Duchenne Muscular DystrophyNCT06103006
Remote Physiotherapy to Protect Physical Health in Duchenne Muscular Dystrophy
A study testing Telerehabilitation in people with Duchenne Muscular Dystrophy, run by Lokman Hekim University at 1 site in Turkey (Türkiye).
Trial summary
Duchenne Muscular Dystrophy (DMD) is a progressive genetic neuromuscular disease characterized by progressive loss of motor function, respiratory failure, and cardiomyopathy required regular physiotherapy. Read more about this study
- Who is it for?
- Ages 5 to 26 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Telerehabilitation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 1 Jan 2021to 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 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Able to communicate verbally and visually,
- Older than 5 years old
Who cannot join
- Individuals who had undergone any surgical operations in the past 6 months,
- Had a severe cognitive and breathing impairment,
- Using mechanical ventilator continually or intermittent,
- Having febrile infection
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Remote Physiotherapy to Protect Physical Health in Duchenne Muscular Dystrophy: Telerehabilitation
Duchenne Muscular Dystrophy (DMD) is a progressive genetic neuromuscular disease characterized by progressive loss of motor function, respiratory failure, and cardiomyopathy required regular physiotherapy. With the outbreak of the pandemic rehabilitation centers that make up the weekly physiotherapy routine of children with disabilities have slowed down or even stopped their activities. So DMD who have additional diseases such as respiratory muscle weakness, spinal deformity, obesity, and cardiac dysfunction have also been negatively affected. The 'telerehabilitation' method, which is well planned and prepared for the abilities and needs of patients and caregivers, is seen as a good option at this point.
Studies, reporting the feasibility and safety of telerehabilitation in joint replacement, multiple sclerosis, and post-operative conditions, report that the length of stay was reduced, there was access to the same level of service regardless of the distance, and there was no travel cost. Despite these advantages, the framework and applicability of telerehabilitation programs have been investigated limited and not focused on effectiveness of telerehabilitation in patients with DMD.
According to the current knowledge, telerehabilitation in DMD is a subject that needs to be investigated in terms of its benefits. So, in this study, it was aimed to show the telerehabilitation's feasibility and its effects on performance level, endurance, fall frequency, pulmonary functions, and satisfaction level with the program in individuals with DMD.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Telerehabilitation
- 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.
TelerehabilitationOther
The structured telerehabilitation program focused on 4 specific objectives: breathing, stretching, posture, and stability. The duration was between 20-30 minutes. Exercises involving the major muscle groups (upper, lower extremity, and trunk muscle groups) were applied by the physiotherapists. Strengthening and stretching sets and repetitions were started with 1 set of 8-15 reps (progress to >1 set).
| Study group | Role | What they receive |
|---|---|---|
| Telerehabilitation Group | Experimental | Participants in this group will receive telerehabilitation exercises. Receives: Other: Telerehabilitation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Endurance
Number of times the individuals can flexed and extend their knees in a sitting position for 30 seconds. Also, the number of elbow flexion/extension was recorded
Measured: Change from baseline at 8 weeks
Modified Upper Extremity Performance Test
Flexion the shoulder to 90 degrees, flexion the shoulder above 90 degrees (above eye level), abduction of the shoulder 90 degrees, abduction of the shoulder above 90 degrees, doing the same movements with weight, bringing the empty glass to the mouth in a sitting position, bringing the full glass to the mouth were evaluated. It was scored as 0 point (cannot), 1 point (can do with compensation), 2 points (does independently). In addition, the time to pick up 5 coins (50 cents size coin or similar) on the table with one hand was recorded .
Measured: Change from baseline at 8 weeks
Secondary outcomes (7)
10 Meter (m) Walking Test
The distance of 10 m was determined in a suitable indoor environment, the individual was asked to walk this distance and the time that the patient walked was recorded.
Measured: Change from baseline at 8 weeks
Stand Up from the Supine Position Test
Depending on the severity of the disease, this period is extended. Depending on the weakness of the hip and shoulder girdle and trunk muscles, there is a movement model that the individuals use by climbing over himself. While the individual is getting up from the ground, the mother and the physiotherapist keep time with a stopwatch.
Measured: Change from baseline at 8 weeks
Single Breath Count (SBC)
Single breath count (SBC) is the maximum measure of serial counting of numbers in normal speaking voice after a maximal inhalation. SBC has good correlation with the gold standard measures of pulmonary function test (PFT), peak expiratory flow rate (PEFR), and forced expiratory volume in the first second (FEV1). The value reached is recorded when the patient counts audibly in a single expiration after maximum inspiratory effort. The instruction is: "Breathe well, count as many counts as possible while exhaling". The total number is recorded and evaluated in 4 categories: >30: 1, 20-29:2, 10-19: 3, <9:4.
Measured: Change from baseline at 8 weeks
Patient Satisfaction Survey
15 items survey was developed by the researchers. 5 likert pointed scale range was "5: excellent" and "1=bad". The items were about internet/connection, physiotherapist's guidance, timing, level of knowledge, reassurance, comfort, information/suggestions, the intelligibility of information, and general satisfaction. A total score was used to calculate percent value [(100 X Total score)/15].
Measured: After 8 weeks
Brooke Upper Extremity Scale
The Brooke scale was used to classify upper extremity functioning with scores from 1 to 6 (higher scores indicate worse functionality).
Measured: Change from baseline at 8 weeks
Brooke Lower Extremity Scale
The functional ability for all of the subjects with DMD was also ranked by a physical therapist using the Brooke Lower Extremity Functional Scale. This scale ranges from a grade of 1 (able to walk and climb stairs independently) to a grade of 10 (confined to bed).
Measured: Change from baseline at 8 weeks
Frequency of falling
The number of falls that occurred per day before the study and the frequency of falls after 8 weeks of exercise were questioned verbally.
Measured: Change from baseline at 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2023
- Study start1 Jan 2021
- Primary completion1 Jan 2022
- Study completion10 Feb 2022
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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lokman Hekim University
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Mustafa Sarı, Lokman Hekim University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06103006
- Sponsor's protocol ID
- 003/2021
- Status last verified
- Oct 2023
- First submitted
- 22 Oct 2023
- Last updated
- 27 Oct 2023
- 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 27 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Remote Physiotherapy to Protect Physical Health in Duchenne Muscular Dystrophy: Telerehabilitation
- ClinicalTrials.gov ID
- NCT06103006
- Acronym / sponsor ID
- 003/2021
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Lokman Hekim UniversityOther government
- Study start
- 1 Jan 2021 (actual)
- Primary completion
- 1 Jan 2022 (actual)
- Study completion
- 10 Feb 2022 (actual)
- First posted
- 26 Oct 2023 (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.
Duchenne Muscular Dystrophy (DMD) is a progressive genetic neuromuscular disease characterized by progressive loss of motor function, respiratory failure, and cardiomyopathy required regular physiotherapy. With the outbreak of the pandemic rehabilitation centers that make up the weekly physiotherapy routine of children with disabilities have slowed down or even stopped their activities. So DMD who have additional diseases such as respiratory muscle weakness, spinal deformity, obesity, and cardiac dysfunction have also been negatively affected. The 'telerehabilitation' method, which is well planned and prepared for the abilities and needs of patients and caregivers, is seen as a good option at this point.
Studies, reporting the feasibility and safety of telerehabilitation in joint replacement, multiple sclerosis, and post-operative conditions, report that the length of stay was reduced, there was access to the same level of service regardless of the distance, and there was no travel cost. Despite these advantages, the framework and applicability of telerehabilitation programs have been investigated limited and not focused on effectiveness of telerehabilitation in patients with DMD.
According to the current knowledge, telerehabilitation in DMD is a subject that needs to be investigated in terms of its benefits. So, in this study, it was aimed to show the telerehabilitation's feasibility and its effects on performance level, endurance, fall frequency, pulmonary functions, and satisfaction level with the program in individuals with DMD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Telerehabilitation Group | Experimental | Other: TelerehabilitationParticipants in this group will receive telerehabilitation exercises. |
Who may qualify
- Ages
- 5 to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Able to communicate verbally and visually,
- Older than 5 years old
Key exclusion criteria (4 listed)
- Individuals who had undergone any surgical operations in the past 6 months,
- Had a severe cognitive and breathing impairment,
- Using mechanical ventilator continually or intermittent,
- Having febrile infection
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 |
|---|---|---|
| TelerehabilitationThe structured telerehabilitation program focused on 4 specific objectives: breathing, stretching, posture, and stability. The duration was between 20-30 minutes. Exercises involving the major muscle groups (upper, lower extremity, and trunk muscle groups) were applied by the physiotherapists. Strengthening and stretching sets and repetitions were started with 1 set of 8-15 reps (progress to >1 set). | Other | Telerehabilitation Group |
Primary outcome
Endurance
Time frame: Change from baseline at 8 weeks
Number of times the individuals can flexed and extend their knees in a sitting position for 30 seconds. Also, the number of elbow flexion/extension was recorded
Modified Upper Extremity Performance Test
Time frame: Change from baseline at 8 weeks
Flexion the shoulder to 90 degrees, flexion the shoulder above 90 degrees (above eye level), abduction of the shoulder 90 degrees, abduction of the shoulder above 90 degrees, doing the same movements with weight, bringing the empty glass to the mouth in a sitting position, bringing the full glass to the mouth were evaluated. It was scored as 0 point (cannot), 1 point (can do with compensation), 2 points (does independently). In addition, the time to pick up 5 coins (50 cents size coin or similar) on the table with one hand was recorded .
7 secondary outcomes
10 Meter (m) Walking Test
Time frame: Change from baseline at 8 weeks
Stand Up from the Supine Position Test
Time frame: Change from baseline at 8 weeks
Single Breath Count (SBC)
Time frame: Change from baseline at 8 weeks
Patient Satisfaction Survey
Time frame: After 8 weeks
Brooke Upper Extremity Scale
Time frame: Change from baseline at 8 weeks
Brooke Lower Extremity Scale
Time frame: Change from baseline at 8 weeks
Frequency of falling
Time frame: Change from baseline at 8 weeks
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 27 Oct 2023.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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?
A study testing Telerehabilitation in people with Duchenne Muscular Dystrophy, run by Lokman Hekim 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 2 inclusion and 4 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 27 Oct 2023. 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?
Endurance (time frame: Change from baseline at 8 weeks). 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 27 Oct 2023.