The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy"
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Functional Capacity
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 10 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Functional Capacity
时间窗: 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.
次要结局
- Pulmonary Function(Baseline and 12 weeks)
- Balance and Mobility(Baseline and 12 weeks)
- Quality of Life in patients with neuromuscular disease(Baseline and at 12 weeks)
- Fatigue(Baseline and 12 weeks)
- Evaluation of SMN protein levels in blood biochemistry(Baseline and 12 weeks)
- Submaximal Exercise Test(Baseline and 12 weeks)
- Motor Function(Baseline and 12 weeks)
- Motor Performance(Baseline and 12 weeks)
- Muscle strength(Baseline and 12 weeks)
- Functional Lower Extremity Strength,(Baseline and 12 weeks)
研究者
sezanmergen
Msc -Physiotherapist
Istanbul Medipol University Hospital