What side effects are associated with this type of intervention?

Physical and occupational therapy for Spinal Muscular Atrophy (SMA) are generally well-tolerated and aim to improve motor function and physical health. Common side effects may include muscle soreness, fatigue, and, in rare cases, overuse injuries. These therapies are designed to be low-risk and are tailored to the individual's capabilities to minimize adverse effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Spinal Muscular Atrophy (SMA), including nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and risdiplam (Evrysdi). These treatments primarily focus on genetic and molecular approaches to improve motor function and physical health in SMA patients. Nusinersen is an antisense oligonucleotide that modifies SMN2 gene splicing, onasemnogene abeparvovec is a gene therapy that delivers a functional copy of the SMN1 gene, and risdiplam is an oral medication that increases SMN protein production. These treatments, combined with physical and occupational therapy, aim to enhance motor function and overall physical health in SMA patients.

What other types of research exist?

Promising novel alternatives to traditional outpatient rehabilitation programs for SMA patients include: 1) Genetic-based therapies such as gene replacement therapy (e.g., Zolgensma) and antisense oligonucleotides (e.g., Spinraza). 2) Non-invasive ventilatory support to improve respiratory function and overall quality of life. 3) Myoelectric powered wearable orthotics (UE-MPWO) for enhancing upper extremity function. 4) Complementary therapies like Qigong massage and mind-body therapies (e.g., yoga) which have shown benefits in motor function and physical health.

← Back to Search

Rehabilitation for Spinal Muscular Atrophy

N/A

Recruiting

Research Sponsored by Holland Bloorview Kids Rehabilitation Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

SMA (Type I, II or III) diagnosis

Receives genetic based therapy

Must not have

Tracheostomy or use of daytime ventilation (excluding ventilation used during naps)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 0, week 12, week 24

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a rehabilitation program for young children with Spinal Muscular Atrophy (SMA) who have received genetic treatments. The program includes regular physical and occupational therapy sessions and home exercises. The goal is to see if this helps improve their physical abilities. A supervised, home-based progressive resistance training exercise program is feasible, safe, and well tolerated in children with SMA.

Who is the study for?

This trial is for young children aged 6 months to 3 years with Spinal Muscular Atrophy (SMA) who are treated with genetic therapies. They must be able to attend weekly therapy in Ontario, handle instructions in English, and have a guardian's consent. Children requiring tracheostomy or daytime ventilation (except during naps) cannot participate.

What is being tested?

The study tests a rehabilitation program combining occupational and physical therapy for SMA-affected children receiving genetic treatments. It involves a structured 12-week therapy followed by another 12 weeks where only home exercises are prescribed.

What are the potential side effects?

Since the intervention is non-drug based, focusing on physical and occupational therapy, traditional medication side effects aren't expected. However, there may be fatigue or muscle soreness associated with increased physical activity.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with Spinal Muscular Atrophy (SMA) Type I, II, or III.

Select...

I am receiving treatment that targets my cancer's genetic changes.

Select...

I am between 6 months and 3 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I use a tracheostomy or need ventilation during the day.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 0, week 12, week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 0, week 12, week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 0, week 12, week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Acceptability of Intervention Measure (AIM), Intervention Appropriateness Measure (IAM), Feasibility of Intervention Measure (FIM)

Bayley Scales of Infant and Toddler DevelopmentTM, 4th Edition

Canadian Occupational Performance Measure (COPM)

+7 more

Other study objectives

Demographics Survey

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: InterventionExperimental Treatment1 Intervention

Participants will participate in the rehabilitation intervention.

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Spinal Muscular Atrophy (SMA) include genetic-based therapies such as nusinersen, onasemnogene abeparvovec, and risdiplam. Nusinersen works by modifying the splicing of SMN2 gene transcripts to increase the production of functional SMN protein. Onasemnogene abeparvovec delivers a functional copy of the SMN1 gene via a viral vector, directly addressing the genetic cause of SMA. Risdiplam also promotes the production of functional SMN protein by modifying SMN2 gene splicing. These treatments are crucial as they target the underlying genetic defect, leading to improved motor function and physical health. Complementary to these genetic therapies, physical and occupational therapy, as studied in the outpatient rehabilitation program, are essential for maximizing motor function, preventing contractures, and enhancing overall quality of life for SMA patients.

Management of motor rehabilitation in individuals with muscular dystrophies. 1st Consensus Conference report from UILDM - Italian Muscular Dystrophy Association (Rome, January 25-26, 2019).Establishing a telerehabilitation program for patients with Duchenne muscular dystrophy in the COVID-19 pandemic.The Role of Rehabilitation in the Management of Patients with Charcot-Marie-Tooth Disease: Report of Two Cases.