Exergaming for Spinal Muscular Atrophy

(INFORM SMA Trial)

Recruiting at 4 trial locations

Overseen By Harika Dasari

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre

Must be taking: Disease-modifying therapy

Disqualifiers: Severe scoliosis, Contractures, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

22 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether an 8-week home-based video game, Tales from the Magic Keep™, can improve life satisfaction for kids and teens with spinal muscular atrophy (SMA). SMA affects muscle strength, and the trial aims to determine if playing this game at home surpasses regular care. Ideal candidates are kids aged 8 to 18 with a confirmed SMA diagnosis who can sit without support for 10 seconds. Participants should also be receiving treatment that modifies the disease. As an unphased trial, this study provides a unique opportunity for participants to contribute to innovative research that could enhance the quality of life for those with SMA.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, it mentions that participants should be treated with disease-modifying therapy, so you may need to continue with that treatment.

What prior data suggests that this exergaming intervention is safe for children and youth with spinal muscular atrophy?

Research shows that "Tales from the Magic Keep," a home-based exercise game, is easy and enjoyable for children and teens with neuromuscular disorders to use at home. Studies have not found any serious side effects, indicating the game's safety for young users. This trial is labeled "not applicable" in phase, meaning it doesn't follow the usual stages of drug testing. However, existing research suggests it is safe, as it focuses on user enjoyment and ease of use rather than potential risks. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the exergaming intervention for spinal muscular atrophy (SMA) because it offers a fun and engaging way for patients to exercise, which can be especially beneficial for those with mobility challenges. Unlike traditional physical therapy, which can be repetitive and less engaging, "Tales from the Magic Keep™" uses interactive gameplay to motivate patients to stay active at home. This method not only encourages consistent exercise but also has the potential to improve motor function and quality of life for individuals with SMA. The blend of gaming and therapy is a novel approach that could revolutionize how we support physical health in SMA patients.

What evidence suggests that this exergaming intervention is effective for spinal muscular atrophy?

Research has shown that exergaming, such as the game Tales from the Magic Keep, offers a promising method for assisting children and teens with Spinal Muscular Atrophy (SMA). In this trial, participants in the exergaming intervention arm will play Tales from the Magic Keep at home for 8 weeks. The game is designed for ease of use and, while limited information exists on its impact on job satisfaction, it specifically targets individuals with SMA and has been well-received by users. The goal is to encourage movement, which is crucial for managing SMA. Early research suggests that participants are likely to continue using it regularly. ^{1 3 4 6 7}

Who Is on the Research Team?

Maryam Oskoui

Principal Investigator

McGill University

Danielle Levac

Principal Investigator

Université de Montréal

Are You a Good Fit for This Trial?

This trial is for children and adolescents aged 8-18 with spinal muscular atrophy (SMA) who can raise their hands to their mouth but not a light weight, are on disease-modifying therapy, have confirmed genetic SMA diagnosis, and can sit unaided for at least 10 seconds.

Inclusion Criteria

I have a confirmed genetic diagnosis of 5q SMA.

I can sit by myself without support for at least 10 seconds.

I can move my hands to my mouth but can't lift a light weight to it.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive an 8-week home-based exergaming intervention, playing Tales from the Magic Keep™ on the Azure Kinect platform, 3 times per week for at least 20 minutes

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments including the Canadian Occupational Performance Measure (COPM)

8 weeks

Open-label extension

Participants may opt into an 8-week open-label extension phase, continuing the exergaming intervention

8 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tales from the Magic Keep™

Trial Overview The study compares an 8-week home-based exergaming program called Tales from the Magic Keep™ against usual care. It aims to see if this fun video game-like exercise can improve how satisfied kids with SMA feel about their daily activities.

How Is the Trial Designed?

Group I: Exergaming Intervention for 8 Weeks Experimental Treatment 1 Intervention

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

Group II: Usual Care for 8 weeks. Active Control 1 Intervention

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

Who Is Running the Clinical Trial?

McGill University Health Centre/Research Institute of the McGill University Health Centre

Lead Sponsor

Trials

476

Recruited

170,000+

Maryam Oskoui, MD, MSc, FRCPC, FAAN

Lead Sponsor

Trials

1

Recruited

20+

Published Research Related to This Trial

A study of 56 untreated young individuals with spinal muscular atrophy (SMA) showed that their performance on the 10-meter walk/run test (10MWRT) improved from ages 3 to 8, remained stable from ages 9 to 10, and then gradually declined from ages 11 to 18.

Factors such as having SMA type 3b, a higher SMN2 copy number (4 vs. 3), and lower body mass were linked to faster 10MWRT times, providing insights into how these variables may influence walking speed in ambulant SMA patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Natural history of 10-meter walk/run test performance in spinal muscular atrophy: A longitudinal analysis. Krosschell, KJ., Townsend, EL., Kiefer, M., et al.[2023]

A 19-month study involving 14 ambulatory SMA patients showed that daily exercise is safe and does not negatively impact strength, function, or fatigue, suggesting it should be encouraged for these individuals.

While the exercise did not significantly improve walking distance or other functional measures, there was a notable 4.9% improvement in percent-predicted VO2 max after 6 months, indicating some benefits in exercise capacity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Single-Blind, Randomized, Controlled Clinical Trial of Exercise in Ambulatory Spinal Muscular Atrophy: Why are the Results Negative? Montes, J., Garber, CE., Kramer, SS., et al.[2023]

A 12-week home-based progressive resistance training program for children with spinal muscular atrophy (SMA) was found to be feasible and safe, with 90% of sessions completed without any study-related adverse events.

Trends indicated improvements in muscle strength and motor function, suggesting that exercise may be beneficial for children with SMA, paving the way for future research in this area.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Resistance strength training exercise in children with spinal muscular atrophy. Lewelt, A., Krosschell, KJ., Stoddard, GJ., et al.[2021]

A 6-year-old boy with Spinal Muscular Atrophy Type II showed good activity tolerance while using a pediatric gait exoskeleton at home for two months, indicating potential benefits for mobility in children with this condition.

The child's performance improved over time, with decreased fatigue perception and stable vital signs, suggesting that exoskeleton technology may enhance the quality of life for children with neuromuscular diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Using a robotic exoskeleton at home: An activity tolerance case study of a child with spinal muscular atrophy. Garces, E., Puyuelo, G., Sánchez-Iglesias, I., et al.[2022]

The 6-minute walk test (6MWT) is a reliable measure of walking ability in patients with spinal muscular atrophy (SMA), and this study highlights the need for normative data to compare SMA patients with healthy individuals.

Analysis of data from three pediatric and six adult studies revealed significant differences in % predicted values for the 6MWT, indicating variability in walking ability assessments between children and adults with SMA.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Limitations of 6-minute walk test reference values for spinal muscular atrophy. Goodwin, AM., Cornett, KMD., McKay, MJ., et al.[2020]

Disease-modifying therapies for spinal muscular atrophy (SMA) are significantly improving clinical outcomes, and physical therapists are essential in conducting clinical trials for these therapies, highlighting the need for their education on best practices.

The SMA Clinical Trial Readiness Program developed a Best Practices Clinical Evaluator Toolkit based on feedback from physical therapists, which aims to bridge knowledge gaps and enhance the effectiveness of rehabilitation practices for individuals with SMA.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical and Research Readiness for Spinal Muscular Atrophy: The Time Is Now for Knowledge Translation. Krosschell, KJ., Dunaway Young, S., Peterson, I., et al.[2023]

A single randomized controlled trial involving 14 participants with spinal muscular atrophy (SMA) type 3 showed that a six-month combined strength and aerobic exercise program did not lead to significant improvements in walking distance, cardiopulmonary capacity, or functional motor scores compared to usual care, indicating uncertain efficacy of exercise training in this population.

The study reported no serious adverse events related to the exercise program, but the overall quality of evidence was very low due to study limitations, highlighting the need for more rigorous research to determine the effects of exercise on SMA type 3.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Physical exercise training for type 3 spinal muscular atrophy. Bartels, B., Montes, J., van der Pol, WL., et al.[2023]

The Endurance Shuttle Tests (ESNHPT, ESBBT, and ESWT) were validated as reliable measures of fatigability in 61 patients with Spinal Muscular Atrophy (SMA), showing that a significant percentage of patients experienced increased fatigability compared to healthy controls.

These tests demonstrated good test-retest reliability (ICC's from .78 to .91) and are effective for documenting physical impairments in SMA, making them promising tools for clinical trials focused on treatment efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fatigability in spinal muscular atrophy: validity and reliability of endurance shuttle tests. Bartels, B., de Groot, JF., Habets, LE., et al.[2021]

A new game using the Microsoft Kinect sensor was developed to objectively measure upper limb movement in patients with spinal muscular atrophy (SMA), showing promise as a tool for assessing disease progression and treatment efficacy.

In a study of 18 ambulant SMA type III patients and 19 healthy controls, no significant differences in active range of motion were found, although differences in hand velocity were noted, indicating the need for further validation of this measurement approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Feasibility of Using Microsoft Kinect to Assess Upper Limb Movement in Type III Spinal Muscular Atrophy Patients. Chen, X., Siebourg-Polster, J., Wolf, D., et al.[2022]

A 12-week home-based functional exercise program for individuals with spinal and bulbar muscular atrophy (SBMA) was well tolerated and did not result in significant improvements in overall muscle function or quality of life compared to a stretching control group of 50 participants.

However, a subgroup analysis revealed that low-functioning individuals in the functional exercise group showed improvements in specific functional scores, suggesting that targeted exercise may benefit certain patients and should be explored further.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized controlled trial of exercise in spinal and bulbar muscular atrophy. Shrader, JA., Kats, I., Kokkinis, A., et al.[2018]

Citations

1. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1002/mus.70032

Feasibility of a Home‐Based Exergaming Intervention for ... The primary aim of this study was to assess adherence, acceptability, and usability of Tales from the Magic Keep in children and adolescents ...

2. researchgate.net researchgate.net/publication/395808233_Feasibility_of_a_Home-Based_Exergaming_Intervention_for_Youth_With_Spinal_Muscular_Atrophy

Feasibility of a Home-Based Exergaming Intervention for ... The primary objective of this study was to assess the feasibility of a fit‐for‐purpose home‐based exergaming intervention in children and youth with SMA and ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06396325

NCT06396325 | A Registry Based Randomized-Controlled ... 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 ...

4. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/pdf/10.1002/mus.70032

Feasibility of a Home‐Based Exergaming Intervention for ... The primary aim of this study was to assess adherence, accept- ability, and usability of Tales from the Magic Keep in children and adolescents ...

5. nature.com nature.com/articles/d41586-025-03507-5

Ancient graffiti and brain complexities: Books in brief Over the past few decades, brain research has produced effective treatments for pain, insomnia, postpartum depression, paralysis and spinal ...

6. neurology.org neurology.org/doi/10.1212/WNL.0000000000203145

Feasibility of a Home-Based Exergame Therapy for Youth ... We have developed a home-based exergame (Tales from the Magic Keep™) specifically for youth with neuromuscular disorders in which functional upper limb and ...

7. arxiv.org arxiv.org/html/2403.16107v1

Designing Upper-Body Gesture Interaction with and for ... Twelve people with Spinal Muscular Atrophy designed upper-body gestures for VR commands. Two of them participated in person, while the other ten ...