Rehabilitation for Spinal Muscular Atrophy

Recruiting in Palo Alto (17 mi)

Age: < 18

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: Holland Bloorview Kids Rehabilitation Hospital

No Placebo Group

Approved in 1 jurisdiction

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

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.

Inclusion Criteria

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

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

I am between 6 months and 3 years old.

Exclusion Criteria

I use a tracheostomy or need ventilation during the day.

Participant Groups

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.

1Treatment groups

Experimental Treatment

Group I: InterventionExperimental Treatment1 Intervention

Participants will participate in the rehabilitation intervention.

Rehabilitation Intervention is already approved in United States for the following indications:

🇺🇸 Approved in United States as Physical Rehabilitation Intervention for: