Duchenne Muscular DystrophyNCT06295718
Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
An observational study of Duchenne Muscular Dystrophy, run by Sahra Şirvan at 1 site in Turkey (Türkiye).
Trial summary
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is being investigated. Read more about this study
- Who is it for?
- Ages 5 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 20 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 1 Jul 2023to 10 May 2024
- Phase
- Observational
- 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 5 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: This research is a descriptive type observational study, and the population of the study will be 20 volunteer cases who were diagnosed with Duchenne Muscular Dystrophy at Istanbul University-Cerrahpaşa Faculty of Medicine Child Neurology Polyclinic and who meet the exclusion criteria. The study will be carried out at Istanbul University-Cerrahpaşa Faculty of Health Sciences, Department of Physiotherapy and Rehabilitation.
Who may be able to join
- Being diagnosed with Duchenne muscular dystrophy
- Being between the ages of 5-18
- Having ambulation skills
- Having internet access and technical requirements
- Having the ability to follow movement instructions
- Volunteering to participate in the study
Who cannot join
- Having cognitive or behavioral problems that may interfere with evaluation
- Having communication problems that may prevent evaluation
- Having a level of contracture that may prevent evaluation
- Having a systemic disease that may prevent evaluation
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
İstanbul University - Cerrahpaşa
Istanbul, Istanbul, 34758
About this study
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is being investigated. However, studies have generally focused on a single evaluation parameter such as physical function, a special evaluation method or a special evaluation tool. The aim of this study is to investigate whether remote assessment of functional performance and quality of life in DMD patients is valid and reliable. If a valid and reliable tele-evaluation method that includes functional performance and quality of life parameters is found to be valid and reliable, the travel burden on patients and caregivers can be eased, patients\' stress and anxiety related to travel can be reduced, caregivers can save time and energy and provide patients with the best possible treatment.
Read the detailed description
DMD is a progressive muscle disease that causes difficulties in mobility and impairment of vital functions as a result of X chromosome-linked dystrophin deficiency. Symptoms appear between the ages of 3 and 5, and in untreated cases, loss of ambulation occurs around the age of 8-12. Long sitting periods due to wheelchair use increase the risk of contracture, osteopenia, fracture and scoliosis, and accompanying cardiorespiratory complications cause mortality in the early 20s in most cases. Determining functional performance in DMD is a guide for determining appropriate treatment options and objectively evaluating the effectiveness of treatment. However, the evaluation methods applied are performed in clinical environments that require frequent travel and require both time and physical effort. The aim of this study is to investigate whether remote assessment of functional performance and quality of life in DMD patients is valid and reliable. For this purpose, 20 volunteers diagnosed with DMD at Istanbul University-Cerrahpaşa Child Neurology Polyclinic and who meet the inclusion criteria will be evaluated in two ways, online and face-to-face, in terms of functional performance and quality of life parameters. In addition, scoring will be performed by two different physiotherapists during the evaluation and the reliability of the tele-evaluation among the evaluators will be examined. Functional performance timed performance tests, Brooke Upper Extremity Functional Classification and Vignos Lower Extremity Functional Classification; Quality of life will be evaluated with PedsQL-3.0 Neuromuscular Module and PedsQL Multidimensional Fatigue Scale. Thanks to valid and reliable tele-assessment methods for DMD patients, travel burden can be alleviated for both patients and caregivers, and disease-related stress and anxiety can be reduced. In this way, caregivers can save time and energy while providing the best care to patients.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Tele-assessment, Timed Performance Test, Quality of Life, Fatigue
- Medical subject headings
- Muscular Dystrophy, Duchenne, Fatigue
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Ambulatory Children with Duchenne Muscular Dystrophy | Participants who meet the inclusion criteria will be evaluated synchronously online using the Zoom platform in terms of the functional performance and quality of life parameters specified below, after ensuring the presence of a suitable environment and a stable internet connection. During the online evaluation, patients will be asked to perform the specified functions in front of the camera. In addition, the survey questions necessary to evaluate the quality of life will be expressed verbally to the participants. Inter-rater reliability will be examined by comparing the scores after being performed separately by two different physiotherapists. The reliability and validity of the online assessment will be investigated by repeating the same assessments online and face-to-face. * Timed Performance Tests * Upper Extremity Functions * Lower Extremity Functions * Quality of Life |
What is being measured
The primary outcome is the main question the study is designed to answer.
Timed Performance Tests
Timed performance tests are one of the frequently preferred outcome measures in patients with DMD. The time patients spend performing certain functions is recorded. The following functions will be evaluated in this study: • Getting up from the ground • Standing up from a chair • Don't wear a t-shirt • T-shirt removal • Collect 6 coins with one hand • Timed Up and Go Test
Measured: 2 days
Brooke Upper Extremity Functional Classification (BUEFS)
Brooke Upper Extremity Functional Classification (BUEFS) will be used to evaluate the upper extremity functions of the cases. BUEFS is a valid, reliable and practical scale used to classify the upper extremity functions of individuals with DMD. It was developed by Brooke et al. in 1981. Individuals are classified between 1 and 6 according to the upper extremity activities they can perform, and higher stages indicate decreased functionality of the upper extremity.
Measured: 2 days
Vignos Lower Extremity Functional Classification (VAEFS)
Vignos Lower Extremity Functional Classification (VAEFS) will be used to evaluate the lower extremity functions of the cases. VAEFS is a scale consisting of 10 stages that evaluates the level of ambulation in neuromuscular diseases. It was developed by Vignos et al. in 1963. Individuals are classified between 1 and 10 according to their ambulation level, and higher stages indicate a decrease in ambulation level. While the patient in the first stage can walk and climb stairs without support, in the highest stage he is bedridden.
Measured: 2 days
PedsQL-3.0 Neuromuscular Module by PedsQL Multidimensional Fatigue Scale
The patients' quality of life will be evaluated using the PedsQL-3.0 Neuromuscular Module- the PedsQL Multidimensional Fatigue Scale.
Measured: 2 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2024
- Study start1 Jul 2023
- Primary completion2 Mar 2024
- Study completion10 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sahra Şirvan
- Sponsor type
- University, hospital or other
- Collaborators
- Yonca Zenginler Yazgan, İrem Nur Şener
- Responsible party
- Sponsor-investigator, Sahra Şirvan, Istanbul University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06295718
- Sponsor's protocol ID
- 2023/51
- Status last verified
- May 2026
- First submitted
- 29 Feb 2024
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
- ClinicalTrials.gov ID
- NCT06295718
- Acronym / sponsor ID
- 2023/51
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Tele-assessment, Timed Performance Test, Quality of Life, Fatigue
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Sahra ŞirvanUniversity, hospital or other
- Collaborators
- Yonca Zenginler Yazgan, İrem Nur Şener
- Study start
- 1 Jul 2023 (actual)
- Primary completion
- 2 Mar 2024 (actual)
- Study completion
- 10 May 2024 (actual)
- First posted
- 6 Mar 2024 (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.
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is being investigated. However, studies have generally focused on a single evaluation parameter such as physical function, a special evaluation method or a special evaluation tool. The aim of this study is to investigate whether remote assessment of functional performance and quality of life in DMD patients is valid and reliable. If a valid and reliable tele-evaluation method that includes functional performance and quality of life parameters is found to be valid and reliable, the travel burden on patients and caregivers can be eased, patients\' stress and anxiety related to travel can be reduced, caregivers can save time and energy and provide patients with the best possible treatment.
Detailed description
DMD is a progressive muscle disease that causes difficulties in mobility and impairment of vital functions as a result of X chromosome-linked dystrophin deficiency. Symptoms appear between the ages of 3 and 5, and in untreated cases, loss of ambulation occurs around the age of 8-12. Long sitting periods due to wheelchair use increase the risk of contracture, osteopenia, fracture and scoliosis, and accompanying cardiorespiratory complications cause mortality in the early 20s in most cases. Determining functional performance in DMD is a guide for determining appropriate treatment options and objectively evaluating the effectiveness of treatment. However, the evaluation methods applied are performed in clinical environments that require frequent travel and require both time and physical effort. The aim of this study is to investigate whether remote assessment of functional performance and quality of life in DMD patients is valid and reliable. For this purpose, 20 volunteers diagnosed with DMD at Istanbul University-Cerrahpaşa Child Neurology Polyclinic and who meet the inclusion criteria will be evaluated in two ways, online and face-to-face, in terms of functional performance and quality of life parameters. In addition, scoring will be performed by two different physiotherapists during the evaluation and the reliability of the tele-evaluation among the evaluators will be examined. Functional performance timed performance tests, Brooke Upper Extremity Functional Classification and Vignos Lower Extremity Functional Classification; Quality of life will be evaluated with PedsQL-3.0 Neuromuscular Module and PedsQL Multidimensional Fatigue Scale. Thanks to valid and reliable tele-assessment methods for DMD patients, travel burden can be alleviated for both patients and caregivers, and disease-related stress and anxiety can be reduced. In this way, caregivers can save time and energy while providing the best care to patients.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ambulatory Children with Duchenne Muscular Dystrophy | – | –Participants who meet the inclusion criteria will be evaluated synchronously online using the Zoom platform in terms of the functional performance and quality of life parameters specified below, after ensuring the presence of a suitable environment and a stable internet connection. During the online evaluation, patients will be asked to perform the specified functions in front of the camera. In addition, the survey questions necessary to evaluate the quality of life will be expressed verbally to the participants. Inter-rater reliability will be examined by comparing the scores after being performed separately by two different physiotherapists. The reliability and validity of the online assessment will be investigated by repeating the same assessments online and face-to-face. * Timed Performance Tests * Upper Extremity Functions * Lower Extremity Functions * Quality of Life |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- This research is a descriptive type observational study, and the population of the study will be 20 volunteer cases who were diagnosed with Duchenne Muscular Dystrophy at Istanbul University-Cerrahpaşa Faculty of Medicine Child Neurology Polyclinic and who meet the exclusion criteria. The study will be carried out at Istanbul University-Cerrahpaşa Faculty of Health Sciences, Department of Physiotherapy and Rehabilitation.
Key inclusion criteria (6 listed)
- Being diagnosed with Duchenne muscular dystrophy
- Being between the ages of 5-18
- Having ambulation skills
- Having internet access and technical requirements
- Having the ability to follow movement instructions
Key exclusion criteria (4 listed)
- Having cognitive or behavioral problems that may interfere with evaluation
- Having communication problems that may prevent evaluation
- Having a level of contracture that may prevent evaluation
- Having a systemic disease that may prevent evaluation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Timed Performance Tests
Time frame: 2 days
Timed performance tests are one of the frequently preferred outcome measures in patients with DMD. The time patients spend performing certain functions is recorded. The following functions will be evaluated in this study: • Getting up from the ground • Standing up from a chair • Don't wear a t-shirt • T-shirt removal • Collect 6 coins with one hand • Timed Up and Go Test
Brooke Upper Extremity Functional Classification (BUEFS)
Time frame: 2 days
Brooke Upper Extremity Functional Classification (BUEFS) will be used to evaluate the upper extremity functions of the cases. BUEFS is a valid, reliable and practical scale used to classify the upper extremity functions of individuals with DMD. It was developed by Brooke et al. in 1981. Individuals are classified between 1 and 6 according to the upper extremity activities they can perform, and higher stages indicate decreased functionality of the upper extremity.
Vignos Lower Extremity Functional Classification (VAEFS)
Time frame: 2 days
Vignos Lower Extremity Functional Classification (VAEFS) will be used to evaluate the lower extremity functions of the cases. VAEFS is a scale consisting of 10 stages that evaluates the level of ambulation in neuromuscular diseases. It was developed by Vignos et al. in 1963. Individuals are classified between 1 and 10 according to their ambulation level, and higher stages indicate a decrease in ambulation level. While the patient in the first stage can walk and climb stairs without support, in the highest stage he is bedridden.
PedsQL-3.0 Neuromuscular Module by PedsQL Multidimensional Fatigue Scale
Time frame: 2 days
The patients' quality of life will be evaluated using the PedsQL-3.0 Neuromuscular Module- the PedsQL Multidimensional Fatigue Scale.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Sahra Şirvan at 1 site in Turkey (Türkiye).
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 6 inclusion and 4 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: finished, as of the sponsor's last update on 14 May 2026. 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 Turkey (Türkiye). 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Timed Performance Tests (time frame: 2 days). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 May 2026; AzTrials last refreshed its copy on 8 Nov 2026.