Outpatient rehabilitation for young children with spinal muscular atrophy
Twice Weekly Outpatient Rehabilitation Intervention for Young Children With Spinal Muscular Atrophy Treated With Genetic Based Therapies: A Feasibility Study
This study is testing a 12-week outpatient rehab program for young children with spinal muscular atrophy to see if it helps improve their physical abilities while they receive genetic therapies.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 6 Months to 6 Years |
| Sex | All |
| Sponsor | Holland Bloorview Kids Rehabilitation Hospital Academic / other |
| Locations | 1 site (Toronto, Ontario) |
| Trial ID | NCT05638750 on ClinicalTrials.gov |
What this trial studies
This study evaluates an outpatient rehabilitation program designed for children aged 6 months to less than 6 years with Spinal Muscular Atrophy (SMA) who are receiving genetic therapies. Participants will engage in a 12-week program consisting of 45 minutes each of occupational and physical therapy weekly, along with prescribed home exercises. Following the therapy, there will be a 12-week period of only home exercises, with assessments conducted at the start and end of the program, as well as after the no therapy phase. The goal is to assess the feasibility and impact of this rehabilitation approach on physical function.
Who should consider this trial
Good fit: Ideal candidates are children aged 6 months to less than 6 years diagnosed with SMA who are receiving genetic-based therapies.
Not a fit: Patients who live outside of Ontario or require tracheostomy or daytime ventilation may not benefit from this study.
Why it matters
Potential benefit: If successful, this program could enhance physical function and quality of life for young children with SMA receiving genetic therapies.
How similar studies have performed: While there is limited evidence on rehabilitation for SMA post-genetic therapy, similar approaches in other conditions have shown promise, making this a potentially novel intervention.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * SMA (Type I, II or III) diagnosis; * Receives genetic based therapy; * Aged 6 months to less than 6 years old; * Able to participate in weekly therapy at Holland Bloorview (i.e., can attend in-person sessions); * Able to bring appropriate respiratory equipment to weekly therapy sessions, if required; * Substitute decision makers (SDMs) must be able to speak and read English; * Child participant must be able to understand/follow directions in English, as age appropriate; * SDMs must consent to participate on behalf of their child. Exclusion Criteria: * Live outside of Ontario; * Tracheostomy or use of daytime ventilation (excluding ventilation used during naps).
Where this trial is running
Toronto, Ontario
- Holland Bloorview Kids Rehabilitation Hospital — Toronto, Ontario, Canada (Recruiting)
Study contacts
- Study coordinator: Laura McAdam, MD
- Email: neuromuscular.research@hollandbloorview.ca
- Phone: 416-425-6220
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.