Rehabilitation in SOD1 ALS Treated With Tofersen

The Washington University

Status: Invitation-only

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

Treatments: Behavioral: Rehabilitation

Study type: Observational

Funder types: Other

Identifiers

NCT05725759

202208170

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

10 participants in 1 patient group

Rehabilitation Arm

Description:

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

Treatment:

Behavioral: Rehabilitation