Duchenne Muscular DystrophyNCT03552874
Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy and Upper Limb Function, run by Hacettepe University.
Trial summary
Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage. Read more about this study
- Who is it for?
- Ages 5 to 10 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy, Upper Limb Function, Respiratory Function Test
- What is being tested?
- The Performance of Upper Limb Questionnaire (PUL)
- How many people
- 47 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2017to 31 Mar 2018
- 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 10 years
- Men only
- Healthy volunteers accepted
- Child
Study population: Fourty seven participants including 31 children with DMD and 16 healthy peers were included
Who may be able to join
- children with DMD was;
- age between 5-10 years
- being on treatment with corticosteroids for more than 6 months
- being at Level I according to Brooke Upper and Lower Extremity Classification Systems
- Healthy peers;
- age between 5-10 years
- not having any diagnosed disease
Who cannot join
- Children has undergo surgery and disease affect upper extremity or pulmonary function in last six months.
About this study
Official title: The Comparison of Pulmonary and Upper Extremity Functions Between Children With Duchenne Muscular Dystrophy and Healthy Peers
Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage. The aim of this study was to investigate the differences in pulmonary and upper limb functions between children with DMD in early stage and healthy peers.
- Being tested
- The Performance of Upper Limb Questionnaire (PUL)
- 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.
The Performance of Upper Limb Questionnaire (PUL)Other
Also called Pulmonary Function Test (PFT)
| Study group | Role | What they receive |
|---|---|---|
| Patient Group | Children whose ages between 5-10 years with Duchenne Muscular Dystrophy. Receives: Other: The Performance of Upper Limb Questionnaire (PUL) | |
| Healthy Group | Healthy peers Receives: Other: The Performance of Upper Limb Questionnaire (PUL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pulmonary Function Test (PFT)
The pulmonary function tests of the participants were evaluated with a spirometry. The child was asked to perform the spirometric tests for three times with maximum effort. The child was motivated before each attempt for stronger and longer expiration.The result of Pulmonary function Test were recorded.
Measured: 5 minutes
The Performance Upper Limb (PUL)
Upper limb functions of participants were evaluated with The Performance of Upper Limb (PUL) scale. This scale, which was shown to be reliable in DMD, has 22 items in total. It consists of 3 different levels of function: high, mid, and distal. PUL also has four items determining timed performance.
Measured: 15 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2018
- Study start1 Jan 2017
- Primary completion31 Jul 2017
- Study completion31 Mar 2018
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.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hacettepe University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Numan Bulut, Hacettepe University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03552874
- Sponsor's protocol ID
- GO 16/773
- Status last verified
- Jun 2018
- First submitted
- 25 May 2018
- Last updated
- 12 Jun 2018
- Results posted
- 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 12 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Comparison of Pulmonary and Upper Extremity Functions Between Children With Duchenne Muscular Dystrophy and Healthy Peers
- ClinicalTrials.gov ID
- NCT03552874
- Acronym / sponsor ID
- GO 16/773
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Hacettepe UniversityUniversity, hospital or other
- Study start
- 1 Jan 2017 (actual)
- Primary completion
- 31 Jul 2017 (actual)
- Study completion
- 31 Mar 2018 (actual)
- First posted
- 12 Jun 2018 (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.
Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage. The aim of this study was to investigate the differences in pulmonary and upper limb functions between children with DMD in early stage and healthy peers.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patient Group | – | Other: The Performance of Upper Limb Questionnaire (PUL)Children whose ages between 5-10 years with Duchenne Muscular Dystrophy. |
| Healthy Group | – | Other: The Performance of Upper Limb Questionnaire (PUL)Healthy peers |
Who may qualify
- Ages
- 5 to 10 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- Fourty seven participants including 31 children with DMD and 16 healthy peers were included
Key inclusion criteria (7 listed)
- children with DMD was;
- age between 5-10 years
- being on treatment with corticosteroids for more than 6 months
- being at Level I according to Brooke Upper and Lower Extremity Classification Systems
- Healthy peers;
Key exclusion criteria (1 listed)
- Children has undergo surgery and disease affect upper extremity or pulmonary function in last six months.
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 |
|---|---|---|
| The Performance of Upper Limb Questionnaire (PUL)Also: Pulmonary Function Test (PFT) | Other | Healthy Group, Patient Group |
Primary outcome
Pulmonary Function Test (PFT)
Time frame: 5 minutes
The pulmonary function tests of the participants were evaluated with a spirometry. The child was asked to perform the spirometric tests for three times with maximum effort. The child was motivated before each attempt for stronger and longer expiration.The result of Pulmonary function Test were recorded.
The Performance Upper Limb (PUL)
Time frame: 15 minutes
Upper limb functions of participants were evaluated with The Performance of Upper Limb (PUL) scale. This scale, which was shown to be reliable in DMD, has 22 items in total. It consists of 3 different levels of function: high, mid, and distal. PUL also has four items determining timed performance.
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 12 Jun 2018.
- 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.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy and Upper Limb Function, run by Hacettepe University.
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 7 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 12 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
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?
Pulmonary Function Test (PFT) (time frame: 5 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?
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 12 Jun 2018; AzTrials last refreshed its copy on 6 Nov 2026.