Functional and Cellular Benefits of Aerobic Exercise in Myotonic Dystrophy Type 1 Patients
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- Maximal aerobic capacity
研究概览
简要总结
Myotonic dystrophy type 1 (DM1) is a genetic disease that primarily targets skeletal muscle resulting in severe weakness and muscle loss. As a result, individuals suffering from DM1 become very inactive and lose mobility resulting in a lower quality of life. This study will investigate the effect of a 12-week moderate intensity exercise protocol on skeletal muscle function and cellular benefits in DM1 patients.
详细描述
The present study is a repeated measures design, with sampling of the cohorts on two separate occasions. Upon obtaining informed consent, participants will be assigned into one of two groups:
Group 1: DM1 Exercise Group DM1 diagnosed, physically inactive males, n = 6 DM1 diagnosed, physically inactive females, n = 7
Group 2: Healthy controls (no exercise) Healthy, physically inactive males n = 6 Healthy, physically inactive females n = 7
Introduction - Day 1 (Visit 1); On the initial visit, the potential participant will come to McMaster Children's Hospital to meet with a study coordinator involved in the project. The coordinator will explain the study in detail, answer any questions, and review the consent form. Upon obtaining informed consent, eligible participants will complete a medical screening questionnaire to determine their readiness to perform exercise. Participant characteristics will then be measured. At this time, anthropometric measurements (i.e., height, weight, waist circumference) and a dual energy x-ray absorptiometry (DXA) measurement will be performed. This must be done in order to verify that the participant meets the inclusion criteria of being normal weight (BMI 18.5 - 24.9 kg/m2) or overweight (BMI 25 - 29.9 kg/m2). Following this screening, participants will undergo an ECG to measure any conductance blockage and perform a maximal cycling test to determine their aerobic capacity (V02max). Maximal aerobic testing will then be followed by another 12 lead ECG to detect any possible structural cardiac issues in our participants. Thereafter, participants will begin the following the study timeline, all of which will occur at the McMaster Children's Hospital, 2H Neurometabolic Clinic.
Baseline testing - Day 3 (Visit 2); Participants will arrive the following day to undergo functional testing which will include 6-minute walk test, 5x sit to stand, timed up and go, grip strength and maximal voluntary knee extension using the Biodex dynamometer. Participants will then complete the SF-36.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •DM1 participants:
- •Male or female clinically diagnosed with DM1
- •CTG repeats 100-1000
- •Normal weight (BMI 18.5 - 24.9 kg/m2) or overweight (BMI 25 - 29.9 kg/m2)
- •Age matched controls:
- •Healthy men and women
- •Normal weight (BMI 18.5 - 24.9 kg/m2) or overweight (BMI 25 - 29.9 kg/m2)
- •Physically inactive (< 1-2 hour of formal exercise/week)
排除标准
- •Obese (BMI > 30.0 kg/m2)
- •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-
- •Known liver disease
- •Cognitive impairments limiting ability to provide informed consent
- •Active musculoskeletal injuries and/or severe osteoarthritis
结局指标
主要结局
Maximal aerobic capacity
时间窗: 3 months from enrolment
VO2max in mL/min/kg-1
次要结局
- 5x sit to stand(3 months from enrolment)
- Mitochondrial protein content(3 months after intervention is complete)
- 6 minute walk test(3 months from enrolment)
- Grip strength(3 months from enrolment)
- Body composition(3 months from enrolment)
- Leg muscle strength(3 months from enrolment)
- timed up and go(3 months from enrolment)
- Muscle fibre cross sectional area(3 months after intervention is complete)
研究者
Mark Tarnopolsky
Principal Investigator, Professor, Director of Neuromuscular and Neurometabolic Clinic
McMaster University