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

NCT04415268 | Withdrawn

• 1 other identifier: CIPI/20/119
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis in Children

Keywords

Cystic Fibrosis; Electrostimulation; Combined Training; Physical Fitness

Outcome Measures

Primary Outcomes (2)

• Change in Strength

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

  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)

  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)

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

• Changes in Forced vital capacity (FVC)

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

• Changes in FEV1/FVC ratio (FEV1%)

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

• Changes in Forced expiratory flow (FEF)

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

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

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

Study Arms (1)

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).

Behavioral: Exercise; Behavioral: Unsupervised exercise; Drug: CFTR Modulators

Interventions

Exercise BEHAVIORAL

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

Multitreatment

Unsupervised exercise BEHAVIORAL

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

Multitreatment

CFTR Modulators DRUG

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

Also known as: EudraCT Number 2019-000833-37

Multitreatment

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Universidad Europea de Madrid: lead

Study Sites (1)

Escuela de Doctorado e Investigacion, Universidad Europea

Villaviciosa de Odón, 28670, Spain

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Exercise

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Study Officials

• Margarita Perez-Ruiz, MD

  Universidad Politecnica de Madrid

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Full Professor MD, PhD

Model Details: Single-group non-randomized, prospective. Within-subject multitreatment

Study Record Dates

• First Submitted: May 28, 2020
• First Posted: June 4, 2020
• Study Start: June 9, 2020
• Primary Completion: January 29, 2021
• Study Completion: May 15, 2021
• Last Updated: December 11, 2023

Record last verified: 2023-12

Locations

ES (1)