Home-based Training and Supplementation in DM1 Patients

Launched by MCMASTER UNIVERSITY · Apr 28, 2023

Keywords

ClinConnect Summary

This clinical trial is exploring how home-based exercise training, combined with a special supplement, can help improve the health and quality of life for people with myotonic dystrophy type 1 (DM1). DM1 is a rare genetic condition that primarily affects muscles, leading to weakness and difficulties with everyday activities. The study will last for 16 weeks and will assess how this training and supplementation might help with muscle strength, body composition, and overall function in patients.

To participate, individuals must be between 19 and 60 years old and have a confirmed diagnosis of DM1. They should also be physically inactive, meaning they don’t engage in regular exercise, and have scores within a specific range on a walking test. However, there are several health conditions that could exclude someone from participating, such as obesity or significant heart problems. Those who join the study can expect to receive either the supplement or a placebo (a substance with no active ingredients) while engaging in the training program. This research is important as there is currently no cure for DM1, and finding ways to improve patients' lives is crucial.

Gender

ALL

Eligibility criteria

• Age and sex-matched controls inclusion criteria:
• • Healthy men and women
• • Normal BMI (BMI 18.5 - 24.9 kg/m2)
• Inclusion Criteria for DM1 patients:
• • Male or female clinically diagnosed with DM1 (age 19 - 60 y).
• • CTG repeats 100-1000.
• • Normal weight (BMI 18.5 - 24.9 kg/m2) or overweight (BMI 25 - 29.9 kg/m2).
• • Physically inactive (\< 1 hour of formal exercise/week).
• • 6-minute walk test score between 250 - 500 meters
• • ECG with PR interval \< 225 ms and QRS duration \< 125 ms.
• Exclusion Criteria for DM1 patients:
• • Smoking
• • Obese (BMI \> 30.0 kg/m2)
• • Physically active (\> 1-2 hour of formal exercise/week)
• • 6-minute walk test score \<250 meters, chronic (\> 2 weeks)
• • Use of narcotic analgesic or anti-inflammatory drugs
• • Type 1 or 2 diabetes (more than one anti-diabetic drug)
• • Cardiovascular disease (recent myocardial infarction (\< 6 months)
• • Uncontrolled hypertension requiring more than 2 medications.
• • Congestive heart failure requiring more than one medication for control.
• • Cardiac conduction block (as above)
• • Renal disease (creatinine \> 140)
• • Known liver disease
• • Cognitive impairments limiting ability to provide informed consent
• • Previous stroke with residual hemiparesis
• • Active musculoskeletal injuries and/or severe osteoarthritis
• • Significant weight loss in the 3-month period prior to the study
• • Severe peripheral neuropathy
• • Severe osteoporosis
• • Use of medications known to affect protein metabolism (i.e. corticosteroids)
• • Chronic obstructive or restrictive pulmonary disease (FVC \< 70% of age predicted mean value)
• • Asthma requiring more than two medications.