Type

Interventional

Intervention

Exercise

Unsupervised exercise

CFTR Modulators

About this trial

This is an interventional treatment trial for Cystic Fibrosis in Children focused on measuring Cystic Fibrosis, Electrostimulation, Combined Training, Physical Fitness

Eligibility Criteria

6 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

Escuela de Doctorado e Investigacion, Universidad EuropeaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Multitreatment

Arm Description

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).

Outcomes

Primary Outcome Measures

Change in Strength

Changes in strength will be measured using a five repetition maximum test (5RM)

Change in Cardiorespiratory Fitness

Changes in cardiorespiratory fitness will be measured using a cardiopulmonary exercise test (CPET)

Secondary Outcome Measures

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)

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)

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)

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)

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.

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.

Sweat chloride level

Chloride concentration in sweat (mEq/L) will be measured in the laboratory using an MK II Chloride Analyzer 926S

Full Information

NCT ID

NCT04415268

First Posted

May 28, 2020

Last Updated

June 2, 2020

Sponsor

Universidad Europea de Madrid

1. Study Identification

Unique Protocol Identification Number

NCT04415268

Brief Title

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

2. Study Status

Record Verification Date

June 2020

Overall Recruitment Status

Unknown status

Study Start Date

June 9, 2020 (Anticipated)

Primary Completion Date

January 29, 2021 (Anticipated)

Study Completion Date

May 15, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Universidad Europea de Madrid

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis in Children

Keywords

Cystic Fibrosis, Electrostimulation, Combined Training, Physical Fitness

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Model Description

Single-group non-randomized, prospective. Within-subject multitreatment

Masking

None (Open Label)

Allocation

N/A

Enrollment

11 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Multitreatment

Arm Type

Experimental

Arm Description

Intervention Type

Behavioral

Intervention Name(s)

Exercise

Intervention Description

Surpervised exercise program: Includes a concurrent exercise intervention (strength training and aerobic training), 3 days a week, 60 minutes sessions.

Intervention Type

Behavioral

Intervention Name(s)

Unsupervised exercise

Intervention Description

Unsupervised exercise program: The same exercise program learned during the supervised phase will be practiced at home.

Intervention Type

Drug

Intervention Name(s)

CFTR Modulators

Other Intervention Name(s)

EudraCT Number 2019-000833-37

Intervention Description

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

Primary Outcome Measure Information:

Title

Change in Strength

Description

Changes in strength will be measured using a five repetition maximum test (5RM)

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Change in Cardiorespiratory Fitness

Description

Changes in cardiorespiratory fitness will be measured using a cardiopulmonary exercise test (CPET)

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Secondary Outcome Measure Information:

Title

Changes in Forced expiratory volume in 1 second (FEV1)

Description

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)

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Changes in Forced vital capacity (FVC)

Description

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)

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Changes in FEV1/FVC ratio (FEV1%)

Description

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)

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Changes in Forced expiratory flow (FEF)

Description

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)

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Changes in Physical Activity Questionnaire (PAQ) for children and adolescents

Description

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Change in quality of life: Cystic Fibrosis-Questionnaire-Revised (CFQ-R)

Description

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

Title

Sweat chloride level

Description

Chloride concentration in sweat (mEq/L) will be measured in the laboratory using an MK II Chloride Analyzer 926S

Time Frame

Four assessment points throughout the study: baseline and after each 8-week intervention

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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).

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Margarita Perez Ruiz, MD

Phone

+34912115200

Ext

3010

margarita.perez@universidadeuropea.es

First Name & Middle Initial & Last Name or Official Title & Degree

Veronica Sanz, MD

Phone

+34915035900

veross81@yahoo.es

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Margarita Perez Ruiz, MD

Organizational Affiliation

Universidad Europea de Madrid

Official's Role

Principal Investigator

Facility Information:

Facility Name

Escuela de Doctorado e Investigacion, Universidad Europea

City

Villaviciosa De Odón

ZIP/Postal Code

28670

Country

Spain

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Margarita Perez Ruiz, MD

Phone

+34912115200

Ext

3010

margarita.perez@universidadeuropea.es

First Name & Middle Initial & Last Name & Degree

Veronica Sanz, MD

Phone

+34915035900

veross81@yahoo.es

12. IPD Sharing Statement

Learn more about this trial

Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis

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