Neuromuscular DiseasesNCT05544994
The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy
A study testing Aerobic Exercise Training and Home exercise program in people with Neuromuscular Diseases and Spinal Muscular Atrophy Type 3, run by Istanbul Medipol University Hospital at 1 site in Turkey (Türkiye).
Trial summary
In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments.One of these new treatments, Nusinersen, targets SMN2, which is a slightly different copy of SMN1, and increases SMN protein… Read more about this study
- Who is it for?
- Ages 10 to 50 years
- Conditions the study is for
- Neuromuscular Diseases, Spinal Muscular Atrophy Type 3
- What is being tested?
- Aerobic Exercise Training and Home exercise program
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 28 Jul 2022to 30 Sep 2024
- 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 10 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Have been diagnosed of SMA Type 3 confirmed by genetic analysis,
- Between the ages of 10-50,
- Being able to walk 25 m without assistive device
Who cannot join
- Using research drugs for SMA treatment other than Nusinersen treatment,
- Having a serious systemic disease that may prevent exercise,
- Have had a lower extremity injury and/or surgery in the last 6 months,
- To be applying a regular aerobic training program in the last 6 months
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site, 1 recruiting
- Recruiting
Istanbul Faculty of Medicine, Department of Neurological Sciences
Istanbul, Fatih
Sezan Mergen KILIÇ, MSc PT, 05324569628, sezanmer@hotmail.com
About this study
Official title: The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy"
In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments.One of these new treatments, Nusinersen, targets SMN2, which is a slightly different copy of SMN1, and increases SMN protein levels.
Preclinical studies have provided evidence that neuroprotection is strongly formed, with exercise significantly increasing motor neuron survival independent of SMN expression.
Read more
In a limited number of clinical studies prior to Nusinersen treatment, it was reported that aerobic exercise training improved maximum oxygen uptake (VO2 max) without causing muscle damage, but still caused fatigue.
The aim of this study is to determine the effect of aerobic exercise training on motor and respiratory functions, exercise capacity, fatigue and quality of life in SMA Type III patients who can walk and receive Nusinersen therapy. Twenty cases aged 10-50 years with genetically confirmed SMA diagnosis will be included in this study. The cases to be included in the study will be randomized into 2 groups as the training and control groups.
In addition to the routine physiotherapy program, medium-intensity Aerobic Exercise Training will be given to the study group for 12 weeks. Before and 12 weeks after the training, the cases will be evaluated with the Six Minute Walking Test, Submaximal Exercise Test, SMN protein level, function and strength assessments, (FVC) value, fatigue and quality of life scales.
In clinical trials, the supporting evidence for aerobic interventions in SMA is limited.
Additional studies on aerobic intervention parameters (frequency, intensity and duration) are needed.The results of this study will determine the feasibility of aerobic exercise training and provide important guidance for the clinical management of SMA patients.
Read the detailed description
Spinal Muscular Atrophy (SMA) is a neuromuscular disorder characterized by degeneration of alpha motor neurons in the spinal cord, leading to progressive muscle atrophy and weakness. Mutation or deletion of the survival motor neuron1 (SMN1) gene causes insufficient production of the SMN protein, which ensures motor neuron survival and maintenance in the spinal cord.
In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments. One of these new treatments, Nusinersen targets SMN2, which is a slightly different copy of SMN1, increases SMN protein levels. Preclinical studies have provided evidence that exercise significantly increases motor neuron survival independent of SMN expression, and that neuroprotection is strongly formed. In a limited number of clinical studies prior to Nuinersen therapy, it was reported that aerobic exercise training improved maximum oxygen uptake (VO2 max) without causing muscle damage, but still caused fatigue and had no significant beneficial effect on physical functioning. This study aims to determine the effect of aerobic exercise training on motor and respiratory functions, exercise capacity, fatigue and quality of life in SMA Type III patients who can walk and receive Nusinersen treatment. Twenty patients between the ages of 10 and 50 with genetically confirmed SMA diagnosis will be included in this study. The patients will be randomly divided into 2 groups as the training and control groups. In addition to the routine physiotherapy program, the study group will be given a moderate-intensity Aerobic Exercise Training for 12 weeks. Before and after 12 weeks of training, subjects will be evaluated with the Six-Minute Walk Test, ,Submaximal Exercise Test, SMN protein level and function and strength assessments, (FVC) value, fatigue and quality of life scales.
In clinical trials, the supporting evidence for aerobic interventions in SMA is limited. It has been reported that SMA patients cannot tolerate excessive exercise intensity and that recovery may be prevented due to excessive fatigue. Additional studies on aerobic intervention parameters (frequency, intensity and duration) are needed. The results of this study will determine the feasibility of aerobic exercise training and will have an impact on clinical practice by providing important guidance to the clinical management of SMA patients.
- Being tested
- Aerobic Exercise Training, Home exercise program
- Keywords
- Spinal Muscular Atrophy Type 3, Aerobic Training, SMN protein level, Physiotherapy
- Medical subject headings
- Neuromuscular Diseases, Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aerobic Exercise TrainingOther
Aerobic training will be performed 3 days a week for 12 weeks at 60%-%75 of their maximum hearth rate with 30 minutes total duration consisting of 5 min warm up and 5 min cool down period in treatment group.
Home exercise programOther
Home exercise program will be given. This home program will include stretching, breathing, normal joint movement, for 3 to 5 days a week.
| Study group | Role | What they receive |
|---|---|---|
| Aerobic Exercise Training Group | Experimental | Aerobic training will be performed 3 days a week for 12 weeks at 60% -%75 of their maximum hearth rate with 30 minutes total duration consisting of 5 min warm up and 5 min cool down period in treatment group.
Home exercise program will be given. This home program will include stretching, breathing, normal joint movement for 3 to 5 days a week Receives: Other: Aerobic Exercise Training; Other: Home exercise program |
| Control Group | Active comparator | Home exercise program will be given. This home program will include stretching, breathing, normal joint movement, for 3 or 5 days a week. Receives: Other: Home exercise program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional Capacity
Change from baseline functional capacity test at 12 weeks. Functional capacity will be assessed by the 6 minute walking test.The 6MWT, an objective evaluation of functional capacity, measures the maximum distance a person can walk in six minutes over a 25-meter linear course.
Measured: Baseline and 12 weeks
Secondary outcomes (10)
Submaximal Exercise Test
Change from baseline Submaximal Exercise Capacity Test at 12 weeks. It will be evaluated by Incremental Submaximal Exercise Test. The tests will be performed by using an cycle ergometer.
Measured: Baseline and 12 weeks
Pulmonary Function
Change from baseline Forced Vital Capacity (FVC) in respiratory function test at 12 weeks. Pulmonary Function will be assessed by measuring forced expiratory vital capacity (FVC) as percent predicted for age and height.
Measured: Baseline and 12 weeks
Motor Function
Change from baseline Hammersmith Functional Motor Scale, Expanded (HFMSE) at 12 weeks . The HFMSE is a 33-item scale designed for SMA type 2 and 3 patients.Items are scored on a 0-2 scale with a full score of 2 being normal without assistance, a partial score of 1 requiring a modified method but no assistance, and a zero score being unable to achieve the task without assistance.
Measured: Baseline and 12 weeks
Motor Performance
Change from baseline 10 Meter Walk/Run Test at 12 weeks . This test measures the time it takes a subject to walk or run 10 meters as fast as possible without compromising the subject's safety.
Measured: Baseline and 12 weeks
Balance and Mobility
Change from baseline Timed Up and Go Test (TUG) Test at 12 weeks The TUG test, which is a method to assess balance and mobility, measures the time it takes an individual to stand up from a chair, walk 3 meters, turn around, and sit down in the same chair.Ten seconds or less indicate normal mobility.
Measured: Baseline and 12 weeks
Muscle strength
Change from baseline Manual Muscle Testing (MMT) at 12 weeks Manual muscle testing (MMT) augmented by hand held dynamometry (HHD) provides a comprehensive evaluation of muscle strength.
Measured: Baseline and 12 weeks
Quality of Life in patients with neuromuscular disease
Change from baseline quality of life scale at 12 weeks The Pediatric quality of life™ 3.0 Neuromuscular Module (PedsQL™ 3.0 NMM) was designed to assess health-related quality of life (HRQoL) among aged 2-18 years with NMD.The scale consists of 25 items under 3 categories. .Items are scored on a Likert-type scale from 0 (never poses a problem) to 4 (always poses a problem). Scoring is between 0-100 .Higher scores from the Module indicate better health-related quality of life.For patients 18 years and older, The Short Form (SF-36), a widely used health-related quality of life measure. The Short Form (SF-36) Health Survey is a 36-item, patient-reported survey of patient health.
Measured: Baseline and at 12 weeks
Fatigue
Change from baseline Fatigue Scale at 12 weeks.
The PedsQL Multidimensional Fatigue Scale comprises parallel child self-report and parent proxy-reportformats. A 5-point response scale is utilized across child and a dolescent self-report for ages 8-18 and paren proxy-report .
Items are reverse scored and linearly transformed to a 0-100 scale.Higher PedsQL Multidimen-sional Fatigue Scale scores indicate better HRQOL.
The Fatigue Severity Scale (FSS) is a 9-item scale which measures the severity of fatigue and its effect on a person's activities and lifestyle in adult patients with a variety of disorders .A self-report scale of nine items about fatigue,The higher the score, the more severe the fatigue is and the more it affects the person's activities.
Measured: Baseline and 12 weeks
Functional Lower Extremity Strength,
Change from baseline Five Times Sit to Stand Test at 12 weeks.
The five Times Sit to Stand Test( 5XSS) scoring is based on the amount of time a patient is able to transfer from a seated to a standing position and back to sitting five times.
The lower the time to complete the test the better the outcome of the test. The Minimal Detectable Change(MDC) time for the test is within 3.6 to 4.2 second and Minimal clinically important difference (MCID) is 2.3 seconds.
Measured: Baseline and 12 weeks
Evaluation of SMN protein levels in blood biochemistry
Change from baseline SMN protein levels at 12 weeks. The response of patients' SMN protein levels to exercise will be evaluated before and after 12 weeks of training.
Measured: Baseline and 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2022
- Study start28 Jul 2022
- Primary completion30 Jun 2024 (estimated)
- Study completion30 Sep 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Investigator, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Istanbul Medipol University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Istanbul University
- Responsible party
- Principal investigator, sezanmergen, Istanbul Medipol University Hospital
- Investigators
- Sezan Mergen KILIÇ, MSc PT, Principal investigator, Istanbul University, Istanbul Faculty of Medicine,Department of Neurological SciencesFatma Karantay Mutluay,, Professor, Study director, Medipol University, Health Sciences Faculty, Physiotherapy and RehabilitationFatma Yeşim Parman, Professor, Study director, Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05544994
- Sponsor's protocol ID
- Aerobic Training
- Status last verified
- Sep 2023
- First submitted
- 19 Aug 2022
- Last updated
- 28 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- 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 28 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy"
- ClinicalTrials.gov ID
- NCT05544994
- Acronym / sponsor ID
- Aerobic Training
- Keywords
- Spinal Muscular Atrophy Type 3, Aerobic Training, SMN protein level, Physiotherapy
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Istanbul Medipol University HospitalUniversity, hospital or other
- Collaborators
- Istanbul University
- Study start
- 28 Jul 2022 (actual)
- Primary completion
- 30 Jun 2024 (estimated)
- Study completion
- 30 Sep 2024 (estimated)
- First posted
- 19 Sep 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.
In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments.One of these new treatments, Nusinersen, targets SMN2, which is a slightly different copy of SMN1, and increases SMN protein levels.
Preclinical studies have provided evidence that neuroprotection is strongly formed, with exercise significantly increasing motor neuron survival independent of SMN expression.
In a limited number of clinical studies prior to Nusinersen treatment, it was reported that aerobic exercise training improved maximum oxygen uptake (VO2 max) without causing muscle damage, but still caused fatigue.
The aim of this study is to determine the effect of aerobic exercise training on motor and respiratory functions, exercise capacity, fatigue and quality of life in SMA Type III patients who can walk and receive Nusinersen therapy. Twenty cases aged 10-50 years with genetically confirmed SMA diagnosis will be included in this study. The cases to be included in the study will be randomized into 2 groups as the training and control groups.
In addition to the routine physiotherapy program, medium-intensity Aerobic Exercise Training will be given to the study group for 12 weeks. Before and 12 weeks after the training, the cases will be evaluated with the Six Minute Walking Test, Submaximal Exercise Test, SMN protein level, function and strength assessments, (FVC) value, fatigue and quality of life scales.
In clinical trials, the supporting evidence for aerobic interventions in SMA is limited.
Additional studies on aerobic intervention parameters (frequency, intensity and duration) are needed.The results of this study will determine the feasibility of aerobic exercise training and provide important guidance for the clinical management of SMA patients.
Detailed description
Spinal Muscular Atrophy (SMA) is a neuromuscular disorder characterized by degeneration of alpha motor neurons in the spinal cord, leading to progressive muscle atrophy and weakness. Mutation or deletion of the survival motor neuron1 (SMN1) gene causes insufficient production of the SMN protein, which ensures motor neuron survival and maintenance in the spinal cord.
In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments. One of these new treatments, Nusinersen targets SMN2, which is a slightly different copy of SMN1, increases SMN protein levels. Preclinical studies have provided evidence that exercise significantly increases motor neuron survival independent of SMN expression, and that neuroprotection is strongly formed. In a limited number of clinical studies prior to Nuinersen therapy, it was reported that aerobic exercise training improved maximum oxygen uptake (VO2 max) without causing muscle damage, but still caused fatigue and had no significant beneficial effect on physical functioning. This study aims to determine the effect of aerobic exercise training on motor and respiratory functions, exercise capacity, fatigue and quality of life in SMA Type III patients who can walk and receive Nusinersen treatment. Twenty patients between the ages of 10 and 50 with genetically confirmed SMA diagnosis will be included in this study. The patients will be randomly divided into 2 groups as the training and control groups. In addition to the routine physiotherapy program, the study group will be given a moderate-intensity Aerobic Exercise Training for 12 weeks. Before and after 12 weeks of training, subjects will be evaluated with the Six-Minute Walk Test, ,Submaximal Exercise Test, SMN protein level and function and strength assessments, (FVC) value, fatigue and quality of life scales.
In clinical trials, the supporting evidence for aerobic interventions in SMA is limited. It has been reported that SMA patients cannot tolerate excessive exercise intensity and that recovery may be prevented due to excessive fatigue. Additional studies on aerobic intervention parameters (frequency, intensity and duration) are needed. The results of this study will determine the feasibility of aerobic exercise training and will have an impact on clinical practice by providing important guidance to the clinical management of SMA patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Aerobic Exercise Training Group | Experimental | Other: Aerobic Exercise Training, Other: Home exercise programAerobic training will be performed 3 days a week for 12 weeks at 60% -%75 of their maximum hearth rate with 30 minutes total duration consisting of 5 min warm up and 5 min cool down period in treatment group. Home exercise program will be given. This home program will include stretching, breathing, normal joint movement for 3 to 5 days a week |
| Control Group | Active comparator | Other: Home exercise programHome exercise program will be given. This home program will include stretching, breathing, normal joint movement, for 3 or 5 days a week. |
Who may qualify
- Ages
- 10 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Have been diagnosed of SMA Type 3 confirmed by genetic analysis,
- Between the ages of 10-50,
- Being able to walk 25 m without assistive device
Key exclusion criteria (4 listed)
- Using research drugs for SMA treatment other than Nusinersen treatment,
- Having a serious systemic disease that may prevent exercise,
- Have had a lower extremity injury and/or surgery in the last 6 months,
- To be applying a regular aerobic training program in the last 6 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 |
|---|---|---|
| Aerobic Exercise TrainingAerobic training will be performed 3 days a week for 12 weeks at 60%-%75 of their maximum hearth rate with 30 minutes total duration consisting of 5 min warm up and 5 min cool down period in treatment group. | Other | Aerobic Exercise Training Group |
| Home exercise programHome exercise program will be given. This home program will include stretching, breathing, normal joint movement, for 3 to 5 days a week. | Other | Aerobic Exercise Training Group, Control Group |
Primary outcome
Functional Capacity
Time frame: Baseline and 12 weeks
Change from baseline functional capacity test at 12 weeks. Functional capacity will be assessed by the 6 minute walking test.The 6MWT, an objective evaluation of functional capacity, measures the maximum distance a person can walk in six minutes over a 25-meter linear course.
10 secondary outcomes
Submaximal Exercise Test
Time frame: Baseline and 12 weeks
Pulmonary Function
Time frame: Baseline and 12 weeks
Motor Function
Time frame: Baseline and 12 weeks
Motor Performance
Time frame: Baseline and 12 weeks
Balance and Mobility
Time frame: Baseline and 12 weeks
Muscle strength
Time frame: Baseline and 12 weeks
Quality of Life in patients with neuromuscular disease
Time frame: Baseline and at 12 weeks
Fatigue
Time frame: Baseline and 12 weeks
Functional Lower Extremity Strength,
Time frame: Baseline and 12 weeks
Evaluation of SMN protein levels in blood biochemistry
Time frame: Baseline and 12 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2023.
- 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.
Sezan Mergen KILIÇ, MSc PT principal investigator
Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences
Fatma Karantay Mutluay,, Professor study director
Medipol University, Health Sciences Faculty, Physiotherapy and Rehabilitation
Fatma Yeşim Parman, Professor study director
Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Aerobic Exercise Training and Home exercise program in people with Neuromuscular Diseases and Spinal Muscular Atrophy Type 3, run by Istanbul Medipol University Hospital 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 3 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: status not confirmed, as of the sponsor's last update on 28 Sep 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sezan Mergen KILIÇ, MSc PT (05324569628, sezanmer@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Functional Capacity (time frame: Baseline and 12 weeks). 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 28 Sep 2023.