E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home”

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Brief Title

E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"

Official Title

E-monitoring of Pulmonary Function in Patients With Duchenne Muscular Dystrophy Undergoing Respiratory Rehabilitation at Home"

Brief Summary

      Duchenne muscular dystrophy (DMD) is the most common, progressive, irreversible muscular
      dystrophy. The pulmonary function is crucial for the duration of life in this disease. The
      European Respiratory Society is currently focused on digital health, seeking to define the
      realistic innovations for digital respiratory medicine to support professionals and patients
      during the COVID-19 pandemic. This study aimed to investigate whether it is possible to
      monitor pulmonary function at home by using an individual electronical spirometry system in
      children with Duchenne muscular dystrophy DMD. The second aim of the study is the
      implementation of respiratory telerehabilitation and the assessment of its impact on
      pulmonary function (FVC).
    

Detailed Description

      Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy. DMD is a genetically
      determined, progressive, irreversible disease in which dystrophin dysfunction in skeletal and
      multiple organ muscles is fatal before the age of 20 years. Respiratory muscle failure is the
      most common cause of death.

      In the first years of life, the respiratory system of children with DMD is efficient and does
      not differ from that of healthy peers. From the age of 7 years, the parameters of lung
      function no longer increase, and between the ages of 10 to 12 years, when the child loses the
      ability to walk independently, the lung function rapidly deteriorates.

      Currently, it is not possible to cure the disease, but appropriate medical management may
      improve the quality of life and prolong the survival of patients with DMD.

      The basic tasks include the initiation of early monitoring of respiratory system functions.
      It is recommended that measurement of lung function is started from the age of 5 years.
      Taking the measurement at such an early age is aimed at familiarizing and teaching the child
      about this type of systematic examination and determining the individual maximum parameters
      of lung function in each child. Thanks to systematic measurements, it is possible to detect
      any sharp deterioration as well as the moment when the decreasing lung function requires
      respiratory support, so-called non-invasive ventilation (NIV).

      According to the standards, lung function is assessed by spirometry, which should be
      performed at least once a year, at least every 6 months after losing independent walking, and
      every 3 months after starting non-invasive ventilation.

      The spirometry test assesses forced vital capacity (FVC), which is considered a marker of
      disease progression. An FVC value below 2.1 L is a rationale to start supporting the cough
      reflex, and below 1 L is an indication to start respiratory support, i.e. NIV.

      Implementation of the above-mentioned tests often encounters difficulties that increase when
      the child loses independent walking. Additionally, during the COVID pandemic, spirometry was
      included in the procedures generating aerosols, i.e. high risk of SARS-CoV-2 virus
      transmission. Therefore, it has become necessary to look for other methods of measuring and
      monitoring lung function in children with DMD.

      The presented project aims to evaluate the measurement of lung function at home using an
      individual spirometer called an AIOCARE.

      The AioCare spirometer is a small, convenient device that can be used anywhere. The device
      enables systematic non-invasive monitoring of lung parameters (including FVC measurement) at
      home in children over 5 years of age. The child inhales and exhales forcefully through a
      mouthpiece with antibacterial and antiviral filters. The test results are sent from the
      AioCare spirometer via the AioCare application for iOS and Android (as used by all current
      smartphones). Communication between the AioCare spirometer and the application takes place
      via a Bluetooth 4.0 (BT LE) connection. The spirometry results are available to the
      practitioner in real-time in the AIOCARE Doctor panel.

      Additionally, a module with exercises for training respiratory muscles, included in an
      additional smartphone application, will be added to the daily spirometry test as part of the
      project.

      It will be a series of 4 exercises aimed at strengthening respiratory muscles, to be
      performed before the spirometry test.

      The assumption of the exercises is the possibility of performing them independently, at home,
      without the assistance of a physiotherapist, and without the use of additional equipment.
      Participants will perform forced exhalation exercises with a relaxed epiglottis, exercises to
      improve the mechanics of the chest and the mobility of the shoulder girdle. The exercises
      will be performed each day. The duration of the exercises is approximately 7 minutes. The
      exercise program will be recorded in the form of an instructional video.

      Pulmonary rehabilitation is one of the key issues in DMD patient management. The combination
      of systematic lung function measurement with home respiratory rehabilitation is an innovative
      project. It is a non-invasive test, and the measurement is intended to improve the quality of
      life of DMD patients.

      Aims of the study:

        1. to assess pulmonary function for 12 months using home electronic monitoring (AioCare
           System) in DMD participants aged 7-18 years (number of participants with increase FVC
           %pv, L value).

        2. to assess the acceptance of electronic home monitoring of a respiratory system in this
           group of patients (number of participants accepted home e-monitoring, survey).

        3. to compare the results from home electronic spirometry (AioCare) with results from
           hospital spirometry (Jaeger, Germany) - (value of FVC%pv, L).

        4. to investigate whether it is possible to conduct respiratory physical therapy with the
           use of telerehabilitation in DMD patients (number of patients accepted
           telerehabilitation, survey).

        5. to evaluate the impact of home telerehabilitation on pulmonary function (number of
           patients with increased FVC%pv, L value)

        6. to assess the quality of life of patients subjected to electronic monitoring and
           pulmonary rehabilitation vs. not subjected

      Study group: 200 participants with DMD aged 7-18 years, in the 4 arms (50 participants each):
      (1) subjected to home electronic monitoring exercises with the AIOCARE device, (2) home
      electronic monitoring with AioCareand pulmonary rehabilitation; (3) telerehabilitation of
      respiratory system; (4) control group - no interventions.

      Duration of the project March 2021 - March 2025.
    


Study Type

Interventional


Primary Outcome

Change from Baseline of the mean Forced Vital Capacity in Liters measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation

Secondary Outcome

 Possibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic

Condition

Duchenne Muscular Dystrophy (DMD)

Intervention

AioCare spirometer

Study Arms / Comparison Groups

 arm with intervention (AioCare spirometry)
Description:  50 participants with DMD aged 7-17 years, subjected to home electronic monitoring

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

200

Start Date

March 20, 2021

Completion Date

March 2025

Primary Completion Date

February 2025

Eligibility Criteria

        Inclusion Criteria:

          -  male, ≥7 years and <18 years of age at the time of enrollment in the study;

          -  ability to perform spirometry;

          -  stated willingness to comply with all study procedures and availability for the
             duration of the study.

        Exclusion Criteria:

          -  no consent to participate in the study;

          -  patients under 7 years of age or above 18 years of age;

          -  inability to perform spirometry
      

Gender

Male

Ages

7 Years - 17 Years

Accepts Healthy Volunteers

No

Contacts

Eliza Wasilewska, MD,PhD, +48 56 349 2625, [email protected]

Location Countries

Poland

Location Countries

Poland

Administrative Informations


NCT ID

NCT05516745

Organization ID

EPULMoDMD 001/2021


Responsible Party

Sponsor

Study Sponsor

Medical University of Gdansk


Study Sponsor

Eliza Wasilewska, MD,PhD, Principal Investigator, Medical University Gdansk


Verification Date

August 2022