Duchenne Muscular Dystrophy (DMD)E-PULMoDMDNCT05516745
E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"
A study testing AioCare spirometer and telerehabiliation of the respiratory system in people with Duchenne Muscular Dystrophy (DMD), run by Medical University of Gdansk at 1 site in Poland.
Trial 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… Read more about this study
- Who is it for?
- Ages 7 to 17 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- AioCare spirometer and telerehabiliation of the respiratory system
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 sitePoland
- Study dates
- 20 Mar 2021to Mar 2025
- 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 7 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- no consent to participate in the study;
- patients under 7 years of age or above 18 years of age;
- inability to perform spirometry
Trial locations and contacts
1 site in 1 country.
Eliza Wasilewska, MD,PhD contact
Poland1 site, 1 recruiting
- Recruiting
Medical University
Gdansk, 80-292
Eliza Wasilewska, MD,PhD, +48 56 349 2625, ewasilewska@gumed.edu.pl
About this study
Official title: E-monitoring of Pulmonary Function in Patients With Duchenne Muscular Dystrophy Undergoing Respiratory Rehabilitation at Home"
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).
Read the 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:
- 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).
- 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).
- to compare the results from home electronic spirometry (AioCare) with results from hospital spirometry (Jaeger, Germany) - (value of FVC%pv, L).
- 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).
- to evaluate the impact of home telerehabilitation on pulmonary function (number of patients with increased FVC%pv, L value)
- 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.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- AioCare spirometer, telerehabiliation of the respiratory system
- Keywords
- AioCare spirometer, home spirometry, spirometry e-monitoring, pulmonary rehabilitation, digital medicine, e-health
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AioCare spirometerDevice
In the first part of the trial, all participants from arms 1 and 2 will receive the AioCare spirometer (Healtup, Poland) to home-based monitoring pulmonary function. Hospital spirometry examinations (Jaeger, Germany) evaluating disease progression will be performed periodically. There are to be follow-up visits after 3 months and after 6 months. The end-point visit is planned for 12 months.
telerehabiliation of the respiratory systemOther
A major component of respiratory dysfunction seems to be a decline of respiratory muscle weakness. The proposition is respiratory telerehabilitation to improve the function of respiratory muscles. Telerehabilitation includes breathing exercises for use at home. The participants will be trained in the hospital and will receive video presentations to support the exercises at home.
| Study group | Role | What they receive |
|---|---|---|
| arm with intervention (AioCare spirometry) | Experimental | 50 participants with DMD aged 7-17 years, subjected to home electronic monitoring Receives: Device: AioCare spirometer |
| arm with intervention (AioCare spirometry with telerehabilitation) | Experimental | 50 participants with DMD aged 7-17 years, subjected to home electronic monitoring with the AioCare device and pulmonary rehabilitation exercises Receives: Device: AioCare spirometer; Other: telerehabiliation of the respiratory system |
| arm with intervention (telerehabilitation) | Experimental | 50 participants with DMD aged 7-17 years, subjected to pulmonary rehabilitation exercises Receives: Other: telerehabiliation of the respiratory system |
| control arm (no intervention) | No intervention | 50 participants with DMD aged 7-17 years, subject to a standard of care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.
Measured: 12 months
Change from Baseline of the mean Forced Vital Capacity in %predicted value measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation
Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.
Measured: 12 months
Secondary outcomes (3)
Possibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic
- number of the days with performed spirometry test per patient during the monitoring period
- number and percent of correct spirometry parameters.
Measured: 4 weeks
The number of the participants who performed at least one correct spirometry examination
Feasibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic measured by The number of the participants who performed at least one correct spirometry examination
Measured: 4 weeks
the difference in the value of spirometry results (FVC %pv, L) between home spirometry and spirometry in the hospital
Feasibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic measured by The number of the participants who performed at least one correc the difference in the value of spirometry results (FVC %pv, L) between home spirometry and spirometry in the hospital
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2022
- Study start20 Mar 2021
- Primary completionFeb 2025 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by Medical University of GdanskThe sponsor's other registered studies.
- 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
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Model description
- 4 arms - 1. arm - AioCare device spirometry 2. arm - AioCare device spirometry+ telerehabilitation of the respiratory system 3. arm - telerehabilitation of the respiratory system 4. arm - control arm - standard of care (no monitoring, no telerehabilitation)
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of Gdansk
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Eliza Wasilewska, MD,PhD, Principal investigator, Medical University Gdansk
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05516745
- Sponsor's protocol ID
- EPULMoDMD 001/2021
- Acronym
- E-PULMoDMD
- Status last verified
- Aug 2022
- First submitted
- 22 Sep 2021
- Last updated
- 26 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.
PubMed 29395989Birnkrant DJ, Bushby K, Bann CM, Alman BA, Apkon SD, Blackwell A, Case LE, Cripe L, Hadjiyannakis S, Olson AK, Sheehan DW, Bolen J, Weber DR, Ward LM; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. Epub 2018 Feb 3.
PubMed 29395990Finder JD, Birnkrant D, Carl J, Farber HJ, Gozal D, Iannaccone ST, Kovesi T, Kravitz RM, Panitch H, Schramm C, Schroth M, Sharma G, Sievers L, Silvestri JM, Sterni L; American Thoracic Society. Respiratory care of the patient with Duchenne muscular dystrophy: ATS consensus statement. Am J Respir Crit Care Med. 2004 Aug 15;170(4):456-65. doi: 10.1164/rccm.200307-885ST. No abstract available.
PubMed 15302625Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.
PubMed 11751186Toussaint M, Chatwin M, Soudon P. Mechanical ventilation in Duchenne patients with chronic respiratory insufficiency: clinical implications of 20 years published experience. Chron Respir Dis. 2007;4(3):167-77. doi: 10.1177/1479972307080697.
PubMed 17711917LoMauro A, D'Angelo MG, Aliverti A. Sleep Disordered Breathing in Duchenne Muscular Dystrophy. Curr Neurol Neurosci Rep. 2017 May;17(5):44. doi: 10.1007/s11910-017-0750-1.
PubMed 28397169Miller MR, Hankinson J, Brusasco V, Burgos F, Casaburi R, Coates A, Crapo R, Enright P, van der Grinten CP, Gustafsson P, Jensen R, Johnson DC, MacIntyre N, McKay R, Navajas D, Pedersen OF, Pellegrino R, Viegi G, Wanger J; ATS/ERS Task Force. Standardisation of spirometry. Eur Respir J. 2005 Aug;26(2):319-38. doi: 10.1183/09031936.05.00034805. No abstract available.
PubMed 16055882
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- E-monitoring of Pulmonary Function in Patients With Duchenne Muscular Dystrophy Undergoing Respiratory Rehabilitation at Home"
- ClinicalTrials.gov ID
- NCT05516745
- Acronym / sponsor ID
- E-PULMoDMD / EPULMoDMD 001/2021
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- AioCare spirometer, home spirometry, spirometry e-monitoring, pulmonary rehabilitation, digital medicine, e-health
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Medical University of GdanskUniversity, hospital or other
- Study start
- 20 Mar 2021 (actual)
- Primary completion
- Feb 2025 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 26 Aug 2022 (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.
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:
- 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).
- 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).
- to compare the results from home electronic spirometry (AioCare) with results from hospital spirometry (Jaeger, Germany) - (value of FVC%pv, L).
- 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).
- to evaluate the impact of home telerehabilitation on pulmonary function (number of patients with increased FVC%pv, L value)
- 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 design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- 4 arms - 1. arm - AioCare device spirometry 2. arm - AioCare device spirometry+ telerehabilitation of the respiratory system 3. arm - telerehabilitation of the respiratory system 4. arm - control arm - standard of care (no monitoring, no telerehabilitation)
| Arm | Type | Receives |
|---|---|---|
| arm with intervention (AioCare spirometry) | Experimental | Device: AioCare spirometer50 participants with DMD aged 7-17 years, subjected to home electronic monitoring |
| arm with intervention (AioCare spirometry with telerehabilitation) | Experimental | Device: AioCare spirometer, Other: telerehabiliation of the respiratory system50 participants with DMD aged 7-17 years, subjected to home electronic monitoring with the AioCare device and pulmonary rehabilitation exercises |
| arm with intervention (telerehabilitation) | Experimental | Other: telerehabiliation of the respiratory system50 participants with DMD aged 7-17 years, subjected to pulmonary rehabilitation exercises |
| control arm (no intervention) | No intervention | –50 participants with DMD aged 7-17 years, subject to a standard of care |
Who may qualify
- Ages
- 7 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- no consent to participate in the study;
- patients under 7 years of age or above 18 years of age;
- inability to perform spirometry
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 |
|---|---|---|
| AioCare spirometerIn the first part of the trial, all participants from arms 1 and 2 will receive the AioCare spirometer (Healtup, Poland) to home-based monitoring pulmonary function. Hospital spirometry examinations (Jaeger, Germany) evaluating disease progression will be performed periodically. There are to be follow-up visits after 3 months and after 6 months. The end-point visit is planned for 12 months. | Device | arm with intervention (AioCare spirometry with telerehabilitation), arm with intervention (AioCare spirometry) |
| telerehabiliation of the respiratory systemA major component of respiratory dysfunction seems to be a decline of respiratory muscle weakness. The proposition is respiratory telerehabilitation to improve the function of respiratory muscles. Telerehabilitation includes breathing exercises for use at home. The participants will be trained in the hospital and will receive video presentations to support the exercises at home. | Other | arm with intervention (AioCare spirometry with telerehabilitation), arm with intervention (telerehabilitation) |
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
Time frame: 12 months
Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.
Change from Baseline of the mean Forced Vital Capacity in %predicted value measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation
Time frame: 12 months
Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.
3 secondary outcomes
Possibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic
Time frame: 4 weeks
The number of the participants who performed at least one correct spirometry examination
Time frame: 4 weeks
the difference in the value of spirometry results (FVC %pv, L) between home spirometry and spirometry in the hospital
Time frame: 4 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2022.
- Results publication: PubMed 29395989Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.
- Results publication: PubMed 29395990Birnkrant DJ, Bushby K, Bann CM, Alman BA, Apkon SD, Blackwell A, Case LE, Cripe L, Hadjiyannakis S, Olson AK, Sheehan DW, Bolen J, Weber DR, Ward LM; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. Epub 2018 Feb 3.
- Results publication: PubMed 15302625Finder JD, Birnkrant D, Carl J, Farber HJ, Gozal D, Iannaccone ST, Kovesi T, Kravitz RM, Panitch H, Schramm C, Schroth M, Sharma G, Sievers L, Silvestri JM, Sterni L; American Thoracic Society. Respiratory care of the patient with Duchenne muscular dystrophy: ATS consensus statement. Am J Respir Crit Care Med. 2004 Aug 15;170(4):456-65. doi: 10.1164/rccm.200307-885ST. No abstract available.
- Results publication: PubMed 11751186Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.
- Results publication: PubMed 17711917Toussaint M, Chatwin M, Soudon P. Mechanical ventilation in Duchenne patients with chronic respiratory insufficiency: clinical implications of 20 years published experience. Chron Respir Dis. 2007;4(3):167-77. doi: 10.1177/1479972307080697.
- Results publication: PubMed 28397169LoMauro A, D'Angelo MG, Aliverti A. Sleep Disordered Breathing in Duchenne Muscular Dystrophy. Curr Neurol Neurosci Rep. 2017 May;17(5):44. doi: 10.1007/s11910-017-0750-1.
- Results publication: PubMed 16055882Miller MR, Hankinson J, Brusasco V, Burgos F, Casaburi R, Coates A, Crapo R, Enright P, van der Grinten CP, Gustafsson P, Jensen R, Johnson DC, MacIntyre N, McKay R, Navajas D, Pedersen OF, Pellegrino R, Viegi G, Wanger J; ATS/ERS Task Force. Standardisation of spirometry. Eur Respir J. 2005 Aug;26(2):319-38. doi: 10.1183/09031936.05.00034805. No abstract available.
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RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
Eliza Wasilewska, MD,PhD contact
Eliza Wasilewska, MD,PhD principal investigator
Medical University Gdansk
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing AioCare spirometer and telerehabiliation of the respiratory system in people with Duchenne Muscular Dystrophy (DMD), run by Medical University of Gdansk at 1 site in Poland.
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 3 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: status not confirmed, as of the sponsor's last update on 26 Aug 2022. 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 Poland. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Eliza Wasilewska, MD,PhD (+48 56 349 2625, ewasilewska@gumed.edu.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 12 months). 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?
On the ClinicalTrials.gov record for NCT05516745, which also lists 7 linked publications.
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 26 Aug 2022.