Rehabilitation in SOD1 ALS Treated With Tofersen

NCT05725759 | Unknown

• 1 other identifier: 202208170
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• 1. Change from baseline in Handheld Dynamometry (HHD)

  12 months

• 2. Change from baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Functional Rating (ROADS)

  12 months

• 3. Change from baseline in ALS Assessment Questionnaire, 5-item (ALSAQ-5)

  12 months

• Change from baseline in motor Functional Independence Measure (motor FIM)

  12 months

• Change from baseline in Modified Ashworth Scale (MAS)

  12 months

Study Arms (1)

Rehabilitation Arm

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

Behavioral: Rehabilitation

Interventions

Rehabilitation BEHAVIORAL

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

Also known as: physical therapy, occupational therapy

Rehabilitation Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with SOD1 ALS

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Washington University School of Medicine: lead

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Related Links

• Washington University ALS Center website

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Interventions

Rehabilitation; Physical Therapy Modalities; Occupational Therapy

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Aftercare; Continuity of Patient Care; Patient Care; Therapeutics; Health Services; Health Care Facilities Workforce and Services

Study Design

• Study Type: observational
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Instructor in Neurology

Study Record Dates

• First Submitted: February 1, 2023
• First Posted: February 13, 2023
• Study Start: November 8, 2022
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: January 30, 2024

Record last verified: 2024-01

Locations

US (1)