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Myotonic Dystrophy Type 1 Aerobic Exercise Study (DM1ex)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04187482
Recruitment Status : Active, not recruiting
First Posted : December 5, 2019
Last Update Posted : May 27, 2020
Sponsor:
Information provided by (Responsible Party):
Mark Tarnopolsky, McMaster University

Tracking Information
First Submitted Date  ICMJE December 3, 2019
First Posted Date  ICMJE December 5, 2019
Last Update Posted Date May 27, 2020
Actual Study Start Date  ICMJE December 6, 2019
Estimated Primary Completion Date July 29, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 5, 2019)
Maximal aerobic capacity [ Time Frame: 3 months from enrolment ]
VO2max in mL/min/kg-1
Original Primary Outcome Measures  ICMJE
 (submitted: December 3, 2019)
Maximal aerobic capacity (VO2max) [ Time Frame: 3 months from enrolment ]
Maximal rate of oxygen consumption during progressive exercise
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 5, 2019)
  • 6 minute walk test [ Time Frame: 3 months from enrolment ]
    changes in number of meters walked during the 6 minute walk test
  • 5x sit to stand [ Time Frame: 3 months from enrolment ]
    changes in the time (seconds) needed to complete 5x sit to stand from a chair
  • timed up and go [ Time Frame: 3 months from enrolment ]
    changes in the time (seconds) needed to complete a timed up and go test
  • Leg muscle strength [ Time Frame: 3 months from enrolment ]
    maximal isometric knee extension via Biodex in N*m
  • Grip strength [ Time Frame: 3 months from enrolment ]
    changes in grip strength (kg) using a hand dynamometer
  • Body composition [ Time Frame: 3 months from enrolment ]
    change in fat free mass and fat mass measured via DXA in kg
  • Muscle fibre cross sectional area [ Time Frame: 3 months after intervention is complete ]
    changes in fibre cross sectional area from muscle biopsy of vastus lateralis using immunoflourescence
  • Mitochondrial protein content [ Time Frame: 3 months after intervention is complete ]
    changes in mitochondrial proteins from muscle biopsy of vastus lateralis using western blotting
Original Secondary Outcome Measures  ICMJE
 (submitted: December 3, 2019)
  • Functional testing [ Time Frame: 3 3 months from enrolment ]
    6 minute walk test, timed up and go, 5x sit to stand
  • Strength testing [ Time Frame: 3 months from enrolment ]
    maximal isometric knee extension, grip strength, pinch grip
  • Total lean mass [ Time Frame: 3 months from enrolment ]
    change in total lean mass using dual-energy X-ray absorptiometry
  • biochemical analysis [ Time Frame: 3 months after intervention is complete ]
    mitochondrial protein content, fibre cross sectional area
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Myotonic Dystrophy Type 1 Aerobic Exercise Study
Official Title  ICMJE Functional and Cellular Benefits of Aerobic Exercise in Myotonic Dystrophy Type 1 Patients
Brief Summary 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.
Detailed Description

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.

Follow up testing - Day 5 (Visit 3); Participants will arrive following an overnight fast (including no caffeine for 12 hours) and having abstained from any exercise for the prior 24 hours. Participants will undergo a muscle biopsy from the vastus lateralis.

DM1 participants only:

Exercise training - Visit 4 - 39; Participants will begin the exercise protocol consisting of 3 exercise sessions per week for a 12-week period. All exercise sessions will be done on a cycle ergometer (Lode, Groningen, Netherlands). Each exercise session will consist of 3 minutes of warm up, 30 minutes at 65% V02max and will end with 2 minutes of cool down. Exercise progression over the 12 weeks will go as follows:

  • Weeks 1 and 2: 3 min warm up, 65% VO2max for 30 min, 2 min cool down
  • Weeks 3 and 4: increase to 35 min (+ warm up/cool down)
  • Weeks 5 and 6: drop to 30 min and increase to 70% VO2max
  • Weeks 7 and 8: increase to 35 min (+ warm up/cool down)
  • Weeks 9 and 10: drop to 30 min and increase to 75%VO2 max
  • Weeks 11 and 12: increase to 35 min (+ warm up/cool down)

Endpoint testing - Visit 40; At this time, anthropometric re-measurements (i.e., height, weight, waist circumference) and a dual energy x-ray absorptiometry (DXA) measurement will be performed. Thereafter, participants will undergo an ECG to measure any conductance blockage and perform a maximal cycling test to determine their aerobic capacity (V02peak). Maximal aerobic testing will then be followed by another 12 lead ECG to detect any possible structural cardiac issues in our participants.

Endpoint testing - Visit 41; Participants will arrive the following day to undergo functional testing which will include 6-minute walk test, timed up and go, grip strength and maximal voluntary knee extension using the Biodex dynamometer. Participants will then complete the SF-36.

Endpoint testing - Visit 42; Participants will arrive following an overnight fast (including no caffeine for 12 hours) and having abstained from any exercise for the prior 24 hours. Participants will undergo a muscle biopsy from the vastus lateralis.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Participants are undergoing 12-weeks of aerobic exercise training
Masking: None (Open Label)
Primary Purpose: Supportive Care
Condition  ICMJE
  • Myotonic Dystrophy 1
  • Muscular Dystrophies
Intervention  ICMJE Other: Exercise training
All participants with DM1 will undergo a 12-week aerobic exercise training intervention on a cycle ergometer
Study Arms  ICMJE Experimental: Exercise training group
All participants will be receiving same exercise training intervention
Intervention: Other: Exercise training
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 3, 2019)
13
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 1, 2020
Estimated Primary Completion Date July 29, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

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)

Exclusion Criteria:

  • Smoking
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04187482
Other Study ID Numbers  ICMJE 7091
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Mark Tarnopolsky, McMaster University
Study Sponsor  ICMJE McMaster University
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account McMaster University
Verification Date May 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP