The Effects of Rehabilitation on Functional Outcomes in Patients With SOD1 ALS Treated With Tofersen
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
研究概览
简要总结
The primary objective of this study is to document and describe the effects of a personalized rehabilitation program for patients with SOD1 ALS participating in the tofersen expanded access program. Participants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy. Participants will have an initial assessment performed and an individualized rehabilitation program will be prescribed. Each participant is encouraged to follow the prescribed recommendations that will include scheduled outpatient therapy sessions, functional assessments, and/or a home-based rehabilitation program. Functional assessments will be done at a minimum of every three months.
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of ALS with an SOD1 mutation
- •Current enrollment in the tofersen expanded access program
- •Age greater than 18 years
- •Medically able to participate in outpatient physical therapy and/or home-based rehabilitation, as determined by the treating health care provider
排除标准
- •Any comorbidities or conditions that, in the opinion of the treating healthcare provider, would unacceptably increase the risk of participation in outpatient physical therapy and/or home-based rehabilitation
结局指标
主要结局
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
时间窗: 12 months
ALSFRS-R measures functional disease severity in four functional domains, bulbar function, gross motor skills, fine motor skills, and respiratory. The assessment contains 12 questions scored from 0 (no function) to 4 (full function), with a total possible score of 48, which will indicate the highest level of function.
次要结局
- 1. Change from baseline in Handheld Dynamometry (HHD)(12 months)
- Change from baseline in Maximal Inspiratory Pressure (MIP)(12 months)
- Change from baseline in motor Functional Independence Measure (motor FIM)(12 months)
- Change from baseline in Fatigue Severity Scale (FSS)(12 months)
- 2. Change from baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Functional Rating (ROADS)(12 months)
- Change from baseline in Modified Ashworth Scale (MAS)(12 months)
- 3. Change from baseline in ALS Assessment Questionnaire, 5-item (ALSAQ-5)(12 months)
- Change from baseline in Slow Vital Capacity (SVC)(12 months)
研究者
Sean E Smith
Instructor in Neurology
Washington University School of Medicine