Myotonic Dystrophy, CongenitalNCT06809049
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
A study testing Music and movement education in people with Myotonic Dystrophy, Congenital and Myotonic Dystrophy, Type 1 (DM1), run by Hanns Lochmuller at 1 site in Canada.
Trial summary
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and heart symptoms of DM1. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1
- What is being tested?
- Music and movement education
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteCanada
- Study dates
- 25 Mar 2025to Oct 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants between the ages of 6 to 18 with genetically confirmed congenital or infantile-onset myotonic dystrophy type 1 (DM1).
- Participants willing to stay on stable medication from the day of screening to end of study.
Who cannot join
- Insufficient English language skills to complete required assessments and questionnaires.
- Participant is non-verbal.
- Failure to provide signed informed consent by participant or designated decision maker (i.e. parent, legal guardian or power of attorney).
- Failure to provide signed informed consent by parents/caregivers for dyad participation.
- Participant is not a resident of Canada, due to risk of attrition.
- Patients for whom - in the opinion of the investigator - it would not be safe to participate in the study.
Trial locations and contacts
1 site in 1 country.
About this study
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and heart symptoms of DM1. Additionally, information from the collection of biological samples and wearable devices (accelerometer, EEG headband and ECG chest strap) will be used to identify brain-heart biomarkers and outcome measures for use in future research and trials.
Researchers will compare the results of physical and cognitive assessments for each participant to assessments from baseline after 10 weeks of weekly music sessions. Qualitative measures (questionnaires and focus groups) will inform the feasibility of this intervention for this population. The main questions this study aims to answer are:
Read more
- Are weekly music education sessions feasible for children with DM1?
- Are weekly music education sessions tolerable for children with DM1?
Participants will:
- Attend 45-minute-long music sessions once weekly for 10 weeks.
- Attend two clinic visits for cognitive and physical assessments.
- Provide blood, saliva, stool and urine samples.
- Use wearable devices both at-home and during music sessions.
- Parents/caregivers of participants will complete questionnaires and participate in three focus groups.
Progression from feasibility study to a full-scale clinical trial will be informed by four progression criteria:
- The feasibility of attendance, as assessed by attendance rate to 10 music sessions (≥ 60%)
- Feasibility of attendance, as rated by parents/caregivers of participants (≥60% rate "extremely" or "very" practical to attend)
- Attrition rate of the study, as determined by percentage of participants who complete the study (≥ 60%)
- Overall satisfaction, as rated by parents/caregivers of participants (≥60% rate "very satisfied" or "satisfied")
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Being tested
- Music and movement education
- Keywords
- Music intervention, Myotonic dystrophy, DM1, Rare disease
- Medical subject headings
- Myotonic Dystrophy, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Music and movement educationBehavioural
The music and movement intervention is partially inspired by an adaptive music-education method called Dalcroze eurhythmics. It focuses on free and improvised movement, as well as structure and choreographed sequences. It has been successfully applied to similar populations as an effective, accessible, and sustainable intervention. We hope to use this music and movement education to employ a holistic approach to improve the brain-heart symptoms of children with congenital DM1.
| Study group | Role | What they receive |
|---|---|---|
| Music Intervention | Experimental | All participants will receive weekly music and movement education sessions. Receives: Behavioral: Music and movement education |
What is being measured
The primary outcome is the main question the study is designed to answer.
Attendance to Each Music Session
Participant attendance rate to 10 music sessions.
Measured: Over 10-week block of music sessions
Attendance to Block of Music Sessions
The proportion of participants who attend all 10 music sessions.
Measured: Over 10-week block of music sessions
Qualitative Feedback from Parents/Caregivers Following Each Music Session
Parents/Caregivers will complete short questionnaires following each of the 10 music sessions. Questions will focus on recall of participant mood, tiredness and engagement in the 24 hours immediately following each music session.
Measured: Following each music session through to the final music session.
Secondary outcomes (22)
Change in Physical Performance Assessed by the 6-Minute Walk Test (6MWT)
The 6-minute walk test involves the participant walking unaccompanied along a 30m hallway as far as possible within a 6-minute period.
Measured: Baseline and 1 week following the final music session.
Change from Baseline in Clinical Phenotype Assessed by the Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
The clinician rates the severity of 11 key symptoms over the past week on a 5-point Likert scale from 0 (least severe) to 4 (most severe).
Measured: Baseline and 1 week following the final music session.
Change in Global IQ Assessed by Wechsler Abbreviated Scale of Intelligence Second Edition (WASI-II)
The WASI-II is a standardized measure of overall intelligence, with a higher score corresponding to higher intellectual ability.
Measured: Baseline and 1 week following the final music session.
Change in Attention Assessed by Bells Assessment
In the Bells assessment, the participant is asked to circle all the bells on a page with other images as distractors. The maximum score is 35/35 bells.
Measured: Baseline and 1 week following the final music session.
Change in Working Memory Assessed by NIH List Sorting Working Memory Test
Measured: Baseline and 1 week following the final music session.
Change in Executive Functioning Assessed by Dimensional Change Card Sort
The participant is asked to match a series of picture pairs to a target picture.
Measured: Baseline and 1 week following the final music session.
Change in Visuospatial Skills Assessed by Rex Complex Figure - Copy
Measured: Baseline and 1 week following the final music session.
Change in Episodic Memory Assessed by NIH Picture Sequence Memory Test
Measured: Baseline and 1 week following the final music session.
Change in Receptive Vocabulary Assessed by Peabody Picture Vocabulary Test (PPVT-5)
A measure of receptive vocabulary with scores ranging from 0 (lower receptive vocabulary skills) to 175 (higher receptive vocabulary skills).
Measured: Baseline and 1 week following the final music session.
Change in Mood and Anxiety Assessed by Mood and Feelings Questionnaire (MFQ)
Participants self-report the applicability of phrases as true, sometimes true, or not true.
Measured: Baseline and 1 week following the final music session.
Change in Adaptive Functioning Assessed by Vineland Adaptive Behaviour Scale
Measured: Baseline and 1 week following the final music session.
Change in Quality of Life Assessed by Pediatric Quality of Life Inventory (PedsQL)
Participant and their parents report on the frequency (never to almost always) of specific health-related quality of life problems. A higher score indicates poorer quality of life.
Measured: Baseline and 1 week following the final music session.
Change in Sleep Quality Assessed by Children's Sleep Habits Questionnaire
Parent completed questionnaire to assess participant sleep behaviours. Each question is rate on a 3-point scale. Total scores can range from 33-99, with a score higher than 41 suggesting the presence of a childhood sleep disorder.
Measured: Baseline and 1 week following the final music session.
Assessment of Cardiac Conduction Defects by Electrocardiogram (ECG)
Participants will receive clinical ECG testing to assess for the presence of cardiac conduction defects.
Measured: Baseline and 1 week following the final music session.
Changes in Attention Assessed by Conners Continuous Performance Test Third Edition (CPT3)
Participants are instructed to press the spacebar of a computer each time the letter X appears. A higher score indicates worse performance.
Measured: Baseline and 1 week following the final music session.
Change in Executive Functioning Assessed by Behaviour Rating Inventory of Executive Functioning (BRIEF2)
The score ranges from 80-240, with a higher score indicating greater executive dysfunction.
Measured: Baseline and 1 week following the final music session.
Change in Physical Performance Assessed by the 10-meter walk/run test (10-mW/RT)
For the 10-meter walk/run test, the participant walks/runs without assistance for 12 meters while the time taken to walk from meter 1 to meter 11 is recorded with a stopwatch. The test will be repeated 2-3 times, with a minimum of 10-15 seconds of rest between trials to avoid fatigue.
Measured: Baseline and 1 week following the final music session.
Change in Physical Activity Assessed by Accelerometer
Actigraph wGT3X-BT accelerometer will be worn for continuous tracking of activity for two 7-day periods.
Measured: Baseline and 1 week following the final music session.
Change in Affect Assessed by Positive and Negative Affect Scale - Child (PANAS-C)
Participants self-report the frequency each feeling or emotion was experienced on a 5-point Likert scale.
Measured: Baseline and 1 week following the final music session.
Change in Anxiety Assessed by State-Trait Anxiety Inventory Scale-5 (STAIS-5)
Measured: Baseline and 1 week following the final music session
Change in Sleep Quality Assessed by Electroencephalogram (EEG) Headband
Participants will wear a Muse-EEG headband overnight. Measures of sleep architecture will inform overall sleep quality.
Measured: Nightly for 7 days at baseline and 1 week following the final music session.
Assessment of Heart Rate Variability by Wearable Electrocardiogram (ECG) Chest Strap
Participants will wear a Polar H10 ECG chest strap for at-home monitoring of heart rate.
Measured: Over 7-day period at baseline and following final music intervention.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2025
- Study start25 Mar 2025
- Primary completionOct 2026 (estimated)
- Study completionOct 2026 (estimated)
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, CongenitalStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Participants will be grouped with 3-7 other participants of similar age and ability for the music education sessions. All participants will receive the music intervention.
- Masking
- None (open label)
- Enrolment
- 13 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hanns Lochmuller
- Sponsor type
- University, hospital or other
- Collaborators
- University of Ottawa
- Responsible party
- Sponsor-investigator, Hanns Lochmuller, Children's Hospital of Eastern Ontario
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06809049
- Sponsor's protocol ID
- 24/143X
- Other IDs
- CFREF-2022-00007, BHI 23-118
- Status last verified
- Jul 2026
- First submitted
- 27 Jan 2025
- Last updated
- 28 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified quantitative data including, results of cognitive and physical assessments and wearable device measurements.
Publications
Eisenkolbl A, Burgess T, Howley H, Parkes E, Comeau G, Berard JA, Walker LAS, Woollam A, Yaworski A, McMillan H, Lochmuller H. Music and movement intervention for brain-heart disease in children with myotonic dystrophy type 1 (DM1): a convergent mixed-methods feasibility study protocol. Pilot Feasibility Stud. 2026 Aug 5;12(1):156. doi: 10.1186/s40814-026-01894-8.
PubMed 42972444
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
- ClinicalTrials.gov ID
- NCT06809049
- Acronym / sponsor ID
- 24/143X
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Keywords
- Music intervention, Myotonic dystrophy, DM1, Rare disease
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (estimated)
- Lead sponsor
- Hanns LochmullerUniversity, hospital or other
- Collaborators
- University of Ottawa
- Study start
- 25 Mar 2025 (actual)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 5 Feb 2025 (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.
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and heart symptoms of DM1. Additionally, information from the collection of biological samples and wearable devices (accelerometer, EEG headband and ECG chest strap) will be used to identify brain-heart biomarkers and outcome measures for use in future research and trials.
Researchers will compare the results of physical and cognitive assessments for each participant to assessments from baseline after 10 weeks of weekly music sessions. Qualitative measures (questionnaires and focus groups) will inform the feasibility of this intervention for this population. The main questions this study aims to answer are:
- Are weekly music education sessions feasible for children with DM1?
- Are weekly music education sessions tolerable for children with DM1?
Participants will:
- Attend 45-minute-long music sessions once weekly for 10 weeks.
- Attend two clinic visits for cognitive and physical assessments.
- Provide blood, saliva, stool and urine samples.
- Use wearable devices both at-home and during music sessions.
- Parents/caregivers of participants will complete questionnaires and participate in three focus groups.
Progression from feasibility study to a full-scale clinical trial will be informed by four progression criteria:
- The feasibility of attendance, as assessed by attendance rate to 10 music sessions (≥ 60%)
- Feasibility of attendance, as rated by parents/caregivers of participants (≥60% rate "extremely" or "very" practical to attend)
- Attrition rate of the study, as determined by percentage of participants who complete the study (≥ 60%)
- Overall satisfaction, as rated by parents/caregivers of participants (≥60% rate "very satisfied" or "satisfied")
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Participants will be grouped with 3-7 other participants of similar age and ability for the music education sessions. All participants will receive the music intervention.
| Arm | Type | Receives |
|---|---|---|
| Music Intervention | Experimental | Behavioral: Music and movement educationAll participants will receive weekly music and movement education sessions. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Participants between the ages of 6 to 18 with genetically confirmed congenital or infantile-onset myotonic dystrophy type 1 (DM1).
- Participants willing to stay on stable medication from the day of screening to end of study.
Key exclusion criteria (6 listed)
- Insufficient English language skills to complete required assessments and questionnaires.
- Participant is non-verbal.
- Failure to provide signed informed consent by participant or designated decision maker (i.e. parent, legal guardian or power of attorney).
- Failure to provide signed informed consent by parents/caregivers for dyad participation.
- Participant is not a resident of Canada, due to risk of attrition.
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 |
|---|---|---|
| Music and movement educationThe music and movement intervention is partially inspired by an adaptive music-education method called Dalcroze eurhythmics. It focuses on free and improvised movement, as well as structure and choreographed sequences. It has been successfully applied to similar populations as an effective, accessible, and sustainable intervention. We hope to use this music and movement education to employ a holistic approach to improve the brain-heart symptoms of children with congenital DM1. | Behavioural | Music Intervention |
Primary outcome
Attendance to Each Music Session
Time frame: Over 10-week block of music sessions
Participant attendance rate to 10 music sessions.
Attendance to Block of Music Sessions
Time frame: Over 10-week block of music sessions
The proportion of participants who attend all 10 music sessions.
Qualitative Feedback from Parents/Caregivers Following Each Music Session
Time frame: Following each music session through to the final music session.
Parents/Caregivers will complete short questionnaires following each of the 10 music sessions. Questions will focus on recall of participant mood, tiredness and engagement in the 24 hours immediately following each music session.
22 secondary outcomes
Change in Physical Performance Assessed by the 6-Minute Walk Test (6MWT)
Time frame: Baseline and 1 week following the final music session.
Change from Baseline in Clinical Phenotype Assessed by the Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Time frame: Baseline and 1 week following the final music session.
Change in Global IQ Assessed by Wechsler Abbreviated Scale of Intelligence Second Edition (WASI-II)
Time frame: Baseline and 1 week following the final music session.
Change in Attention Assessed by Bells Assessment
Time frame: Baseline and 1 week following the final music session.
Change in Working Memory Assessed by NIH List Sorting Working Memory Test
Time frame: Baseline and 1 week following the final music session.
Change in Executive Functioning Assessed by Dimensional Change Card Sort
Time frame: Baseline and 1 week following the final music session.
Change in Visuospatial Skills Assessed by Rex Complex Figure - Copy
Time frame: Baseline and 1 week following the final music session.
Change in Episodic Memory Assessed by NIH Picture Sequence Memory Test
Time frame: Baseline and 1 week following the final music session.
Change in Receptive Vocabulary Assessed by Peabody Picture Vocabulary Test (PPVT-5)
Time frame: Baseline and 1 week following the final music session.
Change in Mood and Anxiety Assessed by Mood and Feelings Questionnaire (MFQ)
Time frame: Baseline and 1 week following the final music session.
Change in Adaptive Functioning Assessed by Vineland Adaptive Behaviour Scale
Time frame: Baseline and 1 week following the final music session.
Change in Quality of Life Assessed by Pediatric Quality of Life Inventory (PedsQL)
Time frame: Baseline and 1 week following the final music session.
Change in Sleep Quality Assessed by Children's Sleep Habits Questionnaire
Time frame: Baseline and 1 week following the final music session.
Assessment of Cardiac Conduction Defects by Electrocardiogram (ECG)
Time frame: Baseline and 1 week following the final music session.
Changes in Attention Assessed by Conners Continuous Performance Test Third Edition (CPT3)
Time frame: Baseline and 1 week following the final music session.
Change in Executive Functioning Assessed by Behaviour Rating Inventory of Executive Functioning (BRIEF2)
Time frame: Baseline and 1 week following the final music session.
Change in Physical Performance Assessed by the 10-meter walk/run test (10-mW/RT)
Time frame: Baseline and 1 week following the final music session.
Change in Physical Activity Assessed by Accelerometer
Time frame: Baseline and 1 week following the final music session.
Change in Affect Assessed by Positive and Negative Affect Scale - Child (PANAS-C)
Time frame: Baseline and 1 week following the final music session.
Change in Anxiety Assessed by State-Trait Anxiety Inventory Scale-5 (STAIS-5)
Time frame: Baseline and 1 week following the final music session
Change in Sleep Quality Assessed by Electroencephalogram (EEG) Headband
Time frame: Nightly for 7 days at baseline and 1 week following the final music session.
Assessment of Heart Rate Variability by Wearable Electrocardiogram (ECG) Chest Strap
Time frame: Over 7-day period at baseline and following final music intervention.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2026.
- Derived from this study: PubMed 42972444Eisenkolbl A, Burgess T, Howley H, Parkes E, Comeau G, Berard JA, Walker LAS, Woollam A, Yaworski A, McMillan H, Lochmuller H. Music and movement intervention for brain-heart disease in children with myotonic dystrophy type 1 (DM1): a convergent mixed-methods feasibility study protocol. Pilot Feasibility Stud. 2026 Aug 5;12(1):156. doi: 10.1186/s40814-026-01894-8.
- Individual participant dataSharing planned: Yes. De-identified quantitative data including, results of cognitive and physical assessments and wearable device measurements.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Music and movement education in people with Myotonic Dystrophy, Congenital and Myotonic Dystrophy, Type 1 (DM1), run by Hanns Lochmuller 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 2 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 28 Jul 2026. 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?
This study is still under way. Results are not available in the source record yet.
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?
Attendance to Each Music Session (time frame: Over 10-week block of music sessions). The study lists 3 primary outcomes in all. 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 NCT06809049, 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 Jul 2026.