Outpatient Rehabilitation Intervention for Young Children With SMA

Holland Bloorview Kids Rehabilitation Hospital

Status

Enrolling

Conditions

Spinal Muscular Atrophy

Treatments

Other: Rehabilitation Intervention

Study type

Interventional

Funder types

Other

Identifiers

NCT05638750

0550

Details and patient eligibility

About

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).

Full 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).

Enrollment

10 estimated patients

Sex

All

Ages

6 months to 6 years old

Volunteers

No Healthy Volunteers

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