Remote Exercise Program for Myotonic Dystrophy

Recruiting at 1 trial location

Overseen By Elise Duchesne

Age: 18 - 65

Sex: Any

Trial Phase: Academic

Sponsor: Université du Québec à Chicoutimi

Disqualifiers: Already train, Language barrier, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

40 Participants Needed

Trial Summary

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 Remote activity program for Myotonic Dystrophy?

Research shows that physical exercise programs can improve functioning and quality of life in adults with myotonic dystrophy type 1, suggesting that a remote exercise program might also be beneficial. ^{1 2 3 4 5}

Is the Remote Exercise Program safe for people with myotonic dystrophy?

Research shows that a 12-week high-resistance training program improved muscle strength in patients with myotonic dystrophy without any negative side effects. ^{2 6 7 8 9}

How is the Remote Exercise Program for Myotonic Dystrophy different from other treatments?

The Remote Exercise Program for Myotonic Dystrophy is unique because it allows patients to participate in physical exercise from home using telecommunication networks, making it more accessible and convenient compared to traditional in-person therapies. This approach is particularly beneficial for those with mobility challenges, as it provides a structured exercise regimen without the need to travel to a clinic. ^{1 2 6 10 11}

What is the purpose of this trial?

The COVID-19 pandemic exacerbates health problems by reducing access to adapted and advanced physical rehabilitation for several people who need rehabilitation services, including the population with myotonic dystrophy type 1 (DM1). The PACE tool, an innovative web tool integrating pragmatic physical activity programs, seems to be an interesting and innovative intervention to counter physical deficiencies of people with DM1, which are unfortunately accentuated by the pandemic, while reducing the risk of COVID-19 exposure. Objectives: 1) Evaluate the feasibility, usability and acceptability of the PACE tool in the DM1 population; 2) Evaluate the effects of the intervention on their physical and cognitive health; and 3) Estimate the cost-effectiveness ratio of this intervention. Method: Sixty people (experimental group = 40 and control group = 20) will participate in this randomized intervention study. Participants in the experimental group will be assigned to one of the 35 physical activity programs adapted to their condition of the PACE tool. The program must be performed on a daily basis for a period of 12 weeks. Physical and cognitive health will be assessed before and after the remote intervention via ZOOM, for all participants.

Research Team

Elise Duchesne

Principal Investigator

Université du Québec à Chicoutimi

Eligibility Criteria

This trial is for people aged 18-60 with confirmed Type 1 Myotonic Dystrophy (DM1) who can exercise and consent voluntarily. It's not for those who already train more than three times a week or over 150 minutes weekly, don't speak French or English, or cannot exercise even while seated.

Inclusion Criteria

Subjects must be able to give their consent freely and voluntarily

You need to have a confirmed diagnosis of DM1 through genetic testing, regardless of your age when the symptoms started.

You are capable of exercising.

Exclusion Criteria

Don't speak French or English

You already exercise for more than 3 times a week or more than 150 minutes a week.

You cannot do any physical activity, even while sitting.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants perform daily physical activity programs using the PACE tool for 12 weeks

12 weeks

Remote assessments via ZOOM before and after the intervention

Follow-up

Participants are monitored for changes in functional capacity and physical activity levels

4 weeks

Treatment Details

Interventions

• Remote activity program

Trial Overview The study tests the PACE tool, a web-based program offering physical activity routines tailored to DM1 patients' needs. Participants will use it daily for 12 weeks to see if it improves their physical and cognitive health without risking COVID-19 exposure.

Participant Groups

Group I: Remote activity program Experimental Treatment 1 Intervention

Activity program based on objective evaluation. Possibility of 35 different programs primarily targeting impairments (lower limb, upper limb or balanced)

Who Is Running the Clinical Trial?

Université du Québec à Chicoutimi

Lead Sponsor

Trials

7

Recruited

320+

Findings from Research

A 14-week exercise program, Friskis&Svettis® Open Doors, was feasible and well-tolerated by 35 adults with myotonic dystrophy type 1, with no reported negative effects.

While the program did not show significant improvements in physical functioning or health-related quality of life, it did lead to a slight increase in mental health scores for the training group compared to controls.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Feasibility and effects of a physical exercise programme in adults with myotonic dystrophy type 1: a randomized controlled pilot study. Kierkegaard, M., Harms-Ringdahl, K., Edström, L., 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]

A 12-week high-resistance training program significantly improved muscle strength in patients with myotonic dystrophy, with one-repetition maximum (1RM) increasing from 16.4 kg to 21.8 kg (p = 0.0002).

Despite the strength gains, there were no significant changes in muscle histopathology or cross-sectional area of muscle fibers, indicating that the training was safe and did not exacerbate underlying muscle abnormalities.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of high resistance training in patients with myotonic dystrophy. Tollbäck, A., Eriksson, S., Wredenberg, A., et al.[2008]

The Myotonic Dystrophy Foundation held a workshop in 2015 to discuss the development of drug therapies for myotonic dystrophy, a progressive disorder with no current treatments, involving experts from various fields including academia, industry, and regulatory agencies.

The workshop highlighted the importance of understanding the disease's symptom burden, biomarkers, and regulatory requirements, which will aid in creating a pathway for future drug approvals specific to myotonic dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient-Centered Therapy Development for Myotonic Dystrophy: Report of the Myotonic Dystrophy Foundation-Sponsored Workshop. Hesterlee, S., Amur, S., Bain, LJ., et al.[2020]

In a randomized trial involving 42 adult patients with myotonic dystrophy type 1, mexiletine did not improve the 6-minute walk distance after 6 months, indicating no significant impact on overall mobility.

However, mexiletine did show a positive effect on hand grip myotonia, suggesting it may help with muscle stiffness, while safety assessments revealed no significant differences in adverse events or cardiac conduction between mexiletine and placebo groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial. Heatwole, C., Luebbe, E., Rosero, S., et al.[2021]

References

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

Remote assessment of myotonic dystrophy type 1: A feasibility study. [2022]

2. Sweden pubmed.ncbi.nlm.nih.gov

Feasibility and effects of a physical exercise programme in adults with myotonic dystrophy type 1: a randomized controlled pilot study. [2015]

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

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

4. 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]

5. 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]

6. Sweden pubmed.ncbi.nlm.nih.gov

Effects of high resistance training in patients with myotonic dystrophy. [2008]

7. Germany pubmed.ncbi.nlm.nih.gov

Physical activity in myotonic dystrophy type 1. [2021]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Patient-Centered Therapy Development for Myotonic Dystrophy: Report of the Myotonic Dystrophy Foundation-Sponsored Workshop. [2020]

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

Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial. [2021]

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

A pilot randomised controlled trial of a home-based exercise programme aimed at improving endurance and function in adults with neuromuscular disorders. [2021]

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

A pilot study of an integrated, personalized, respiratory and motor telerehabilitation program for pediatric patients with hereditary neuromuscular disorders. [2023]