Outpatient Rehabilitation Intervention for Young Children With SMA

Last updated: April 5, 2024
Sponsor: Holland Bloorview Kids Rehabilitation Hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Spinal Muscular Atrophy

Muscular Dystrophy

Myasthenia Gravis (Chronic Weakness)

Treatment

Rehabilitation Intervention

Clinical Study ID

NCT05638750
0550
  • Ages 6-6
  • All Genders

Study Summary

An outpatient rehabilitation program for children (6 months to less than 6 years old) with Spinal Muscular Atrophy (SMA) treated with genetic based therapies is being studied. Participants will participate in a 12-week therapy program where they receive 45 minutes each of occupational therapy and physical therapy each week. Home exercises will also be prescribed to be completed 5 days per week. At the end of the therapy program, there will be a 12-week period of no therapy where only home exercises will be completed. Assessments and program evaluation will occur at the beginning (Week 0) and end of the rehabilitation program (Week 24), then at the end of the no therapy block (week 24).

Eligibility Criteria

Inclusion

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-personsessions);
  • Able to bring appropriate respiratory equipment to weekly therapy sessions, ifrequired;
  • Substitute decision makers (SDMs) must be able to speak and read English;
  • Child participant must be able to understand/follow directions in English, as ageappropriate;
  • SDMs must consent to participate on behalf of their child.

Exclusion

Exclusion Criteria:

  • Live outside of Ontario;
  • Tracheostomy or use of daytime ventilation (excluding ventilation used during naps).

Study Design

Total Participants: 10
Treatment Group(s): 1
Primary Treatment: Rehabilitation Intervention
Phase:
Study Start date:
January 01, 2022
Estimated Completion Date:
December 31, 2024

Study Description

The standard of care for SMA has historically been physical therapy (PT) and occupational therapy (OT) often focused on strategies that reduce the risk of secondary side effects such as joint tightness. Recently, three genetic based therapies: nusinersen, onasemnogene abeparvovec and risdiplam, have been approved as treatment by Health Canada. Genetic based therapies have provided improvements in physical function for children with SMA. Currently, there is no evidence-based guidance regarding rehabilitation to increase function for children with SMA that have received genetic based therapies. Additionally, there is a lack of published evidence regarding the type of rehabilitation programs and the impact that rehabilitation has on physical function.

An outpatient rehabilitation program for children (6 months to less than 6 years old) with Spinal Muscular Atrophy (SMA) treated with genetic based therapies is being proposed and its feasibility being evaluated. Participants will participate in a 12-week therapy program where they receive 45 minutes each of occupational therapy and physical therapy each week. Home exercises will also be prescribed to be completed 5 days per week. At the end of the therapy program, there will be a 12-week period of no therapy where only home exercises will be completed. Assessments and program evaluation will occur at the beginning (Week 0) and end of the rehabilitation program (Week 24), then at the end of the no therapy block (week 24).

Connect with a study center

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario M4G 1R8
    Canada

    Active - Recruiting

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