The Effects of Rehabilitation on Functional Outcomes in Patients With SOD1 ALS Treated With Tofersen
Overview
- Phase
- N/A
- Intervention
- Not specified
- Conditions
- Amyotrophic Lateral Sclerosis
- Sponsor
- Washington University School of Medicine
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
- Status
- Enrolling by Invitation
- Last Updated
- 2 years ago
Overview
Brief Summary
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.
Investigators
Sean E Smith
Instructor in Neurology
Washington University School of Medicine
Eligibility Criteria
Inclusion Criteria
- •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
Exclusion Criteria
- •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
Outcomes
Primary Outcomes
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
Time Frame: 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.
Secondary Outcomes
- 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)