Duchenne Muscular DystrophyNCT05274555
Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy and Upper Extremity Problem, run by Dokuz Eylul University at 1 site in Turkey (Türkiye).
Trial summary
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD). Materials and methods: A total of 41 children with DMD have participated in the study. Read more about this study
- Who is it for?
- Ages 5 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Upper Extremity Problem
- What is being tested?
- Assessment
- How many people
- 41 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 27 Mar 2019to 29 Jun 2021
- 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 study was planned as a methodological research. A total of 41 children with DMD between the ages of 5-18 were included in the study.
Who may be able to join
- being diagnosed with DMD, being between the ages of 5-18, not having any additional neurological disorder, volunteering to participate in the research
Who cannot join
- patients diagnosed after 10 years of age, patients who had never taken corticosteroids, and patients aged 14 years or older but were still ambulatory
Trial locations and contacts
1 site in 1 country.
About this study
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD).
Materials and methods: A total of 41 children with DMD have participated in the study. Upper and lower extremities functional levels were assessed with Vignos Scale and Brooke Upper Extremity Functional Rating Scale, respectively. The construct validity of the questionnaire was determined using the correlation between the ULSQ and ABILHAND-Kids. The Cronbach alpha value was calculated to determine internal consistency. To determine test-retest reliability, 17 randomly selected children were evaluated seven days after the first evaluation, and the "Intraclass Correlation Coefficient (ICC)" value was calculated.
- Being tested
- Assessment
- 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.
AssessmentOther
The Upper Limb Short Questionnaire was administered by the researcher in face-to-face sessions with the children and their caregivers.
The lower extremity functions and ambulation levels of the children were evaluated by the researcher using the Vignos Scale.
The Brooke Upper Extremity Functional Rating Scale and ABILHAND-Kids were used to evaluate children's upper extremity functions.
What is being measured
The primary outcome is the main question the study is designed to answer.
Upper Limb Short Questionnaire
The Upper Limb Short Questionnaire consists of questions about UE function (5 items), pain (6 items), and stiffness (3 items). ULSQ can be used as an identifier of arm-hand limitations during the clinical investigation. The total score changes between 0 and 14. Lower scores present children have more problems in the upper extremities.
Measured: 2 years
ABILHAND-Kids
That evaluates upper extremity function according to 18 different activities performed by children. The total score calculates by summing the grades that children get from each item and change between 0 and 36. Lower scores present lower ability while higher scores indicate higher ability of the hand/upper extremity in the ABILHAND-Kids.
Measured: 2 years
The Vignos Scale
This scale classifies patients' walking abilities in 10 grades ranging from 1 to 10. At the first level, the patient can walk and climb the steps without assistance, but at the last level, patient is confined to bed.
Measured: 2 years
The Brooke Upper Extremity Functional Rating Scale
It classifies upper extremity function with 6 different levels based on the children's upper extremity movements. Level 1 means patient can start the movement with the arms at the sides and fully joins the hands above the head, Level 6 is defined as unable to raise their hands to their mouths and cannot use their hands functionally.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2022
- Study start27 Mar 2019
- Primary completion25 Apr 2021
- Study completion29 Jun 2021
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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dokuz Eylul University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dilan Savaş, Dokuz Eylul University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05274555
- Sponsor's protocol ID
- 2019/07-65
- Status last verified
- Jun 2022
- First submitted
- 2 Mar 2022
- Last updated
- 9 Jun 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05274555
- Acronym / sponsor ID
- 2019/07-65
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Dokuz Eylul UniversityUniversity, hospital or other
- Study start
- 27 Mar 2019 (actual)
- Primary completion
- 25 Apr 2021 (actual)
- Study completion
- 29 Jun 2021 (actual)
- First posted
- 10 Mar 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.
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD).
Materials and methods: A total of 41 children with DMD have participated in the study. Upper and lower extremities functional levels were assessed with Vignos Scale and Brooke Upper Extremity Functional Rating Scale, respectively. The construct validity of the questionnaire was determined using the correlation between the ULSQ and ABILHAND-Kids. The Cronbach alpha value was calculated to determine internal consistency. To determine test-retest reliability, 17 randomly selected children were evaluated seven days after the first evaluation, and the "Intraclass Correlation Coefficient (ICC)" value was calculated.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- This study was planned as a methodological research. A total of 41 children with DMD between the ages of 5-18 were included in the study.
Key inclusion criteria (1 listed)
- being diagnosed with DMD, being between the ages of 5-18, not having any additional neurological disorder, volunteering to participate in the research
Key exclusion criteria (1 listed)
- patients diagnosed after 10 years of age, patients who had never taken corticosteroids, and patients aged 14 years or older but were still ambulatory
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 |
|---|---|---|
| AssessmentThe Upper Limb Short Questionnaire was administered by the researcher in face-to-face sessions with the children and their caregivers. The lower extremity functions and ambulation levels of the children were evaluated by the researcher using the Vignos Scale. The Brooke Upper Extremity Functional Rating Scale and ABILHAND-Kids were used to evaluate children's upper extremity functions. | Other | – |
Primary outcome
Upper Limb Short Questionnaire
Time frame: 2 years
The Upper Limb Short Questionnaire consists of questions about UE function (5 items), pain (6 items), and stiffness (3 items). ULSQ can be used as an identifier of arm-hand limitations during the clinical investigation. The total score changes between 0 and 14. Lower scores present children have more problems in the upper extremities.
ABILHAND-Kids
Time frame: 2 years
That evaluates upper extremity function according to 18 different activities performed by children. The total score calculates by summing the grades that children get from each item and change between 0 and 36. Lower scores present lower ability while higher scores indicate higher ability of the hand/upper extremity in the ABILHAND-Kids.
The Vignos Scale
Time frame: 2 years
This scale classifies patients' walking abilities in 10 grades ranging from 1 to 10. At the first level, the patient can walk and climb the steps without assistance, but at the last level, patient is confined to bed.
The Brooke Upper Extremity Functional Rating Scale
Time frame: 2 years
It classifies upper extremity function with 6 different levels based on the children's upper extremity movements. Level 1 means patient can start the movement with the arms at the sides and fully joins the hands above the head, Level 6 is defined as unable to raise their hands to their mouths and cannot use their hands functionally.
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 9 Jun 2022.
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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 and Upper Extremity Problem, run by Dokuz Eylul 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 1 inclusion and 1 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 9 Jun 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?
Upper Limb Short Questionnaire (time frame: 2 years). 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 9 Jun 2022.