Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies. (DMDrespy2024)

Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy.

The purpose of this study is to analyze the effectiveness of a 5-weeks respiratory digital intervention program in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.

Study Overview

• Status: Enrolling by invitation
• Conditions: Muscular Dystrophy; Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
• Intervention / Treatment: Other: Digital respiratory intervention program

Detailed Description

It will be done 4 times a week for a period of 5 weeks, for a duration of 55-60 minutes (20 sessions), with an intensity adapted to the characteristics of each participant. An intervention program will be used based on respiratory muscle training exercises, upper limb strength work, yoga, diaphragmatic awareness exercises, always trying to do them through the use of games, and even try to include exercises that use motor imagery.

It is advisable during intervention program to have a playful environment stimulated for the children, and that the intervention program is carried out and stimulated through videos and therapeutic teaching to the caregivers in the first evaluation session, as well as with a weekly follow-up using WhatsApp as forms of communication, or a telephone call to ensure correct follow-up of the intervention program.

It is essential that to carry out the intervention, videos obtained from YouTube, own videos prepared to explain the exercises in a playful way applied to children, and even other applications such as Spotify to work with music, will be used, being offered in case of Zoom calls may be necessary to help optimally resolve doubts that arise when carrying out the program by video call.

At the end of the respiratory digital physiotherapy program, variables will be evaluated by the principal examiner.

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Spain
  • Málaga, Spain
    • Rocío Martín-Valero

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participants with Duchenne muscular dystrophy and patients with Becker muscular dystrophy will be chosen.
• Aged between 5 and 20 years.
• Child who could walk and follow the intervention program, and were diagnosed according to the International Classification of Diseases, 10 Revision. code (G71-01); and who had an NSSA score greater than 20.

Exclusion Criteria:

• Patients who present associated heart disease.
• Patients who presentor fractures, a heart rate greater than 120 beats per minute, or an oxygen saturation less than 89% will be excluded.
• Participants who do not have the capacity to have internet at home to perform the intervention, as well as all patients whose caregivers did not agree to sign the informed consent.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Patients with Duchenne muscular dystrophy and Becker muscular dystrophy; A group of 12 patients with Duchenne muscular dystrophy and Becker muscular dystrophy that will be following a digital respiratory physiotherapy program.

Other: Digital respiratory intervention program; A 4 times a week for a period of 5 weeks will be done, for a duration of 55-60 minutes (20 sessions), with an intensity adapted to the characteristics of each participant. Moreover, an intervention program will be used based on respiratory muscle training exercises, upper limb strength work, yoga, diaphragmatic awareness exercises, always trying to do them through the use of games, and even try to include exercises that use motor imagery. The intervention program is carried out and stimulated through videos and therapeutic teaching to the caregivers in the first evaluation session, as well as as well as with a weekly follow-up using WhatsApp or Zoom as forms of communication, or a telephone call to ensure correct follow-up of the intervention program. Moreover, YouTube videos, own videos prepared by the physiotherapy will be used to help optimally resolve doubts that arise when carrying out the program by video call.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Analyze the improvement of the forced vital capacity (FVC).	Forced vital capacity is a respiratory parameter which is obtained from spirometry and which provides objective, valid information. It will be performed using spirometry before and after the digital respiratory intervention program. The spirometer that will be used is the "EasyOne Air Spirometer that complies with ISO 26782 standards, following the national spirometric recommendations of the ATS/ERS 2019.	Baseline, up to 5 weeks.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Perform an anthropometric study of the patient's characteristics, taking into account weight; and also a general physical examination.	An anthropometric study of the patient's characteristics will be carried out, taking into account age, weight, as well as the average values that should be established based on age; as well as a general physical examination of the patient to analyze if they will be considered fit or not to carry out the program. The weight will be check by "SECA 700 MECHANICAL COLUMN SCALE WITH SECA 220 STADIOMETER", and the height will be analyze by a measuring tape.	Baseline, up to 5 weeks.
Perform an anthropometric study of the patient's characteristics, taking into account age.	An anthropometric study of the patient's characteristics will be carried out, taking into account age, as well as the average values that should be established based on age; as well as a general physical examination of the patient to analyze if they will be considered fit or not to carry out the program.	Baseline, up to 5 weeks.
Perform an anthropometric study of the patient's characteristics, taking into account height.	An anthropometric study of the patient's characteristics will be carried out, taking into account height, as well as the average values that should be established based on age; as well as a general physical examination of the patient to analyze if they will be considered fit or not to carry out the program.	Baseline, up to 5 weeks.
Perform an anthropometric study of the patient's characteristics, taking into account sex.	An anthropometric study of the patient's characteristics will be carried out, taking into account height, as well as the average values that should be established based on sex; as well as a general physical examination of the patient to analyze if they will be considered fit or not to carry out the program.	Baseline, up to 5 weeks.
Examine changes in forced expiratory pressure	Forced expiratory pressure is a respiratory parameter obtained through spirometry, which provides important information about the patient's respiratory status. Measuring it will be done with the same characteristics as that previously mentioned in the FVC.	Baseline, up to 5 weeks.
Analyze changes in maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP).	Changes in the strength of the inspiratory and expiratory muscles will be assessed using a pressure meter with a reading range of ±300 cm H20 and an accuracy of 3% (MicroRPM® model, Vyaire Medical GmbH, Hoechberg, Germany) following the national recommendations of the Spanish Society of Pulmonology and Thoracic Surgery (SEPAR) (16).	Baseline, up to 5 weeks.
Check if there are changes in the sniff nasal inspiratory pressure (SNIP)	The existence of changes in the diaphragm force index is assessed, measuring it in exactly the same way as the MIP and PEM.	Baseline, up to 5 weeks.
Evaluate the fatigue in patients using an EPInfant test.	The amount of effort the child perceives during the sessions is assessed. It shows 11 descriptors numbered from 0 to 10 and 5 verbal descriptors located every 2 levels of the numbered descriptors, along with a drawing of a child doing physical activity in an increasing manner, increasing in intensity from left to right. The higher the numerical value, the worse the result is considered.	Baseline, up to 5 weeks.
Evaluate the quality of life of patients using a scale called "Kindscreen-52".	52-item scale carried out in Ravens-Sieberer U and validated in Spanish by Rajmil L, which assesses physical and psychological well-being, mood and emotions, self-perception, autonomy and relationship with friends and social life. . The scores were transformed into a scale from 0 to 100 to facilitate their interpretation: the higher the score, the higher HRQoL.	Baseline, up to 5 weeks.
North Star Ambulatory Assessment o NSAA	Designed to capture the construct of ambulatory function in DMD. 17-item scale that grades performance of various functional skills on a scale from 0 (unable), 1 (completes independently but with modifications), and 2 (completed without compensation). Total score 0 - 34 with a higher score denoting a higher level of function. It includes two timed tests. Timed rise from floor (RFF) and timed 10 metre walk / run test (10MWR). Validated and regularly used in clinical trials as well as clinic. Well understood by families. The best score is 34 points.	Baseline, up to 5 weeks.

Collaborators and Investigators

• Sponsor: University of Malaga
• Investigators: Study Director: Rocío Martín-Valero, PhD, University of Malaga

Study record dates

Study Major Dates

• Study Start (Actual): February 5, 2025
• Primary Completion (Estimated): July 30, 2025
• Study Completion (Estimated): December 30, 2025

Study Registration Dates

• First Submitted: March 22, 2024
• First Submitted That Met QC Criteria: April 8, 2024
• First Posted (Actual): April 12, 2024

Study Record Updates

• Last Update Posted (Actual): April 1, 2025
• Last Update Submitted That Met QC Criteria: March 26, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: Pulmonary rehabilitation; Duchenne Muscular Dystrophy; Inspiratory muscle training; Breathing exercises; Becker Muscular Dystrophy; Expiratory muscle training
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: UMalagaDRIP

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No