Exercise + Multivitamin Supplement for Myotonic Dystrophy

(DM1HBET Trial)

Overseen By Joshua P Nederveen, PhD

Age: 18 - 65

Sex: Any

Trial Phase: Phase 3

Sponsor: McMaster University

Must not be taking: Narcotics, Anti-inflammatories, Corticosteroids, others

Disqualifiers: Obesity, Diabetes, Cardiovascular disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how a combination of exercise and a multivitamin supplement might benefit individuals with myotonic dystrophy type 1 (DM1), a genetic condition that weakens muscles and complicates daily tasks. The researchers aim to determine if these treatments can enhance muscle health and overall quality of life. Participants will either take the multivitamin supplement or a placebo (a harmless pill resembling the real supplement) while following a home-based exercise plan for 16 weeks. Those diagnosed with DM1, who are not very active and have normal or slightly above-normal weight, might be suitable for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you cannot participate if you use narcotic painkillers, anti-inflammatory drugs, or medications affecting protein metabolism. If you have certain conditions like diabetes or asthma, you may need to limit the number of medications you use.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

A previous study showed that exercise improved certain physical abilities, such as walking and standing, without causing serious side effects. Specifically, participants experienced no harmful effects and improved in tasks like the 6-minute walk test and timed stands test. Other research indicates that aerobic exercise is safe and likely beneficial for individuals with neuromuscular diseases, such as myotonic dystrophy.

Regarding the multi-ingredient supplement (MIS), caution is advised, especially for those with muscle disorders. Some supplements may have side effects or interact harmfully with other medications. However, the specific supplement used in this study was tested in at least one previous human trial, which suggested it was well-tolerated.

Overall, exercise appears safe and possibly beneficial for muscle function. While supplements require careful use, this particular one has shown some safety in earlier trials. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about this trial because it explores a combined approach of exercise and multivitamin supplements for managing myotonic dystrophy, a condition traditionally managed through physical therapy and medications like mexiletine. This trial is unique because it pairs structured home-based exercise with a multi-ingredient supplement, potentially offering a more holistic and accessible management strategy. Unlike standard treatments that primarily focus on symptom relief, this approach might enhance overall muscle function and quality of life through lifestyle modifications. The trial aims to uncover whether this combination can deliver benefits beyond the conventional methods, making it a promising area of research.

What evidence suggests that this trial's treatments could be effective for myotonic dystrophy?

This trial will evaluate the effects of exercise and a multi-ingredient supplement on individuals with myotonic dystrophy type 1 (DM1). Studies have shown that exercise can greatly improve muscle strength and reduce muscle problems in people with DM1. Strength training, in particular, has been linked to stronger muscles and less apathy and anxiety. In this trial, one group of participants will undergo home-based exercise training and take a multi-ingredient supplement, which includes components like thiamine and Coenzyme Q10 (CoQ10). Some research suggests that high doses of thiamine can boost muscle strength, and CoQ10 has been found to help muscle function in muscular dystrophy. Another group will undergo the same exercise regimen but will receive a placebo supplement. While evidence strongly supports the benefits of exercise, the supplement's effectiveness is based on smaller studies and requires further research. Both methods aim to improve muscle health and overall quality of life for those with DM1. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for men and women aged 19-60 with Myotonic Dystrophy type 1 (DM1), having CTG repeats between 100-1000. Participants should have a BMI of 18.5-29.9, be physically inactive, and able to walk between 250-500 meters in six minutes without severe heart rhythm abnormalities.

Inclusion Criteria

I am between 19 and 60 years old and have been diagnosed with DM1.

My genetic test shows I have 100-1000 CTG repeats.

I am a healthy man or woman.

Exclusion Criteria

My BMI is over 30.

I need more than 2 medications to manage my high blood pressure.

Smoking

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo 16 weeks of home-based concurrent training with either a multi-ingredient supplement or placebo

16 weeks

Baseline and post-intervention visits for measurements and sample collection

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Control Group Baseline

Healthy control subjects participate in baseline measurements and outcomes

What Are the Treatments Tested in This Trial?

Interventions

• Concurrent exercise training
• Mult-ingredient supplement
• Placebo

Trial Overview The study tests if a home-based exercise program combined with either a multi-ingredient supplement or placebo can improve muscle function and body composition in DM1 patients over the course of 16 weeks.

How Is the Trial Designed?

Group I: DM1 + HBEXT + MIS Active Control 2 Interventions

Participants will be asked to undergo 16 weeks of home-based training and asked to take one dose of a multi-ingredient supplement per day.

Group II: CONTROL Active Control 1 Intervention

Healthy control subjects who will not undergo study intervention and will be used for baseline measurements and outcomes.

Group III: DM1 + HBEXT + PLA Placebo Group 2 Interventions

Participants will be asked to undergo 16 weeks of home-based training and asked to take one dose of a multi-ingredient supplement placebo per day.

Who Is Running the Clinical Trial?

McMaster University

Lead Sponsor

Trials

936

Recruited

2,630,000+

Citations

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

What is known about the effects of exercise or training to ... It is to be noted that in some of these studies, strength improvement was not observed in all participants, in all muscle groups trained or in ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S2215016124004084

Effect of exercise training on clinical and physiological ... Strength-training effectively alleviates skeletal muscle impairments in myotonic dystrophy type 1. Neuromuscul. Disord., 30 (4) (2020), pp. 283-293, 10.1016 ...

3. neurologylive.com neurologylive.com/view/strength-training-provides-several-noted-benefits-women-myotonic-dystrophy-type-1

Strength Training Provides Several Noted Benefits to ... 12-week supervised strength training program resulted in increased maximal hip and knee extension muscle strength, lowered apathy and anxiety, and induced ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04187482

Myotonic Dystrophy Type 1 Aerobic Exercise Study (DM1ex) As a result, individuals suffering from DM1 become very inactive and lose mobility resulting in a lower quality of life.

5. nmd-journal.com nmd-journal.com/article/S0960-8966(24)00106-8/fulltext

Resistance training in women with myotonic dystrophy type 1 Fundamental and clinical positive effects of strength training have been reported in men with DM1, but its impact on women remains unknown.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12426088/

Efficacy and safety of respiratory strength and endurance ... Twenty-six participants completed the study. The intervention was safe, with no serious adverse events. Significant improvements in primary ...

7. 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]].

8. medicaljournals.se medicaljournals.se/jrm/content/html/10.2340/16501977-2871

A Cochrane Review summary with commentary - HTML One trial with 52 participants reported that aerobic exercise may have little or no effect on muscle strength and pain, but may improve time-scored functional ...

9. ajmc.com ajmc.com/view/strength-training-safely-boosts-muscular-dystrophy-outcomes-but-further-study-needed

Strength Training Safely Boosts Muscular Dystrophy ... Adults living with myotonic dystrophy type 1 (DM1) were able to better their perceived exertion rate and gate, as well (ES range, 0.92-3.83).