Wheelchair Skills Training for Spastic Ataxia and Myotonic Dystrophy

Overseen By Krista L Best, PhD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Laval University

Disqualifiers: Surgery, Concurrent WC training, others

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

Compensation: Varies

Number of Visits: 1

Duration: 5 weeks

20 Participants Needed

Trial Summary

What is the purpose of this trial?

Wheelchairs (WC) are often provided to people with ARSACS and MD1 when they are not able to walk anymore. However, giving someone a MWC alone does not guarantee they will use it safely or properly. Many people who use WC need help from others to get around and they can not always do the things they like to do. This can lead to isolation, stress, and reduced quality of life. In addition, poor use of a MWC could lead to accidents and injuries. Our team recently showed that people with ARSACS have lower MWC skills than other adults who use MWC, and that teaching MWC skills to people with ARSACS seems to work. Now we are ready to test the program with more people with ARSACS and MD1 to see how it can improve MWC mobility and confidence. We also want to hear about people's expectations and experiences with MWC training. People who take part in research will answer questions before and after WC training, and we will follow up with them 3 months later to ask again about their WC use. This projects directly adresses the mobility needs of people with ARSACS and MD1 who use MWC. Our results may improve how therapists provide training for MWC use, which may improve mobility, participation, and quality of life for people with ARSACS and MD1. Learning just one MWC skill could be life-changing. It could mean the difference between leaving the house or not, which could impact the ability to shop for groceries, see friends, or to have a job.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

What data supports the effectiveness of the treatment Wheelchair Skills Training Program for Spastic Ataxia and Myotonic Dystrophy?

The research on myotonic dystrophy shows that rehabilitation programs can improve balance, gait, and muscle strength, which are important for wheelchair skills. This suggests that similar training programs might help people with spastic ataxia and myotonic dystrophy improve their wheelchair skills. ^{1 2 3 4 5}

Is wheelchair skills training generally safe for humans?

A systematic review found that wheelchair skills training programs are generally safe for improving wheelchair skills capacity. ^{6 7 8 9 10}

How does the Wheelchair Skills Training Program differ from other treatments for spastic ataxia and myotonic dystrophy?

The Wheelchair Skills Training Program is unique because it focuses on improving the ability and confidence of individuals to use wheelchairs effectively, which is not typically addressed by other treatments for spastic ataxia and myotonic dystrophy. This program uses specific assessment tools to measure and enhance wheelchair skills, making it distinct from standard medical or physical therapies. ^{2 6 7 11 12}

Eligibility Criteria

This trial is for individuals with ARSACS or Myotonic Dystrophy who rely on manual wheelchairs (MWC) for mobility. Participants should be those experiencing challenges in using their MWC effectively, which may impact their daily activities and quality of life.

Inclusion Criteria

I am over 18, diagnosed with ARSACS or DM1, and use a manual wheelchair often.

Exclusion Criteria

I am not expecting any health issues or surgeries that would stop me from training.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo a standardized wheelchair skills training program

5 weeks

Weekly sessions

Follow-up

Participants are monitored for wheelchair skills and confidence 3 months after training

3 months

1 visit (in-person or virtual)

Treatment Details

Interventions

• Wheelchair Skills Training Program

Trial Overview The study tests a Wheelchair Skills Training Program designed to improve the ability of people with ARSACS and MD1 to use their manual wheelchairs safely and confidently, potentially enhancing their independence and participation in various activities.

Participant Groups

Group I: Wheelchair Skills Training Program (WSTP) Experimental Treatment 1 Intervention

Standardized wheelchair skills training program.

Group II: Control Active Control 1 Intervention

Wait-list control group where participants will receive usual care from their clinician.

Who Is Running the Clinical Trial?

Laval University

Lead Sponsor

Trials

439

Recruited

178,000+

Findings from Research

A study involving 200 adults with myotonic dystrophy type 1 (DM1) identified upper extremity strength, particularly grip and lateral pinch strength, as strong indicators of difficulties in household activities, which can guide rehabilitation needs.

Specific cut-off scores for lateral pinch strength (4.8 kg) were established, showing good sensitivity and specificity, which can help health professionals determine when to refer DM1 patients for rehabilitation services.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessing upper extremity capacity as a potential indicator of needs related to household activities for rehabilitation services in people with myotonic dystrophy type 1. Raymond, K., Auger, LP., Cormier, MF., et al.[2015]

In a 9-year study of 113 adults with myotonic dystrophy type 1, performance-based measures like the Timed Up and Go test and Berg Balance Scale effectively captured changes in mobility, balance, and muscle strength, aligning with patient-reported outcomes.

While most measures showed promising responsiveness, grip strength and manual dexterity assessments were less responsive, highlighting the importance of understanding measurement errors when interpreting long-term changes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Responsiveness of performance-based outcome measures for mobility, balance, muscle strength and manual dexterity in adults with myotonic dystrophy type 1. Kierkegaard, M., Petitclerc, É., Hébert, LJ., et al.[2019]

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 wheelchair skills training program significantly improves manual wheelchair skills capacity in the short term, with a 13.26% increase in skills test scores compared to control groups in randomized controlled trials and a 23.44% increase in non-randomized studies.

While the training shows short-term benefits, the long-term effectiveness of manual wheelchair skills training and the effectiveness of powered wheelchair skills training remain unclear, with insufficient evidence to support lasting advantages.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and safety of wheelchair skills training program in improving the wheelchair skills capacity: a systematic review. Tu, CJ., Liu, L., Wang, W., et al.[2022]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Intrarater reliability and validity of outcome measures in myotonic dystrophy type 1. [2020]

2. England pubmed.ncbi.nlm.nih.gov

Assessing upper extremity capacity as a potential indicator of needs related to household activities for rehabilitation services in people with myotonic dystrophy type 1. [2015]

3. Sweden pubmed.ncbi.nlm.nih.gov

Responsiveness of performance-based outcome measures for mobility, balance, muscle strength and manual dexterity in adults with myotonic dystrophy type 1. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Clinical and Research Readiness for Spinal Muscular Atrophy: The Time Is Now for Knowledge Translation. [2023]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Posture and gait abilities in patients with myotonic dystrophy (Steinert disease). Evaluation on the short-term of a rehabilitation program. [2010]

6. England pubmed.ncbi.nlm.nih.gov

Measurement properties of wheelchair use assessment tools in adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function. [2018]

8. England pubmed.ncbi.nlm.nih.gov

Effectiveness and safety of wheelchair skills training program in improving the wheelchair skills capacity: a systematic review. [2022]

9. England pubmed.ncbi.nlm.nih.gov

The development of a consensus statement for the prescription of powered wheelchair standing devices in Duchenne muscular dystrophy. [2022]

10. England pubmed.ncbi.nlm.nih.gov

Parents' perceptions of power wheelchair prescription for children with a neuromuscular disorder: a scoping review. [2020]

11. England pubmed.ncbi.nlm.nih.gov

Daily activity performance in congenital and childhood forms of myotonic dystrophy type 1: a population-based study. [2020]

12. England pubmed.ncbi.nlm.nih.gov

Fatigability in spinal muscular atrophy: validity and reliability of endurance shuttle tests. [2021]