Clinical trial NCT06396325: A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy
Official title: A Registry-Based Parallel Group Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy, with an Optional Open-Label Extension
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Spinal Muscular Atrophy
Keywords: spinal muscular atrophy
rehabilitation
Exergaming
Pediatrics
Randomized-controlled trial
Lead sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre
Sponsor class: OTHER
First posted: 2024-05-02
Enrollment: 22
Sex: ALL
Minimum age (years): 8
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: This is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational satisfaction of children and youth (8-18 years old age) with spinal muscular atrophy (SMA).

Detailed description: This multisite randomized controlled registry-based trial will evaluate the efficacy of a home-based exergaming intervention, as compared to usual care, on occupational satisfaction of children and youth (aged 8-18 years old) with SMA. Participants in the intervention arm will play the exergame, Tales from the Magic Keep™ on the Azure Kinect platform, 3 times per week, for at least 20 minutes, over 8 weeks (total 8 hours), and continue to access their regular physical activities and rehabilitation services. Those in the usual care arm will continue to access their regular physical activities and rehabilitation services. Occupational satisfaction will be assessed using the Canadian Occupational Performance Measure (COPM) at 8 weeks.

Eligibility criteria: Inclusion Criteria:

* Confirmed genetic diagnosis of 5q SMA
* Age at screening: Aged 8-18 years old
* Participant is able to stay seated independently without support for at least 10 seconds
* Participants has a score of at least 2 points in entry item A of the RULM (i.e., "Can raise 1 or 2 hands to the mouth but cannot raise a 200 g weight in it to the mouth")
* Treated with disease-modifying therapy
* Signed ICF

Exclusion Criteria:

* Inability to comply with study procedures according to the site investigator, prior to randomization
* Severe scoliosis or contractures that would interfere with gameplay or with successful completion of functional assessments, as confirmed by the clinical evaluator
* Planned orthopedic surgery 6 months prior to or throughout intervention and follow-up period (16 weeks post randomization)
* Inability to read in English or French

Primary outcomes:
measure: Satisfaction with occupational performance
timeFrame: Baseline to 16 weeks
Secondary outcomes:
measure: Self- reported fatigue
timeFrame: Baseline to 16 weeks
measure: Independence performing activities of daily living
timeFrame: Baseline to 16 weeks
measure: Upper limb motor activity
timeFrame: Baseline to 16 weeks
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Vancouver
country: Canada
facility: BC Children's Hospital
state: British Columbia
city: Halifax
country: Canada
facility: IWK Health Services
state: Nova Scotia
city: Toronto
country: Canada
facility: Hospital for SickKids
state: Ontario
city: Montreal
country: Canada
facility: Centre de readaptation Marie-Enfant
state: Quebec
Study officials:
affiliation: McGill University
name: Maryam Oskoui
role: PRINCIPAL_INVESTIGATOR
affiliation: Université de Montréal
name: Danielle Levac
role: PRINCIPAL_INVESTIGATOR
References: