Personalized Exercise Training for People With Rare Neuromuscular Disorders: a Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study to Assess the Effects on Physical Function From Personalized Strength and Balance Exercise in a Rehabilitation Setting.
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 120
- 试验地点
- 9
- 主要终点
- Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)
研究概览
简要总结
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders: Charcot-Marie-Tooth (CMT), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy Type 1 (DM1).
The key objectives are:
- To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group
- To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up
- To investigate if improvements in dynamic balance are associated with improvements in physical activity, body composition, estimated motor units, metabolomics, muscle echnogenecity and volume, and other indicators of health and quality of life.
This is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions.
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
- Single (Outcomes Assessor)
盲法说明
The evaluators will be blinded to the treatment group of the participants. It is not possible to blind the participants themselves; however, they will be asked not to share their group allocation with the others, including the evaluators. A staff member may unblind the intervention assignment for any participant with a serious adverse event.
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A confirmed diagnosis of either FSHD, DM1 or CMT
- •18-70 years of age at the time of signing the informed consent.
- •Any gender
- •Ability to stand, rise from a chair and walk at least 10 meters with or without any need of assistive devices
- •Indication for rehabilitation as confirmed by the treating neurologist or physiotherapist
- •Ability to understand and follow instructions in Norwegian
- •Capable of giving signed informed consent
排除标准
- •Pregnancy or planning to become pregnant
- •Any other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart-failure/cardiomyopathy, on-going cancer treatment
- •Alcohol or drug abuse as per their medical chart
- •History of non-compliance to medical advice/follow-up
研究组 & 干预措施
Intervention
The experimental group receives four-months of exercise-based intervention
干预措施: Exercise (Other)
Control
The control group follows usual care
干预措施: Usual Care (Other)
结局指标
主要结局
Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)
时间窗: Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)
The primary outcome measure will be change in dynamic balance from pre-intervention to post-intervention. In addition, there will be two follow-up assessments.
次要结局
- Fatigue severity scale(Baseline, 4-month, 10-month and 16-month (only intervention-group))
- Global Metabolomics(Baseline, 4-month, 10-month)
- Dual Energy X-ray Absorptiometry(4-month, 10-month and 16-month)
- Physical activity(4-month, 10-month and 16-month)
- ABC-scale(Basleine, 4 month, 10 month, 16 month (only intervention group))
- PROMIS-29(Baseline, 4 month, 10 month and 16 month (only intervention group))
- Clinical measures of strength and function(Baseline 4-month, 10-month and 16-month (only intervention group))
- Neuromuscular ultrasound(4-month, 10-month and 16-month)
- Motor unit number estimation(Baseline 4-month, 10-month and 16-month (only intervention-group))
研究者
Kristin Ørstavik
head of Section, MD, PhD
Oslo University Hospital