Remote Exercise Program for Myotonic Dystrophy

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new online tool called PACE, designed for individuals with myotonic dystrophy type 1 (DM1), a genetic disorder that causes muscle weakness and stiffness. The trial will evaluate whether using the tool for daily exercise over 12 weeks can improve physical and mental health without requiring clinic visits, which is particularly beneficial during the COVID-19 pandemic. Participants will be randomly assigned to either use the PACE tool with tailored exercise programs (remote activity program) or join a control group. This trial suits adults diagnosed with DM1 who can exercise and are not already exercising frequently. As an unphased trial, it offers a unique opportunity to contribute to innovative research that could enhance the quality of life for those with DM1.

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 prior data suggests that the PACE tool is safe for people with myotonic dystrophy type 1?

Research has shown that a remote exercise program can be safe and manageable for people with myotonic dystrophy type 1 (DM1). In one study, 35 adults with DM1 successfully completed a similar 14-week exercise program without any negative feedback, suggesting these programs usually do not cause harm.

Another study confirmed that home-based exercise programs are both effective and well-received by people with DM1. Participants managed these exercises without any problems, indicating they can handle this type of activity.

In general, aerobic exercise, like walking or cycling, has proven safe for people with neuromuscular diseases like DM1. This kind of exercise often brings health benefits without causing harm. Overall, these findings suggest that remote exercise programs can be a safe choice for those with DM1. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the remote exercise program for myotonic dystrophy because it offers a flexible and personalized approach that current treatments don't typically provide. Unlike traditional therapies that often require in-person visits, this program allows patients to engage in tailored exercise routines from the comfort of their own home. This remote delivery method is not only convenient but can also be customized to address specific impairments such as lower limb, upper limb, or balance issues with up to 35 different programs. This level of personalization and accessibility has the potential to improve adherence and outcomes for individuals with myotonic dystrophy.

What evidence suggests that this remote activity program is effective for myotonic dystrophy?

Research has shown that remote exercise programs can help people with myotonic dystrophy type 1 (DM1) improve their physical health. Participants in these programs reported better muscle function and overall fitness. One study found that a personalized exercise plan led to noticeable improvements in muscle strength and movement. Participants also noted that these programs were easy to follow and beneficial for their health. Additionally, exercising from home can reduce the risk of catching COVID-19 while still providing effective rehabilitation. This trial will evaluate a remote activity program offering 35 different options targeting impairments such as lower limb, upper limb, or balance. These findings suggest that remote exercise programs could be a promising way to manage DM1 symptoms. ^{3 6 7 8 9}

Who Is on the Research Team?

Elise Duchesne

Principal Investigator

Université du Québec à Chicoutimi

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05072288

A Remote Physical Activity Program in the Population ... A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy · Study Overview · Contacts and Locations · Participation Criteria.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35426155/

Remote assessment of myotonic dystrophy type 1 We tested the feasibility of using RSVs to evaluate patients with myotonic dystrophy type 1 (DM1), including remote quantitative assessment of muscle function.

3. withpower.com withpower.com/trial/phase-myotonic-dystrophy-7-2021-503aa

Remote Exercise Program for Myotonic Dystrophy 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 ...

4. researchgate.net researchgate.net/publication/347160434_Effects_and_Acceptability_of_an_Individualized_Home-Based_10-Week_Training_Program_in_Adults_with_Myotonic_Dystrophy_Type_1

(PDF) Effects and Acceptability of an Individualized Home- ... The program and the device were well accepted and all participants reported many perceived improvements at the end of the program. Conclusions: ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12141421/

Efficacy of Combined Aerobic Exercise and Coaching on ... The aim of this study was to evaluate the efficacy of combined personalized home-based aerobic exercise and coaching on physical fitness in people with NMD, ...

6. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT05072288?term=myotonic+dystrophy+type+1&recrs=abc&draw=2&rank=10

Study Details | A Remote Physical Activity Program in the ... There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/33252090/

Effects and Acceptability of an Individualized Home-Based ... Our results provide encouraging data on the effects and acceptability of a home-based training program for men with the adult form of DM1.

8. sciencedirect.com sciencedirect.com/science/article/pii/S0960896625001488

Levels of exercise exposure among people living with ... Aerobic exercise is the most explored modality of exercise and has been found to be safe and likely beneficial for patients with NMDs [[6], [7], [8], [9]].

9. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6402179/

What is known about the effects of exercise or training to ... Results reported for every patient individually, some show improvements in strength and other have no significant changes. No adverse effects ...