An observational study in Amyotrophic Lateral Sclerosis, Lou Gehrig Disease and Familial Amyotrophic Lateral Sclerosis, sponsored by Washington University School of Medicine. Enrolling by invitation at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-01-30.
Sponsored by Washington University School of Medicine · Observational
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.
717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.
This study's planned enrollment of 10 is below the median of 109 across 217 observational studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →Washington University School of Medicine is the lead sponsor of 1,765 studies on the registry; 271 are open to participants now.
Of its 324 completed or terminated interventional studies of FDA-regulated products, 212 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients with SOD1 ALS
Exclusion Criteria:
Participants will participate in outpatient physical and/or occupational therapies while participating in the tofersen early access program (EAP)
Behavioral: Rehabilitation
Particapants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy.
Also known as: physical therapy, occupational therapy
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
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.
Time frame: 12 months
1. Change from baseline in Handheld Dynamometry (HHD)
Quantitative muscle strength will be evaluated using HHD of multiple muscles using standard participant positioning.
Time frame: 12 months
2. Change from baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Functional Rating (ROADS)
ROADS measures disability across multiple daily activities. For each question, response options are as follows: 0=unable to perform; 1=abnormal, able to perform but with difficulty; and 2=normal, able to perform without difficulty.
Time frame: 12 months
3. Change from baseline in ALS Assessment Questionnaire, 5-item (ALSAQ-5)
Quality of life will be measured using the 5-item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-5) patient-reported outcome (PRO)
Time frame: 12 months
Change from baseline in motor Functional Independence Measure (motor FIM)
Measures level of disability and indicates level of assistance required to perform activities of daily living.
Time frame: 12 months
Change from baseline in Modified Ashworth Scale (MAS)
The MAS is 6-point scale that that grades spasticity, scored from 0 to 4, where 0 is normal muscle tone and 4 is severe increase in muscle tone
Time frame: 12 months
Change from baseline in Slow Vital Capacity (SVC)
Time frame: 12 months
Change from baseline in Maximal Inspiratory Pressure (MIP)
Time frame: 12 months
Change from baseline in Fatigue Severity Scale (FSS)
FSS measures how fatigue interferes with activities. It is a 9-item questionnaire scored on a 7-point scale, 1 = strongly disagree to 7 = strongly agree, and the higher the score equal to the greater the severity of fatigue
Time frame: 12 months
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Washington University School of Medicine