- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03521271
Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy
Study Overview
Status
Conditions
Detailed Description
Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease seen in childhood. DMD is an X linked recessive disorder. DMD is characterized by complete or partial (<3%) deficiency of the cell membrane protein dystrophin.
Dystrophin deficiency results in a permanent deterioration of muscle fibers. This leads to a progressive decrease in muscle strength and functional abilities. The precise mechanism of how the defect of dystrophin leads to degeneration of muscle fibers remains uncertain, but cytoskeletal deterioration, sarcolemmal instability and abnormal calcium homeostasis are thought to play a role in this degeneration.
These patients have symptoms such as limb proximals and progressive muscle weakness in the trunk, gait abnormalities, Gower's sign, various degrees of restriction in daily living activities, and frequent falls. The majority of patients have elevated serum creatine kinase or elevated liver transaminases and less frequently language or general developmental retardation.
Walking ability of this children begins to deteriorate between the ages of 3-6. These patients are generally dependent on wheelchair aged 10-12 years. The loss of walking is the milestone in terms of the progression of the disease. The use of electric wheelchairs limits arm functions such as lengthening and lifting during the late phase of the illness (when the ambulance has been lost and the ambulance is being continued with the wheelchair).
Patients with DMD have an average life span of 30 years with spinal surgery and ventilation support. They spend most of their lives dependent on the wheelchair and need functional use of the upper limbs to maintain the best possible level of independence in their daily life activities throughout their lifetimes.
Although muscle weakness in the proximal limbs is the first finding of the disease, the influence of upper extremity functions gives symptoms after 8 years of age and has a great influence on the level of independence of DMD patients in life since this process. For this reason, rehabilitation approaches for the protection of upper extremity functions from the early period of the disease are of great importance.Effective interventions are necessary to achieve this goal and these variables must be considered when making clinical decisions.
Patients with adult DMD have been shown to be able to perform important functional activities with limited distal motor function in the late phase of disease, but tend to lose these capacities as muscle strength decreases. For this reason, determining the factors (grip strength, thumb opposition, upper extremity joint range of motion, upper extremity muscle strength, etc.) that may affect hand functions, which is an important component in maintaining upper extremity functioning, and how these factors are related to the general functions of the upper extremity it is crucial that this disease leads to physiotherapy rehabilitation programs to be implemented.
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Samanpazarı/Ankara
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Ankara, Samanpazarı/Ankara, Turkey, 06100
- Hacettepe University
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria:
- Having a Duchenne Muscular Dystrophy diagnosis by a practicing physician,
- Between the ages of 8-16 years,
- Having lost ambulation ability,
- Brooke Upper Extremity Functional Classification (1-5)
- To have motivation and co-operation with physiotherapist for evaluations to be made
Exclusion Criteria:
- To maintain the capacity of independent ambulance,
- Having cooperative disorder or serious mental disorder,
- Any spinal and / or upper extremity injuries and / or surgery in the last 6 months,
- Having any systemic disease other than DMD,
- Not volunteering to participate in the study
Study Plan
How is the study designed?
Design Details
- Observational Models: Case-Only
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Brooke Upper Extremity Functional Classification (1-6)
Time Frame: 2 minutes
|
Children's functional levels were assessed with Brooke Upper Extremity Functional Classification (BUEFS) and children with a BUEFS score between 1-5 were included in the study.
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2 minutes
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Passive joint range of motion
Time Frame: 10 minutes
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Passive range of motions were assessed with goniometer and recorded limitations.
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10 minutes
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Upper extremity muscle strength
Time Frame: 20 minutes
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Hand held dynamometer
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20 minutes
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Thumb opposition
Time Frame: 2 minutes
|
Thumb opposition was assessed by Kapandji score (1-10). Score Location achieved
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2 minutes
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Lateral, tripod, two-point pinch strength
Time Frame: 3 minutes
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Pinchmeter
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3 minutes
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Performance of the upper extremity
Time Frame: 15 minutes
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Performance of the Upper Limb (PUL) (0-74) is including three dimension:
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15 minutes
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Hand function
Time Frame: 3 minutes
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ABILHAND-Kids (0-36)
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3 minutes
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Activity limitations
Time Frame: 3 minutes
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ACTIVLIM (0-36)
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3 minutes
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Gross grip strength
Time Frame: 2 minutes
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Hand dynamometer
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2 minutes
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Quality of life assessment of children (0-100)
Time Frame: 5 minutes
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PedsQL-Child report
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5 minutes
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Quality of life assessment of parents (0-100)
Time Frame: 5 minutes
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PedsQL-Parent report
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5 minutes
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- GO 17/307
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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