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Home-based Training and Supplementation in DM1 Patients (DM1HBET)

Primary Purpose

Myotonic Dystrophy 1

Status
Not yet recruiting
Phase
Phase 3
Locations
Canada
Study Type
Interventional
Intervention
Mult-ingredient supplement
Placebo
Concurrent exercise training
Sponsored by
McMaster University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Myotonic Dystrophy 1 focused on measuring myotonic dystrophy, skeletal muscle, mitochondria

Eligibility Criteria

19 Years - 60 Years (Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

  • McMaster University Medical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Placebo Comparator

No Intervention

Arm Label

DM1 + HBEXT + MIS

DM1 + HBEXT + PLA

CONTROL

Arm Description

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.

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.

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

Outcomes

Primary Outcome Measures

Body Composition Index
Changes in ratio of fat-free mass to fat mass index as assessed via DEXA scan
VO2 Max
Changes in cardiorespiratory fitness proxy measure as assessed via VO2 max testing

Secondary Outcome Measures

6-minute walk test
Changes in number of meters walked during the 6 minute walk test
5x sit to stand
Changes in time (s) that is needed to complete 5x sit to stand from a chair
Timed up and go
Changes in the time (seconds) needed to complete a timed up and go test
Leg muscle strength
Changes in maximal isometric knee extension via Biodex in N*m
Grip strength
Changes in grip strength (kg) using a hand dynamometer
Single leg stance test
Changes in whether patients can stand on one leg for 10 s or not
Muscle fibre cross sectional area
Changes in fibre cross sectional area from a muscle biopsy of vastus lateralis using immunoflourescence
Muscle stem cell mitochondrial function
Changes in mitochondrial function in isolated muscle stem cells of DM1 patients via seahorse assay

Full Information

First Posted
April 28, 2023
Last Updated
October 18, 2023
Sponsor
McMaster University
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1. Study Identification

Unique Protocol Identification Number
NCT05848830
Brief Title
Home-based Training and Supplementation in DM1 Patients
Acronym
DM1HBET
Official Title
The Effects of Home-based Exercise Training and Multi-ingredient Supplementation on Functional Outcomes and Skeletal Muscle Adaptations in Patients With Myotonic Dystrophy Type 1
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
January 1, 2024 (Anticipated)
Primary Completion Date
January 1, 2025 (Anticipated)
Study Completion Date
January 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
McMaster University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Myotonic dystrophy type 1 (DM1) is a rare genetic disease that affects about 1 in 2100 people. Patients diagnosed with DM1 present with many symptoms, however, their muscles are mainly affected. DM1 patients experience a gradual loss of muscle, followed by an increase in body fat percentage, which makes them weaker, resulting in difficulties to perform activities of daily living, such as climbing stairs, and understandably, this affects their quality of life. DM1 currently does not have a cure. Therefore, it is very important to find ways in which we can help DM1 patients to improve their symptoms, and hopefully, improve their quality of life, and possibly improve disease prognosis. Exercise is known to improve muscle quality and function. In addition, we hypothesize that a multi-ingredient supplement (MIS) for muscle health and antioxidants for fat loss, might show improved benefits on top of exercise. Therefore, we will investigate the effects of 16-week home-based concurrent training, with MIS or placebo, on body composition, and functional measures. Lastly, we will investigate muscle adaptations in DM1 and following study intervention
Detailed Description
The present study will target patients (male and female) that have been clinically diagnosed with myotonic dystrophy type 1 with 100-1000 CTG repeats, between the ages of 19 - 60. In addition, a healthy population will serve as baseline controls, and they will be matched for age and sex, to the DM1 patients. A total of 40 DM1 patients and 20 healthy controls will be recruited. DM1 patients will be randomized to 20 in the active group and 20 in the placebo. Both males and females will be recruited, targeting 20 males and 20 females. Corresponding 10 healthy males and 10 healthy females will be matched for sex and age, and only participate in baseline visits, measures, and sample collection. Home-based concurrent training (HBCT) represents an exercise intervention with minimal barriers to entry. Our study will assess the effectiveness of this intervention, in combination with a multi-ingredient supplement (MIS) containing protein, creatine, antioxidants, and other dietary supplements in patients with DM1, in improving functional, clinical, and strength measures, as well as the quality of life, as assessed by scores in questionnaires. In addition, we will investigate adaptations to skeletal muscle following HBCT and MIS. A randomized clinical trial of DM1 patients. Our study will include exercise as the over-arching intervention, with two groups nested within, DM1 patients randomized to MIS and placebo, both of which will undergo 16 weeks of home-based exercise training. Measurements and samples will be collected before and after the study intervention. A third group will include age and sex-matched healthy controls. This group will not undergo training + supplement intervention and will be used for baseline comparisons between healthy participants and DM1 patients. Study procedures will include blood draws and muscle biopsies in each visit. In addition, participants will be asked to undergo a cardiorespiratory fitness test (VO2MAX test), a battery of functional tests (6-minute walk test, 5x sit-to-stand, one leg standing, 4-stair climb), strength testing (grip strength and knee extension tests) and lastly, clinical procedures, including ECGs and spirometry. Measurements and sample collection will be done before and after the study intervention.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myotonic Dystrophy 1
Keywords
myotonic dystrophy, skeletal muscle, mitochondria

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 3
Interventional Study Model
Factorial Assignment
Model Description
All participants will undergo 16 weeks of home-based exercise training. Half of them are randomized in an active group with a multi-ingredient supplement, and half will be given a placebo. Both the participants and the investigators are blinded to the groups.
Masking
ParticipantCare ProviderInvestigator
Masking Description
Both the participants and the investigators are blinded to the active and placebo groups. A third party, Gruppo Nutrition (Windsor, ON) will conduct the randomization for our supplemental interventions and manage blinding procedures. Neither the participants nor the investigators will be unblinded to the supplement randomization. Lastly, researchers will only be unblinded upon finishing the data analysis.
Allocation
Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
DM1 + HBEXT + MIS
Arm Type
Active Comparator
Arm Description
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.
Arm Title
DM1 + HBEXT + PLA
Arm Type
Placebo Comparator
Arm Description
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.
Arm Title
CONTROL
Arm Type
No Intervention
Arm Description
Healthy control subjects who will not undergo study intervention and will be used for baseline measurements and outcomes.
Intervention Type
Dietary Supplement
Intervention Name(s)
Mult-ingredient supplement
Intervention Description
A mult-ingredient supplement containing Protein, both whey and casein, Creatine, Vitamin D, and Calcium. As well as antioxidants and other dietary supplements including Vitamin E, CoQ10, Alpha Lipoic Acid, L-Arginine, Beet Root Extract, Green Coffee Bean Extract, Green Tea Extract, Black Tea Extract, Curcumin, and Forskolin
Intervention Type
Dietary Supplement
Intervention Name(s)
Placebo
Intervention Description
The placebo sachet will contain micro-crystalline cellulose (inactive compound) and will be identical in look and taste to the active supplement.
Intervention Type
Behavioral
Intervention Name(s)
Concurrent exercise training
Intervention Description
All participants will undergo 16-weeks of exercise training, containing 3 days/week of resistance training and 2days/ week of aerobic training.
Primary Outcome Measure Information:
Title
Body Composition Index
Description
Changes in ratio of fat-free mass to fat mass index as assessed via DEXA scan
Time Frame
4 months from enrolment
Title
VO2 Max
Description
Changes in cardiorespiratory fitness proxy measure as assessed via VO2 max testing
Time Frame
4 months from enrolment
Secondary Outcome Measure Information:
Title
6-minute walk test
Description
Changes in number of meters walked during the 6 minute walk test
Time Frame
4 months from enrolment
Title
5x sit to stand
Description
Changes in time (s) that is needed to complete 5x sit to stand from a chair
Time Frame
4 months from enrolment
Title
Timed up and go
Description
Changes in the time (seconds) needed to complete a timed up and go test
Time Frame
4 months from enrolment
Title
Leg muscle strength
Description
Changes in maximal isometric knee extension via Biodex in N*m
Time Frame
4 months from enrolment
Title
Grip strength
Description
Changes in grip strength (kg) using a hand dynamometer
Time Frame
4 months from enrolment
Title
Single leg stance test
Description
Changes in whether patients can stand on one leg for 10 s or not
Time Frame
4 months from enrolment
Title
Muscle fibre cross sectional area
Description
Changes in fibre cross sectional area from a muscle biopsy of vastus lateralis using immunoflourescence
Time Frame
4 months from enrolment
Title
Muscle stem cell mitochondrial function
Description
Changes in mitochondrial function in isolated muscle stem cells of DM1 patients via seahorse assay
Time Frame
4 months from enrolment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
19 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Mark A Tarnopolsky, MD, PhD
Phone
905-525-9140
Ext
75226
Email
tarnopol@mcmaster.ca
First Name & Middle Initial & Last Name or Official Title & Degree
Joshua P Nederveen, PhD
Phone
905-902-0583
Email
nedervj@mcmaster.ca
Facility Information:
Facility Name
McMaster University Medical Center
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No

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Home-based Training and Supplementation in DM1 Patients

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