A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy

NCT06396325 | Recruiting

• 1 other identifier: MP-37-2022-7289
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 5

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).

Conditions

Spinal Muscular Atrophy

Keywords

spinal muscular atrophy; rehabilitation; Exergaming; Pediatrics; Randomized-controlled trial

Outcome Measures

Primary Outcomes (1)

• Satisfaction with occupational performance

  Canadian Occupational Performance Measure (COPM) Satisfaction

  Baseline to 16 weeks

Secondary Outcomes (3)

• Self-reported fatigue

  Baseline to 16 weeks

• Independence performing activities of daily living

  Baseline to 16 weeks

• Upper limb motor activity

  Baseline to 16 weeks

Study Arms (2)

Exergaming Intervention for 8 Weeks

EXPERIMENTAL

Extension Phase: Participants randomized ot the intervention arm will play Tales from the Magic Keep at home for the 8 week open-label extension phase

Other: Tales from the Magic Keep™

Usual Care for 8 weeks.

ACTIVE COMPARATOR

Extension Phase: Participants randomized to waitlist usual care will play Tales from the Magic Keep at home for the 8 week open-label extension phase

Other: Usual Care

Interventions

Tales from the Magic Keep™ OTHER

Exergame targeting upper limb and trunk movement designed for use on the Microsoft Azure Kinect Dose: At least 3 times a week for 20 minutes over 8 weeks (8 hours total) Continue with regularly accessed physical activities and rehabilitation services

Exergaming Intervention for 8 Weeks

Usual Care OTHER

Continue with regularly accessed physical activities and rehabilitation services

Usual Care for 8 weeks.

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Maryam Oskoui, MD, MSc, FRCPC, FAAN: lead

Study Sites (5)

Alberta Children's Hospital

Calgary, Alberta, Canada

RECRUITING

BC Children's Hospital

Vancouver, British Columbia, Canada

RECRUITING

IWK Health Services

Halifax, Nova Scotia, Canada

RECRUITING

Hospital for SickKids

Toronto, Ontario, Canada

RECRUITING

Centre de readaptation Marie-Enfant

Montreal, Quebec, Canada

RECRUITING

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Maryam Oskoui

  McGill University

  PRINCIPAL INVESTIGATOR

• Danielle Levac

  Université de Montréal

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mbaye Ndiaye

CONTACT

5149341934; mbaye.ndiaye@muhc.mcgill.ca

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Full Professor, Department of Pediatrics and Neurology & Neurosurgery

Study Record Dates

• First Submitted: April 29, 2024
• First Posted: May 2, 2024
• Study Start: November 1, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: July 8, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (5)