Myotonic Dystrophy 1NCT04001920
Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1
A study testing Training program in people with Myotonic Dystrophy 1, run by Cynthia Gagnon at 1 site in Canada.
Trial summary
A strength and endurance training program was conducted in adults with myotonic dystrophy type 1 (DM1). Participants underwent a 12-week/18-session supervised training program consisted of 6 exercises: elbow flexion/extension, shoulder horizontal adduction, leg press, and knee flexion/extension. Read more about this study
- Who is it for?
- Ages 20 to 60 years
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- Training program
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteCanada
- Study dates
- 23 Jul 2013to 13 Dec 2013
- Phase
- Interventional
- 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 20 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adults with DM1
- Walk without technical aid
- Muscle impairment rating scale (MIRS) of 3 or 4
Who cannot join
- Severe musculoskeletal disorders
- Any medical contraindication to physical exercise
- Any medical contraindication to muscle biopsies for the biopsy volunteers
Trial locations and contacts
1 site in 1 country.
Canada1 site
Groupe de recherche interdisciplinaire sur les maladies neuromusculaires
Saguenay, Quebec, G7X 7X2
About this study
Official title: Effects of a Multiple Component Training Program on Muscles, Maximal Muscle Strength, Endurance and Functional Performance in Adults With Myotonic Dystrophy Type 1: A Pilot Study
A strength and endurance training program was conducted in adults with myotonic dystrophy type 1 (DM1). Participants underwent a 12-week/18-session supervised training program consisted of 6 exercises: elbow flexion/extension, shoulder horizontal adduction, leg press, and knee flexion/extension. To offer a complete training program aimed at improving function it was divided: the first 6 weeks were dedicated to strength-training, whereas the following weeks focused on endurance-training. To evaluate the effects of the training program, participants completed maximal strength, endurance and functional evaluations. Volunteers could also add a muscle biopsy at the beginning and the end of the program to evaluate physiological parameters.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- Training program
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Training programOther
12-week, 18-session training program. To offer a complete training program aimed at improving function it was divided : the first 6 weeks were dedicated to strength-training (2 sets of 6 repetitions at 80% of 1-RM), whereas the following weeks focused on endurance-training (1 set of 25 repetitions at 40% of 1-RM).
| Study group | Role | What they receive |
|---|---|---|
| Training program | Experimental | 12-week strength and endurance training program Receives: Other: Training program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in maximal muscle strength in the knee extensors measured by quantified muscle testing
Changes in knee extensor maximal muscle strength evaluated by quantified muscle testing with a handheld dynamometer.
Measured: Before and after the 12-week training program
Secondary outcomes (12)
Change in 6 minute walking test disance
Walking endurance change measured by the 6 minute walking test distance.
Measured: Before and after the 12-week training program
Changes in functional capacity measured by the UQAM-YMCA test
Functional capacity evaluated by 18 functional tests
Measured: Before and after the 12-week training program
Changes in sprint capacity measured by the Running-base anaerobic sprint test
Sprint capacity changes measured by the running-base anaerobic sprint test
Measured: Before and after the 12-week training program
Changes in fatigue measured by the Fatigue severity scale
Fatigue questionnaire: 9-item questionnaire rated on a scale from 1 to 7 for each item. A score a higher score means more fatigue.
Measured: Before and after the 12-week training program
Changes in daytime sleepiness measured by the Daytime sleepiness scale (Epworth Sleepiness Scale)
8-item questionnaire, rated on a scale from 0 to 3 for each item, the higher the score, the more the patient has daytime sleepiness.
Measured: Before and after the 12-week training program
Changes in stumbles and falls measured by the Falls efficacy scale
10-item questionnaire about stumbles and falls. All items are rated on a 10-point scale. A higher score means a higher fear of falling. A score over 70 means a significant fear of falling.
Measured: Before and after the 12-week training program
Changes in quality of life measured by the 36-Item Short Form Survey (SF-36)
36-item quality of life questionnaire. The final score is calculated on a total of 100 where the lower the score, the more disability. A score of 100 means no disability.
Measured: Before and after the 12-week training program
Changes in life habits measured by questionnaire
Life habits questionnaire Assessment of life habits is a 77-item questionnaire with a scale from 0 to 9 for each item. A higher score means no disability and a low score means more disability.
Measured: Before and after the 12-week training program
Changes in muscle biopsy-muscle fiber size
To assess changes in muscle fiber size
Measured: Before and after the 12-week training program for volunteers
Changes in muscle biopsy-muscle fiber typing
To assess changes in muscle fiber typing
Measured: Before and after the 12-week training program for volunteers
Changes in muscle biopsy-proportion of centrally nuclated fibers
To assess changes in muscle centrally nucleated fibers proportion
Measured: Before and after the 12-week training program for volunteers
Changes in muscle biopsy-leucocyte infiltration
To assess changes in muscle fiber leucocyte infiltration
Measured: Before and after the 12-week training program for volunteers
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2019
- Study start23 Jul 2013
- Primary completion13 Dec 2013
- Study completion13 Dec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cynthia Gagnon
- Sponsor type
- University, hospital or other
- Collaborators
- Fondation du Grand défi Pierre Lavoie
- Responsible party
- Sponsor-investigator, Cynthia Gagnon, Université de Sherbrooke
- Investigators
- Cynthia Gagnon, Ph.D., Principal investigator, Université de Sherbrooke
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04001920
- Sponsor's protocol ID
- 2013-008
- Status last verified
- Jun 2019
- First submitted
- 21 Jun 2019
- Last updated
- 28 Jun 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Roussel MP, Morin M, Girardin M, Fortin AM, Leone M, Mathieu J, Gagnon C, Duchesne E. Training program-induced skeletal muscle adaptations in two men with myotonic dystrophy type 1. BMC Res Notes. 2019 Aug 20;12(1):526. doi: 10.1186/s13104-019-4554-z.
PubMed 31429798
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of a Multiple Component Training Program on Muscles, Maximal Muscle Strength, Endurance and Functional Performance in Adults With Myotonic Dystrophy Type 1: A Pilot Study
- ClinicalTrials.gov ID
- NCT04001920
- Acronym / sponsor ID
- 2013-008
- Conditions
- Myotonic Dystrophy 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Cynthia GagnonUniversity, hospital or other
- Collaborators
- Fondation du Grand défi Pierre Lavoie
- Study start
- 23 Jul 2013 (actual)
- Primary completion
- 13 Dec 2013 (actual)
- Study completion
- 13 Dec 2013 (actual)
- First posted
- 28 Jun 2019 (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.
A strength and endurance training program was conducted in adults with myotonic dystrophy type 1 (DM1). Participants underwent a 12-week/18-session supervised training program consisted of 6 exercises: elbow flexion/extension, shoulder horizontal adduction, leg press, and knee flexion/extension. To offer a complete training program aimed at improving function it was divided: the first 6 weeks were dedicated to strength-training, whereas the following weeks focused on endurance-training. To evaluate the effects of the training program, participants completed maximal strength, endurance and functional evaluations. Volunteers could also add a muscle biopsy at the beginning and the end of the program to evaluate physiological parameters.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Training program | Experimental | Other: Training program12-week strength and endurance training program |
Who may qualify
- Ages
- 20 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Adults with DM1
- Walk without technical aid
- Muscle impairment rating scale (MIRS) of 3 or 4
Key exclusion criteria (3 listed)
- Severe musculoskeletal disorders
- Any medical contraindication to physical exercise
- Any medical contraindication to muscle biopsies for the biopsy volunteers
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 |
|---|---|---|
| Training program12-week, 18-session training program. To offer a complete training program aimed at improving function it was divided : the first 6 weeks were dedicated to strength-training (2 sets of 6 repetitions at 80% of 1-RM), whereas the following weeks focused on endurance-training (1 set of 25 repetitions at 40% of 1-RM). | Other | Training program |
Primary outcome
Change in maximal muscle strength in the knee extensors measured by quantified muscle testing
Time frame: Before and after the 12-week training program
Changes in knee extensor maximal muscle strength evaluated by quantified muscle testing with a handheld dynamometer.
12 secondary outcomes
Change in 6 minute walking test disance
Time frame: Before and after the 12-week training program
Changes in functional capacity measured by the UQAM-YMCA test
Time frame: Before and after the 12-week training program
Changes in sprint capacity measured by the Running-base anaerobic sprint test
Time frame: Before and after the 12-week training program
Changes in fatigue measured by the Fatigue severity scale
Time frame: Before and after the 12-week training program
Changes in daytime sleepiness measured by the Daytime sleepiness scale (Epworth Sleepiness Scale)
Time frame: Before and after the 12-week training program
Changes in stumbles and falls measured by the Falls efficacy scale
Time frame: Before and after the 12-week training program
Changes in quality of life measured by the 36-Item Short Form Survey (SF-36)
Time frame: Before and after the 12-week training program
Changes in life habits measured by questionnaire
Time frame: Before and after the 12-week training program
Changes in muscle biopsy-muscle fiber size
Time frame: Before and after the 12-week training program for volunteers
Changes in muscle biopsy-muscle fiber typing
Time frame: Before and after the 12-week training program for volunteers
Changes in muscle biopsy-proportion of centrally nuclated fibers
Time frame: Before and after the 12-week training program for volunteers
Changes in muscle biopsy-leucocyte infiltration
Time frame: Before and after the 12-week training program for volunteers
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2019.
- Derived from this study: PubMed 31429798Roussel MP, Morin M, Girardin M, Fortin AM, Leone M, Mathieu J, Gagnon C, Duchesne E. Training program-induced skeletal muscle adaptations in two men with myotonic dystrophy type 1. BMC Res Notes. 2019 Aug 20;12(1):526. doi: 10.1186/s13104-019-4554-z.
- Individual participant dataSharing planned: No
Related trials for Myotonic Dystrophy 1
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
RecruitingObservational study
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
DM1, Myotonic Dystrophy
Enrolling by invitationPhase 2
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
Myotonic Dystrophy Type 1 (DM1)
RecruitingPhase 3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1), DM1
RecruitingPhase 3
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
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.
Cynthia Gagnon, Ph.D. principal investigator
Université de Sherbrooke
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Training program in people with Myotonic Dystrophy 1, run by Cynthia Gagnon 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 3 inclusion and 3 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: finished, as of the sponsor's last update on 28 Jun 2019. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in maximal muscle strength in the knee extensors measured by quantified muscle testing (time frame: Before and after the 12-week training program). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04001920, which also lists 1 linked publication.
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 28 Jun 2019; AzTrials last refreshed its copy on 28 Oct 2026.