Rehabilitation in SOD1 ALS Treated With Tofersen

Study 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.

Official Title
-----------------
The Effects of Rehabilitation on Functional Outcomes in Patients With SOD1 ALS Treated With Tofersen

Conditions
-----------------
Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Motor Neuron Disease, Familial

Intervention / Treatment
-----------------
* Behavioral: Rehabilitation


Participation Criteria
=================
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rehabilitation Arm<br>Participants will participate in outpatient physical and/or occupational therapies while participating in the tofersen early access program (EAP) | Behavioral: Rehabilitation<br>* Particapants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy.<br>* Other names: occupational therapy;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 1. Change from baseline in Handheld Dynamometry (HHD) | Quantitative muscle strength will be evaluated using HHD of multiple muscles using standard participant positioning. | 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. | 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) | 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. | 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 | 12 months | 
| Change from baseline in Slow Vital Capacity (SVC) |  | 12 months | 
| Change from baseline in Maximal Inspiratory Pressure (MIP) |  | 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 | 12 months |