Myotonic Dystrophy 1Phase 3DM1HBETNCT05848830
Home-based Training and Supplementation in DM1 Patients
A Phase 3 study testing Mult-ingredient supplement and Concurrent exercise training in people with Myotonic Dystrophy 1, run by McMaster University at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 19 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- Mult-ingredient supplement and Concurrent exercise training
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteCanada
- Study dates
- 1 Jan 2024to 1 Jan 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Who cannot join
- 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)
Show all 21Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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
Read the 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.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- Mult-ingredient supplement, Placebo, Concurrent exercise training
- Keywords
- myotonic dystrophy, skeletal muscle, mitochondria
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mult-ingredient supplementDietary supplement
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
PlaceboDietary supplement
The placebo sachet will contain micro-crystalline cellulose (inactive compound) and will be identical in look and taste to the active supplement.
Concurrent exercise trainingBehavioural
All participants will undergo 16-weeks of exercise training, containing 3 days/week of resistance training and 2days/ week of aerobic training.
| Study group | Role | What they receive |
|---|---|---|
| DM1 + HBEXT + MIS | Active comparator | 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. Receives: Dietary Supplement: Mult-ingredient supplement; Behavioral: Concurrent exercise training |
| DM1 + HBEXT + PLA | Placebo comparator | 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. Receives: Dietary Supplement: Placebo; Behavioral: Concurrent exercise training |
| CONTROL | No intervention | Healthy control subjects who will not undergo study intervention and will be used for baseline measurements and outcomes. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Body Composition Index
Changes in ratio of fat-free mass to fat mass index as assessed via DEXA scan
Measured: 4 months from enrolment
VO2 Max
Changes in cardiorespiratory fitness proxy measure as assessed via VO2 max testing
Measured: 4 months from enrolment
Secondary outcomes (8)
6-minute walk test
Changes in number of meters walked during the 6 minute walk test
Measured: 4 months from enrolment
5x sit to stand
Changes in time (s) that is needed to complete 5x sit to stand from a chair
Measured: 4 months from enrolment
Timed up and go
Changes in the time (seconds) needed to complete a timed up and go test
Measured: 4 months from enrolment
Leg muscle strength
Changes in maximal isometric knee extension via Biodex in N*m
Measured: 4 months from enrolment
Grip strength
Changes in grip strength (kg) using a hand dynamometer
Measured: 4 months from enrolment
Single leg stance test
Changes in whether patients can stand on one leg for 10 s or not
Measured: 4 months from enrolment
Muscle fibre cross sectional area
Changes in fibre cross sectional area from a muscle biopsy of vastus lateralis using immunoflourescence
Measured: 4 months from enrolment
Muscle stem cell mitochondrial function
Changes in mitochondrial function in isolated muscle stem cells of DM1 patients via seahorse assay
Measured: 4 months from enrolment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2023
- Study start1 Jan 2024 (estimated)
- Primary completion1 Jan 2025 (estimated)
- Study completion1 Jan 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Factorial
- 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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- McMaster University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mark Tarnopolsky, McMaster University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05848830
- Sponsor's protocol ID
- 15550
- Acronym
- DM1HBET
- Status last verified
- Oct 2023
- First submitted
- 28 Apr 2023
- Last updated
- 19 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05848830
- Acronym / sponsor ID
- DM1HBET / 15550
- Conditions
- Myotonic Dystrophy 1
- Keywords
- myotonic dystrophy, skeletal muscle, mitochondria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- McMaster UniversityUniversity, hospital or other
- Study start
- 1 Jan 2024 (estimated)
- Primary completion
- 1 Jan 2025 (estimated)
- Study completion
- 1 Jan 2025 (estimated)
- First posted
- 8 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
- 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.
| Arm | Type | Receives |
|---|---|---|
| DM1 + HBEXT + MIS | Active comparator | Dietary Supplement: Mult-ingredient supplement, Behavioral: Concurrent exercise trainingParticipants will be asked to undergo 16 weeks of home-based training and asked to take one dose of a multi-ingredient supplement per day. |
| DM1 + HBEXT + PLA | Placebo comparator | Dietary Supplement: Placebo, Behavioral: Concurrent exercise trainingParticipants 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. |
| CONTROL | No intervention | –Healthy control subjects who will not undergo study intervention and will be used for baseline measurements and outcomes. |
Who may qualify
- Ages
- 19 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (21 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mult-ingredient supplementA 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 | Dietary supplement | DM1 + HBEXT + MIS |
| PlaceboThe placebo sachet will contain micro-crystalline cellulose (inactive compound) and will be identical in look and taste to the active supplement. | Dietary supplement | DM1 + HBEXT + PLA |
| Concurrent exercise trainingAll participants will undergo 16-weeks of exercise training, containing 3 days/week of resistance training and 2days/ week of aerobic training. | Behavioural | DM1 + HBEXT + MIS, DM1 + HBEXT + PLA |
Primary outcome
Body Composition Index
Time frame: 4 months from enrolment
Changes in ratio of fat-free mass to fat mass index as assessed via DEXA scan
VO2 Max
Time frame: 4 months from enrolment
Changes in cardiorespiratory fitness proxy measure as assessed via VO2 max testing
8 secondary outcomes
6-minute walk test
Time frame: 4 months from enrolment
5x sit to stand
Time frame: 4 months from enrolment
Timed up and go
Time frame: 4 months from enrolment
Leg muscle strength
Time frame: 4 months from enrolment
Grip strength
Time frame: 4 months from enrolment
Single leg stance test
Time frame: 4 months from enrolment
Muscle fibre cross sectional area
Time frame: 4 months from enrolment
Muscle stem cell mitochondrial function
Time frame: 4 months from enrolment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mult-ingredient supplement and Concurrent exercise training in people with Myotonic Dystrophy 1, run by McMaster University at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mark A Tarnopolsky, MD, PhD (905-525-9140 ext. 75226, tarnopol@mcmaster.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Body Composition Index (time frame: 4 months from enrolment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2023.