search
Back to results

Rehabilitation in SOD1 ALS Treated With Tofersen

Primary Purpose

Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis

Status
Enrolling by invitation
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Rehabilitation
Sponsored by
Washington University School of Medicine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Washington University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Rehabilitation Arm

Arm Description

Participants will participate in outpatient physical and/or occupational therapies while participating in the tofersen early access program (EAP)

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

1. Change from baseline in Handheld Dynamometry (HHD)
Quantitative muscle strength will be evaluated using HHD of multiple muscles using standard participant positioning.
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.
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)
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.
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
Change from baseline in Slow Vital Capacity (SVC)
Change from baseline in Maximal Inspiratory Pressure (MIP)
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

Full Information

First Posted
February 1, 2023
Last Updated
February 10, 2023
Sponsor
Washington University School of Medicine
search

1. Study Identification

Unique Protocol Identification Number
NCT05725759
Brief Title
Rehabilitation in SOD1 ALS Treated With Tofersen
Official Title
The Effects of Rehabilitation on Functional Outcomes in Patients With SOD1 ALS Treated With Tofersen
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
November 8, 2022 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Washington University School of Medicine

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Motor Neuron Disease, Familial

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Rehabilitation Arm
Arm Type
Experimental
Arm Description
Participants will participate in outpatient physical and/or occupational therapies while participating in the tofersen early access program (EAP)
Intervention Type
Behavioral
Intervention Name(s)
Rehabilitation
Other Intervention Name(s)
physical therapy, occupational therapy
Intervention Description
Particapants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy.
Primary Outcome Measure Information:
Title
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
Description
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
Secondary Outcome Measure Information:
Title
1. Change from baseline in Handheld Dynamometry (HHD)
Description
Quantitative muscle strength will be evaluated using HHD of multiple muscles using standard participant positioning.
Time Frame
12 months
Title
2. Change from baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Functional Rating (ROADS)
Description
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
Title
3. Change from baseline in ALS Assessment Questionnaire, 5-item (ALSAQ-5)
Description
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
Title
Change from baseline in motor Functional Independence Measure (motor FIM)
Description
Measures level of disability and indicates level of assistance required to perform activities of daily living.
Time Frame
12 months
Title
Change from baseline in Modified Ashworth Scale (MAS)
Description
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
Title
Change from baseline in Slow Vital Capacity (SVC)
Time Frame
12 months
Title
Change from baseline in Maximal Inspiratory Pressure (MIP)
Time Frame
12 months
Title
Change from baseline in Fatigue Severity Scale (FSS)
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States

12. IPD Sharing Statement

Links:
URL
https://alscenter.wustl.edu
Description
Washington University ALS Center website

Learn more about this trial

Rehabilitation in SOD1 ALS Treated With Tofersen

We'll reach out to this number within 24 hrs