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RECRUITING
Updated: Jul 8, 2025

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

Phase
N/A
Early 1
1
2
3
4

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).

Key Information

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

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