Duchenne Muscular DystrophyNCT05244395
Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy
A study testing Duchenne Muscular Dystrophy Gait Assessment Scale in people with Duchenne Muscular Dystrophy and Gait Disorders in Children, run by Hacettepe University at 1 site in Turkey (Türkiye).
Trial summary
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which included 10 physiotherapists who had experience in the management of patients with DMD over… Read more about this study
- Who is it for?
- Ages 5 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Gait Disorders in Children, Gait Disorders, Neurologic
- What is being tested?
- Duchenne Muscular Dystrophy Gait Assessment Scale
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 10 Feb 2020to 10 Feb 2022
- 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 5 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Being diagnosed with DMD
- Be between Levels I-V according to Brooke Lower Limb Extremity Functional Classification
- Being between the ages of 5-18
- Agreeing to participate in the research voluntarily
Who cannot join
- Insufficient cooperation with the physiotherapist,
- Have had any injury and/or surgery of the lower extremities in the last 6 months,
- Having neurological problems in addition to DMD. -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy: Gait Assessment Scale for Duchenne Muscular Dystrophy, and Its Validity and Reliability
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which included 10 physiotherapists who had experience in the management of patients with DMD over the 2-round Delphi method, and the Content Validity Index (CVI) was calculated. The final version of the DMD-GAS that was agreed upon the experts consisted of 10 items, and each item scored between 0 and 2. The intra-rater reliability was established by the video analysis of children with a 1-month interval and inter-rater reliability was determined by the scores of 3 physiotherapists. The criterion validity was determined by investigating the relationship between the total score of the DMD-GAS and Motor Function Measure (MFM), 6 Minute Walk Test (6MWT), and the data obtained from GAITRite system.
- Being tested
- Duchenne Muscular Dystrophy Gait Assessment Scale
- Keywords
- Duchenne Muscular Dystrophy, gait pattern, scale development, reliability and validity, gait analysis
- Medical subject headings
- Muscular Dystrophy, Duchenne, Gait Disorders, Neurologic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Duchenne Muscular Dystrophy Gait Assessment ScaleOther
a special gait assessment scale for Duchenne Muscular Dystrophy patients
| Study group | Role | What they receive |
|---|---|---|
| Duchenne Muscular Dystrophy Gait Assessment Scale | Experimental | The DMD-GAS was developed using the two-round Delphi method. After a detailed literature review, the items of scale were compiled. The DMD-GAS was designed to consist of 10 items and each item scored as 0,1,2. The DMD-GAS was presented to the expert group (10 physical therapists), who had experience in the management of patients with DMD. According to the scale items, it is classified as 2: no compensation, 1: minimal compensation, and 0: excessive compensation for the relevant body part. Receives: Other: Duchenne Muscular Dystrophy Gait Assessment Scale |
What is being measured
The primary outcome is the main question the study is designed to answer.
Duchenne Muscular Dystrophy Gait Assessment Scale (DMD-GAS)
The scale which developed specifically for DMD with this study. The DMD-GAS was designed to consist of 10 items and each item scored as 0 (excessive compensation in the relevant body part), 1 (minimal compensation) or 2 (no compensation). Low score indicate compensated gait.
Measured: 15-20 minutes
Motor Function Measure (MFM)
MFM, which was valid and reliable in Neuromuscular Diseases, was used for gross motor function assessment. The items in MFM, which evaluate functions in 3 different sections (D1, standing position and transfers; D2, axial and proximal motor function; D3, distal motor function) in 32 items in total, are scored between 0-3. 0; cannot initiate any movement and maintain the starting position, 1; partially completes the exercise, 2; performs the exercise slowly and visibly clumsily, with compensations, 3; performs the exercise in the specified standard pattern. High scores indicate higher motor function and the result is expressed as a percentage of the maximum possible score to enable comparison with other scores
Measured: 20-30 minutes
Secondary outcomes (1)
6 meter walking test (6MWT)
6MWT is an assessment that was developed by Balke in the 1960s and has validity and reliability for DMD patients. The maximum distance that the patient can walk in 6 minutes is recorded as meters.
Measured: 6 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2022
- Study start10 Feb 2020
- Primary completion30 Nov 2021
- Study completion10 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Hacettepe UniversityThe 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hacettepe University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Güllü AYDIN YAĞCIOĞLU, Hacettepe University
- Investigators
- Güllü Aydın Yağcıoğlu, PhD, Principal investigator, Hacettepe University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05244395
- Sponsor's protocol ID
- GO 20/1058
- Status last verified
- Feb 2022
- First submitted
- 8 Feb 2022
- Last updated
- 7 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Aydin Yagcioglu G, Alemdaroglu Gurbuz I, Topuz S, Yilmaz O. Development of a new instrument to evaluate gait characteristics of individuals with Duchenne Muscular Dystrophy: Gait Assessment Scale for Duchenne Muscular Dystrophy, and its validity and reliability. Early Hum Dev. 2023 Oct;185:105843. doi: 10.1016/j.earlhumdev.2023.105843. Epub 2023 Aug 23.
PubMed 37672897
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy: Gait Assessment Scale for Duchenne Muscular Dystrophy, and Its Validity and Reliability
- ClinicalTrials.gov ID
- NCT05244395
- Acronym / sponsor ID
- GO 20/1058
- Keywords
- Duchenne Muscular Dystrophy, gait pattern, scale development, reliability and validity, gait analysis
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Hacettepe UniversityUniversity, hospital or other
- Study start
- 10 Feb 2020 (actual)
- Primary completion
- 30 Nov 2021 (actual)
- Study completion
- 10 Feb 2022 (actual)
- First posted
- 17 Feb 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.
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which included 10 physiotherapists who had experience in the management of patients with DMD over the 2-round Delphi method, and the Content Validity Index (CVI) was calculated. The final version of the DMD-GAS that was agreed upon the experts consisted of 10 items, and each item scored between 0 and 2. The intra-rater reliability was established by the video analysis of children with a 1-month interval and inter-rater reliability was determined by the scores of 3 physiotherapists. The criterion validity was determined by investigating the relationship between the total score of the DMD-GAS and Motor Function Measure (MFM), 6 Minute Walk Test (6MWT), and the data obtained from GAITRite system.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Duchenne Muscular Dystrophy Gait Assessment Scale | Experimental | Other: Duchenne Muscular Dystrophy Gait Assessment ScaleThe DMD-GAS was developed using the two-round Delphi method. After a detailed literature review, the items of scale were compiled. The DMD-GAS was designed to consist of 10 items and each item scored as 0,1,2. The DMD-GAS was presented to the expert group (10 physical therapists), who had experience in the management of patients with DMD. According to the scale items, it is classified as 2: no compensation, 1: minimal compensation, and 0: excessive compensation for the relevant body part. |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Being diagnosed with DMD
- Be between Levels I-V according to Brooke Lower Limb Extremity Functional Classification
- Being between the ages of 5-18
- Agreeing to participate in the research voluntarily
Key exclusion criteria (3 listed)
- Insufficient cooperation with the physiotherapist,
- Have had any injury and/or surgery of the lower extremities in the last 6 months,
- Having neurological problems in addition to DMD. -
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 |
|---|---|---|
| Duchenne Muscular Dystrophy Gait Assessment Scalea special gait assessment scale for Duchenne Muscular Dystrophy patients | Other | Duchenne Muscular Dystrophy Gait Assessment Scale |
Primary outcome
Duchenne Muscular Dystrophy Gait Assessment Scale (DMD-GAS)
Time frame: 15-20 minutes
The scale which developed specifically for DMD with this study. The DMD-GAS was designed to consist of 10 items and each item scored as 0 (excessive compensation in the relevant body part), 1 (minimal compensation) or 2 (no compensation). Low score indicate compensated gait.
Motor Function Measure (MFM)
Time frame: 20-30 minutes
MFM, which was valid and reliable in Neuromuscular Diseases, was used for gross motor function assessment. The items in MFM, which evaluate functions in 3 different sections (D1, standing position and transfers; D2, axial and proximal motor function; D3, distal motor function) in 32 items in total, are scored between 0-3. 0; cannot initiate any movement and maintain the starting position, 1; partially completes the exercise, 2; performs the exercise slowly and visibly clumsily, with compensations, 3; performs the exercise in the specified standard pattern. High scores indicate higher motor function and the result is expressed as a percentage of the maximum possible score to enable comparison with other scores
1 secondary outcome
6 meter walking test (6MWT)
Time frame: 6 minutes
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 7 Mar 2022.
- Derived from this study: PubMed 37672897Aydin Yagcioglu G, Alemdaroglu Gurbuz I, Topuz S, Yilmaz O. Development of a new instrument to evaluate gait characteristics of individuals with Duchenne Muscular Dystrophy: Gait Assessment Scale for Duchenne Muscular Dystrophy, and its validity and reliability. Early Hum Dev. 2023 Oct;185:105843. doi: 10.1016/j.earlhumdev.2023.105843. Epub 2023 Aug 23.
- Individual participant dataSharing planned: Undecided
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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.
Güllü Aydın Yağcıoğlu, PhD principal investigator
Hacettepe University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Duchenne Muscular Dystrophy Gait Assessment Scale in people with Duchenne Muscular Dystrophy and Gait Disorders in Children, run by Hacettepe University 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 4 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: finished, as of the sponsor's last update on 7 Mar 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 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?
Duchenne Muscular Dystrophy Gait Assessment Scale (DMD-GAS) (time frame: 15-20 minutes). 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 NCT05244395, which also lists 1 linked publication.
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 7 Mar 2022.