Myotonic Dystrophy 1NCT05400629
Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1
A study testing Training program in people with Myotonic Dystrophy 1, run by Université de Sherbrooke at 1 site in Canada.
Trial summary
20 women with myotonic dystrophy type 1 (DM1) will complete a 12-week lower-limb strength training program. The training program consist of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. Read more about this study
- Who is it for?
- Ages 30 to 65 years, women only
- 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
- 12 participants
- Where
- 1 siteCanada
- Study dates
- 1 Jan 2022to 10 Jan 2023
- 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 30 to 65 years
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- DM1 diagnosis must be confirmed by genetic analysis;
- Women sex, aged between 30 and 65 years old;
- Be able to walk without assistance;
- Consent of the neurologist must be given to participate in this study;
- Must reside in the Saguenay-Lac-St-Jean region;
- Subjects must be able to give their consent freely and voluntarily.
Who cannot join
- Patients with any other form of muscular dystrophy are excluded;
- Any contraindication for strenuous exercise or muscle biopsy.
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
20 women with myotonic dystrophy type 1 (DM1) will complete a 12-week lower-limb strength training program. The training program consist of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. Training sessions will be closely supervise and take place twice a week. It is hypothesize that the training program will induce muscular hypertrophy despite the genetic defect. The training program should also have positive effects on function. The participants will be evaluated at baseline, week 6, week 12, month 6 and month 9 to quantify the effects of the training program and if these effects will be maintained over time.
- 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 strength training program of the lower limbs consisting of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. All exercises will be perform between 6 and 8 maximal repetitions.
| Study group | Role | What they receive |
|---|---|---|
| Training program | Experimental | 12-week strength training program of the lower limbs consisting of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. All exercises will be perform between 6 and 8 maximal repetitions. Receives: Other: Training program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in maximal isometric muscle strength of the knee extensors
Changes in maximal isometric muscle strength of the knee extensors measure by quantify muscle testing using a handheld dynamometer
Measured: At baseline, week 6, week 12, month 3 and month 6
Secondary outcomes (37)
Changes in maximal isometric muscle strength of the knee flexors
Changes in maximal isometric muscle strength of the knee flexors measure by quantify muscle testing using a handheld dynamometer
Measured: At baseline and week 12
Changes in maximal isometric muscle strength of the hip flexors
Changes in maximal isometric muscle strength of the hip flexors measure by quantify muscle testing using a handheld dynamometer
Measured: At baseline and week 12
Changes in maximal isometric muscle strength of the hip extensors
Changes in maximal isometric muscle strength of the hip extensors measure by quantify muscle testing using a handheld dynamometer
Measured: At baseline and week 12
Changes in maximal isometric muscle strength of the hip abductors
Changes in maximal isometric muscle strength of the hip abductors measure by quantify muscle testing using a handheld dynamometer
Measured: At baseline and week 12
Changes in maximal isometric muscle strength of the ankle dorsiflexors
Changes in maximal isometric muscle strength of the ankle dorsiflexors measure by quantify muscle testing using a handheld dynamometer
Measured: At baseline and week 12
Changes in maximal isometric grip strength
Changes in maximal isometric muscle strength of prehension measure by quantify muscle testing using a hand grip dynamometer
Measured: At baseline and week 12
Changes in 1-repetition maximum strength of the leg extension exercise
Changes in 1-repetition maximum strength of the leg extension exercise
Measured: At baseline, week 6 and week 12
Changes in 1-repetition maximum strength of the leg press exercise
Changes in 1-repetition maximum strength of the leg press exercise
Measured: At baseline, week 6 and week 12
Changes in 1-repetition maximum strength of the hip abduction exercise
Changes in 1-repetition maximum strength of the hip abduction exercise
Measured: At baseline, week 6 and week 12
Changes in 1-repetition maximum strength of the squat exercise
Changes in 1-repetition maximum strength of the squat exercise
Measured: At baseline, week 6 and week 12
Changes in comfortable walking speed in the 10-meter walk test
Changes in comfortable walking speed in the 10-meter walk test
Measured: At baseline, week 6, week 12, month 6 and month 9
Changes in maximal walking speed in the 10-meter walk test
Changes in maximal walking speed in the 10-meter walk test
Measured: At baseline, week 6, week 12, month 6 and month 9
Changes in the number of repetitions in the 30-seconds sit-to-stand test
Changes in the number of repetitions in the 30-seconds sit-to-stand test
Measured: At baseline, week 6, week 12, month 6 and month 9
Changes in the score of the lower extremity functional scale
Changes in the score of the lower extremity functional scale (LEFS). The LEFS is an 80-point questionnaire with 20 items scored from 0 to 4. A score of 80 means no disfunction while a score of 0 means maximal disfunction.
Measured: At baseline, week 12, month 6 and month 9
Changes in the score of the myotonic dystrophy health index
Changes in the score of the myotonic dystrophy health index (MDHI). The MDHI is a 114-item questionnaire scored from 0 to 100, where 0 means no disability and 100 means maximal disability.
Measured: At baseline, week 6, week 12, month 6 and month 9
Changes in the score of the Fatigue and Daytime Sleepiness Scale
Changes in the score of the Fatigue and Daytime Sleepiness Scale (FDSS). The FDSS is a 12-item questionnaire where all questions are scored from 0 to 2. A higher score means more daytime sleepiness and fatigue.
Measured: At baseline, week 6, week 12, month 6 and month 9
Changes in the Marin apathy scale
Changes in the Marin apathy scale. The Marin apathy scale is scored by the clinician where he interviews the subject and then scores an 18-item list on a scale of 1 to 4. A high score means more apathy.
Measured: At baseline, week 12, month 6 and month 9
Changes in the Hospital Anxiety and Depression Scale
Changes in the Hospital Anxiety and Depression Scale (HADS). The HADS is a 14-item questionnaire with a scale from 0 to 3 for each item. A high score means high depression and anxiety.
Measured: At baseline, week 6, week 12 and month 6
Changes in muscle biopsy of the vastus lateralis: muscle fiber typing
Changes in muscle biopsy of the vastus lateralis: muscle fiber typing
Measured: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: muscle fiber size
Changes in muscle biopsy of the vastus lateralis: muscle fiber size (smallest diameter of the fiber)
Measured: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: muscle proteomics
Changes in muscle biopsy of the vastus lateralis: muscle proteomics (protein expression)
Measured: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: muscle transcriptomics
Changes in muscle biopsy of the vastus lateralis: muscle transcriptomics (RNA expression analysis)
Measured: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: mitochondrial function
Changes in muscle biopsy of the vastus lateralis: mitochondrial function. Analysis of mitochondrial markers and enzymes
Measured: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: Nuclear foci
Changes in muscle biopsy of the vastus lateralis: Nuclear foci (changes in accumulation of nuclear foci)
Measured: At baseline and week 12
Changes in the time to perform of the Timed Up and Go
Changes in the time to perform of the Timed Up and Go
Measured: At baseline, week 6, week 12, month 6 and month 9
Changes in the time to perform the Timed stair test
Changes in the time to perform the Timed stair test
Measured: At baseline, week 6, week 12, month 6 and month 9
Change in the score of the Brief Pain Inventory
Change in the score of the Brief Pain Inventory. The Brief Pain Inventory is a self-administer test that asses the dimensions of pain such as intensity of pain, functional incapacities, social and family repercussions, and psychological distress.
Measured: At baseline, week 6, week 12, month 6 and month 9
Self-perceived changes in daily life
Self-perceived changes in daily life
Measured: At week 12
Change in the number of falls
Number of falls in the last week and in the last month
Measured: At baseline, week 12, month 6 ans month 9
Change in score of the DM1-Activ questionnaire
Change in score of the DM1-Activ questionnaire. This questionnaire asses activity and social participation with DM1 population. It contains 20 items with a maximal socre of 40 points, a lower score represent a bigger impact of the disease on activities and social participation.
Measured: At baseline, week 6, week 12, month 6 ans month 9
Change in blood : biochemical markers (CK, Protein C reactive, leukocytes, cytokines, etc.)
Change in blood : biochemical markers (CK, Protein C reactive, leukocytes, cytokines, etc.)
Measured: At baseline and week 12
Change in urine sample : exercise biomarkers (-Omics markers)
Change in urine sample : exercise biomarkers (-Omics markers)
Measured: At baseline and week 12
Change in saliva sample : exercise biomarkers (-Omics markers)
Change in saliva sample : exercise biomarkers (-Omics markers)
Measured: At baseline and week 12
Change of the center of pressure (COP) measure in static posturography : open eyes bipodal position
Change of the center of pressure (COP) in static posturography : open eyes bipodal position
Measured: At baseline, week 6, week 12, 6 months and 9 months
Change of the center of pressure (COP) measure in static posturography : closed eyes bipodal position
Change of the center of pressure (COP) in static posturography : closed eyes bipodal position
Measured: At baseline, week 6, week 12, 6 months and 9 months
Change of the center of pressure (COP) measure in static posturography : open eyes semi-tandem position
Change of the center of pressure (COP) in static posturography : open eyes semi-tandem position
Measured: At baseline, week 6, week 12, 6 months and 9 months
Change of the center of pressure (COP) measure in static posturography : open eyes tandem position
Change of the center of pressure (COP) in static posturography : open eyes tandem position
Measured: At baseline, week 6, week 12, 6 months and 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2022
- Study start1 Jan 2022
- Primary completion30 Jun 2022
- Study completion10 Jan 2023
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.
- More trials run by Université de SherbrookeThe sponsor's other registered studies.
- 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
- Sequential
- Model description
- Participants will complete a 12-week strength training program and return to their normal activities for the following 6 months after the end of the training program.
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Université de Sherbrooke
- Sponsor type
- University, hospital or other
- Collaborators
- Rare Disease Foundation
- Responsible party
- Principal investigator, Élise Duchesne, Université de Sherbrooke
- Investigators
- Elise Duchesne, Ph.D, Principal investigator, Université du Québec à Chicoutimi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05400629
- Sponsor's protocol ID
- 2022-005
- Status last verified
- Feb 2023
- First submitted
- 24 May 2022
- Last updated
- 10 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT05400629
- Acronym / sponsor ID
- 2022-005
- Conditions
- Myotonic Dystrophy 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Université de SherbrookeUniversity, hospital or other
- Collaborators
- Rare Disease Foundation
- Study start
- 1 Jan 2022 (actual)
- Primary completion
- 30 Jun 2022 (actual)
- Study completion
- 10 Jan 2023 (actual)
- First posted
- 1 Jun 2022 (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.
20 women with myotonic dystrophy type 1 (DM1) will complete a 12-week lower-limb strength training program. The training program consist of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. Training sessions will be closely supervise and take place twice a week. It is hypothesize that the training program will induce muscular hypertrophy despite the genetic defect. The training program should also have positive effects on function. The participants will be evaluated at baseline, week 6, week 12, month 6 and month 9 to quantify the effects of the training program and if these effects will be maintained over time.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Participants will complete a 12-week strength training program and return to their normal activities for the following 6 months after the end of the training program.
| Arm | Type | Receives |
|---|---|---|
| Training program | Experimental | Other: Training program12-week strength training program of the lower limbs consisting of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. All exercises will be perform between 6 and 8 maximal repetitions. |
Who may qualify
- Ages
- 30 to 65 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- DM1 diagnosis must be confirmed by genetic analysis;
- Women sex, aged between 30 and 65 years old;
- Be able to walk without assistance;
- Consent of the neurologist must be given to participate in this study;
- Must reside in the Saguenay-Lac-St-Jean region;
Key exclusion criteria (2 listed)
- Patients with any other form of muscular dystrophy are excluded;
- Any contraindication for strenuous exercise or muscle biopsy.
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 strength training program of the lower limbs consisting of 5 different exercises: Leg extension, leg press, hip abduction, squat and plantar flexion. All exercises will be perform between 6 and 8 maximal repetitions. | Other | Training program |
Primary outcome
Changes in maximal isometric muscle strength of the knee extensors
Time frame: At baseline, week 6, week 12, month 3 and month 6
Changes in maximal isometric muscle strength of the knee extensors measure by quantify muscle testing using a handheld dynamometer
37 secondary outcomes
Changes in maximal isometric muscle strength of the knee flexors
Time frame: At baseline and week 12
Changes in maximal isometric muscle strength of the hip flexors
Time frame: At baseline and week 12
Changes in maximal isometric muscle strength of the hip extensors
Time frame: At baseline and week 12
Changes in maximal isometric muscle strength of the hip abductors
Time frame: At baseline and week 12
Changes in maximal isometric muscle strength of the ankle dorsiflexors
Time frame: At baseline and week 12
Changes in maximal isometric grip strength
Time frame: At baseline and week 12
Changes in 1-repetition maximum strength of the leg extension exercise
Time frame: At baseline, week 6 and week 12
Changes in 1-repetition maximum strength of the leg press exercise
Time frame: At baseline, week 6 and week 12
Changes in 1-repetition maximum strength of the hip abduction exercise
Time frame: At baseline, week 6 and week 12
Changes in 1-repetition maximum strength of the squat exercise
Time frame: At baseline, week 6 and week 12
Changes in comfortable walking speed in the 10-meter walk test
Time frame: At baseline, week 6, week 12, month 6 and month 9
Changes in maximal walking speed in the 10-meter walk test
Time frame: At baseline, week 6, week 12, month 6 and month 9
Changes in the number of repetitions in the 30-seconds sit-to-stand test
Time frame: At baseline, week 6, week 12, month 6 and month 9
Changes in the score of the lower extremity functional scale
Time frame: At baseline, week 12, month 6 and month 9
Changes in the score of the myotonic dystrophy health index
Time frame: At baseline, week 6, week 12, month 6 and month 9
Changes in the score of the Fatigue and Daytime Sleepiness Scale
Time frame: At baseline, week 6, week 12, month 6 and month 9
Changes in the Marin apathy scale
Time frame: At baseline, week 12, month 6 and month 9
Changes in the Hospital Anxiety and Depression Scale
Time frame: At baseline, week 6, week 12 and month 6
Changes in muscle biopsy of the vastus lateralis: muscle fiber typing
Time frame: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: muscle fiber size
Time frame: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: muscle proteomics
Time frame: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: muscle transcriptomics
Time frame: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: mitochondrial function
Time frame: At baseline and week 12
Changes in muscle biopsy of the vastus lateralis: Nuclear foci
Time frame: At baseline and week 12
Changes in the time to perform of the Timed Up and Go
Time frame: At baseline, week 6, week 12, month 6 and month 9
Changes in the time to perform the Timed stair test
Time frame: At baseline, week 6, week 12, month 6 and month 9
Change in the score of the Brief Pain Inventory
Time frame: At baseline, week 6, week 12, month 6 and month 9
Self-perceived changes in daily life
Time frame: At week 12
Change in the number of falls
Time frame: At baseline, week 12, month 6 ans month 9
Change in score of the DM1-Activ questionnaire
Time frame: At baseline, week 6, week 12, month 6 ans month 9
Change in blood : biochemical markers (CK, Protein C reactive, leukocytes, cytokines, etc.)
Time frame: At baseline and week 12
Change in urine sample : exercise biomarkers (-Omics markers)
Time frame: At baseline and week 12
Change in saliva sample : exercise biomarkers (-Omics markers)
Time frame: At baseline and week 12
Change of the center of pressure (COP) measure in static posturography : open eyes bipodal position
Time frame: At baseline, week 6, week 12, 6 months and 9 months
Change of the center of pressure (COP) measure in static posturography : closed eyes bipodal position
Time frame: At baseline, week 6, week 12, 6 months and 9 months
Change of the center of pressure (COP) measure in static posturography : open eyes semi-tandem position
Time frame: At baseline, week 6, week 12, 6 months and 9 months
Change of the center of pressure (COP) measure in static posturography : open eyes tandem position
Time frame: At baseline, week 6, week 12, 6 months and 9 months
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 10 Feb 2023.
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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.
Elise Duchesne, Ph.D principal investigator
Université du Québec à Chicoutimi
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 Université de Sherbrooke 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 2 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 10 Feb 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. 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?
Changes in maximal isometric muscle strength of the knee extensors (time frame: At baseline, week 6, week 12, month 3 and month 6). 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 10 Feb 2023.