Clinical trial NCT05638750: Outpatient Rehabilitation Intervention for Young Children With SMA
Official title: Twice Weekly Outpatient Rehabilitation Intervention for Young Children With Spinal Muscular Atrophy Treated With Genetic Based Therapies: A Feasibility Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Spinal Muscular Atrophy
Keywords: young children
pediatric
rehabilitation
feasibility study
pilot study
Lead sponsor: Holland Bloorview Kids Rehabilitation Hospital
Sponsor class: OTHER
First posted: 2022-12-06
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).

Detailed 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).

Eligibility 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).

Primary outcomes:
measure: Infant Toddler Quality of Life Questionnaire (ITQOL)
timeFrame: Week 0, Week 12, Week 24
measure: PedsQL
timeFrame: Week 0, Week 12, Week 24
measure: Canadian Occupational Performance Measure (COPM)
timeFrame: Week 0, Week 12, Week 24
measure: Goal Attainment Scaling (GAS)
timeFrame: Week 0, Week 12, Week 24
measure: Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND)
timeFrame: Week 0, Week 12, Week 24
measure: Hammersmith Functional Motor Scale - Expanded (HFMSE)
timeFrame: Week 0, Week 12, Week 24
measure: Bayley Scales of Infant and Toddler DevelopmentTM, 4th Edition
timeFrame: Week 0, Week 12, Week 24
measure: Peabody Developmental Motor Scales (PDMS), 2nd Edition
timeFrame: Week 0, Week 12, Week 24
measure: Acceptability of Intervention Measure (AIM), Intervention Appropriateness Measure (IAM), Feasibility of Intervention Measure (FIM)
timeFrame: Week 12
measure: Home Exercise Diary
timeFrame: Weeks 1-24
Secondary outcomes:
Other outcomes:
measure: Demographics Survey
timeFrame: Week 0
Locations:
city: Toronto
country: Canada
facility: Holland Bloorview Kids Rehabilitation Hospital
state: Ontario
Study officials:
References: