Rehabilitation in SOD1 ALS Treated With Tofersen

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05725759
• Other study ID #: 202208170
• Status: Enrolling by invitation
• Start date: November 8, 2022
• Est. completion date: December 2024

Study information

• Verified date: January 2024
• Source: Washington University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 10
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Familial Amyotrophic Lateral Sclerosis
• Lou Gehrig Disease
• Motor Neuron Disease
• Motor Neuron Disease, Familial
• Sclerosis

Intervention

Behavioral:
• Rehabilitation
Particapants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy.

Locations

Country	Name	City	State
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Washington University School of Medicine

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Change from baseline in Slow Vital Capacity (SVC)		12 months
Secondary	Change from baseline in Maximal Inspiratory Pressure (MIP)		12 months
Secondary	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

External Links

•   Washington University ALS Center website