Duchenne Muscular DystrophyNCT03521271
Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Hacettepe University at 1 site in Turkey (Türkiye).
Trial summary
The aim of this study, determining the factors affecting the hand functions of children with Duchenne Muscular Dystrophy who have lost their independent ambulatory ability and determining the effects on the overall upper extremity performance and quality of life of the determined factors. Read more about this study
- Who is it for?
- Ages 8 to 16 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 23 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 5 Apr 2017to 26 Jan 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 8 to 16 years
- Men only
- No healthy volunteers
- Child
Study population: Patients who applied to the Pediatric Neuromuscular Diseases Unıt of Hacettepe University Physiotherapy and Rehabilitation Department
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Hacettepe University
Ankara, Samanpazarı/Ankara, 06100
About this study
The aim of this study, determining the factors affecting the hand functions of children with Duchenne Muscular Dystrophy who have lost their independent ambulatory ability and determining the effects on the overall upper extremity performance and quality of life of the determined factors.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Upper limb, Hand functions, Performance, Grip Strength
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
Brooke Upper Extremity Functional Classification (1-6)
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.
- Starting with arms at sides, can abduct arms in a full circle until they touch above head
- Can raise arms above head only by flexing elbow or using accesory muscles
- Cannot raise arms above head but can raise a glass of water to mouth (using both hands if necessary)
- Can raise hands to mouth but cannot raise a glass of water to mouth
- Cannot raise hand to mouth but can use hands to hold pen or pick up pennies from table
- Cannot raise hands to mouth and has no useful function of hands
Measured: 2 minutes
Passive joint range of motion
Passive range of motions were assessed with goniometer and recorded limitations.
Measured: 10 minutes
Upper extremity muscle strength
Hand held dynamometer
Measured: 20 minutes
Thumb opposition
Thumb opposition was assessed by Kapandji score (1-10). Score Location achieved
- Radial side of the proximal phalanx of the 2nd phalanx
- Radial side of the middle phalanx of the 2nd phalanx
- Tip of the 2nd phalanx
- Tip of the 3th phalanx
- Tip of the 4th phalanx
- Tip of the 5th phalanx
- Distal interphalangeal joint crease of the 5th phalanx
- Proximal interphalangeal joint crease of the 5th phalanx
- Metacarpophalangeal joint crease of the 5th phalanx
- Distal palmar crease
Measured: 2 minutes
Lateral, tripod, two-point pinch strength
Pinchmeter
Measured: 3 minutes
Performance of the upper extremity
Performance of the Upper Limb (PUL) (0-74) is including three dimension:
- Shoulder dimension (0-16)
- Elbow dimension (0-34)
- Distal dimension (0-24)
Measured: 15 minutes
Hand function
ABILHAND-Kids (0-36)
Measured: 3 minutes
Activity limitations
ACTIVLIM (0-36)
Measured: 3 minutes
Gross grip strength
Hand dynamometer
Measured: 2 minutes
Secondary outcomes (2)
Quality of life assessment of children (0-100)
PedsQL-Child report
Measured: 5 minutes
Quality of life assessment of parents (0-100)
PedsQL-Parent report
Measured: 5 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2018
- Study start5 Apr 2017
- Primary completion26 Jan 2018
- Study completion26 Jan 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hacettepe University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gülcan Altınok, Hacettepe University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03521271
- Sponsor's protocol ID
- GO 17/307
- Status last verified
- May 2018
- First submitted
- 19 Apr 2018
- Last updated
- 11 May 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03521271
- Acronym / sponsor ID
- GO 17/307
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Upper limb, Hand functions, Performance, Grip Strength
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Hacettepe UniversityUniversity, hospital or other
- Study start
- 5 Apr 2017 (actual)
- Primary completion
- 26 Jan 2018 (actual)
- Study completion
- 26 Jan 2018 (actual)
- First posted
- 11 May 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.
The aim of this study, determining the factors affecting the hand functions of children with Duchenne Muscular Dystrophy who have lost their independent ambulatory ability and determining the effects on the overall upper extremity performance and quality of life of the determined factors.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Patients who applied to the Pediatric Neuromuscular Diseases Unıt of Hacettepe University Physiotherapy and Rehabilitation Department
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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
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
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. 1. Starting with arms at sides, can abduct arms in a full circle until they touch above head 2. Can raise arms above head only by flexing elbow or using accesory muscles 3. Cannot raise arms above head but can raise a glass of water to mouth (using both hands if necessary) 4. Can raise hands to mouth but cannot raise a glass of water to mouth 5. Cannot raise hand to mouth but can use hands to hold pen or pick up pennies from table 6. Cannot raise hands to mouth and has no useful function of hands
Passive joint range of motion
Time frame: 10 minutes
Passive range of motions were assessed with goniometer and recorded limitations.
Upper extremity muscle strength
Time frame: 20 minutes
Hand held dynamometer
Thumb opposition
Time frame: 2 minutes
Thumb opposition was assessed by Kapandji score (1-10). Score Location achieved 1. Radial side of the proximal phalanx of the 2nd phalanx 2. Radial side of the middle phalanx of the 2nd phalanx 3. Tip of the 2nd phalanx 4. Tip of the 3th phalanx 5. Tip of the 4th phalanx 6. Tip of the 5th phalanx 7. Distal interphalangeal joint crease of the 5th phalanx 8. Proximal interphalangeal joint crease of the 5th phalanx 9. Metacarpophalangeal joint crease of the 5th phalanx 10. Distal palmar crease
Lateral, tripod, two-point pinch strength
Time frame: 3 minutes
Pinchmeter
Performance of the upper extremity
Time frame: 15 minutes
Performance of the Upper Limb (PUL) (0-74) is including three dimension: * Shoulder dimension (0-16) * Elbow dimension (0-34) * Distal dimension (0-24)
Hand function
Time frame: 3 minutes
ABILHAND-Kids (0-36)
Activity limitations
Time frame: 3 minutes
ACTIVLIM (0-36)
Gross grip strength
Time frame: 2 minutes
Hand dynamometer
2 secondary outcomes
Quality of life assessment of children (0-100)
Time frame: 5 minutes
Quality of life assessment of parents (0-100)
Time frame: 5 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 11 May 2018.
- 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.
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 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 5 inclusion and 5 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 11 May 2018. 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?
Brooke Upper Extremity Functional Classification (1-6) (time frame: 2 minutes). The study lists 9 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 11 May 2018.