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NCT Number: NCT06809049

Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)

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:

1. The feasibility of attendance, as assessed by attendance rate to 10 music sessions (≥ 60%) 2. Feasibility of attendance, as rated by parents/caregivers of participants (≥60% rate "extremely" or "very" practical to attend) 3. Attrition rate of the study, as determined by percentage of participants who complete the study (≥ 60%) 4. Overall satisfaction, as rated by parents/caregivers of participants (≥60% rate "very satisfied" or "satisfied")

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Myotonic Dystrophy, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Myotonic Dystrophy Type 1 Myotonic Dystrophy Type 1 (DM1) Myotonic Dystrophy, Type 1 (DM1) Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

6 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Music and movement education

Behavioral

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.

Primary outcomes

  1. Attendance to Each Music Session

    Time frame: Over 10-week block of music sessions

    Participant attendance rate to 10 music sessions.

  2. 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.

  3. 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.

Secondary outcomes

  1. Change in Physical Performance Assessed by the 6-Minute Walk Test (6MWT)

    Time frame: Baseline and 1 week following the final music session.

    The 6-minute walk test involves the participant walking unaccompanied along a 30m hallway as far as possible within a 6-minute period.

  2. 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.

    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).

  3. 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.

    The WASI-II is a standardized measure of overall intelligence, with a higher score corresponding to higher intellectual ability.

  4. Change in Attention Assessed by Bells Assessment

    Time frame: Baseline and 1 week following the final music session.

    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.

  5. Change in Working Memory Assessed by NIH List Sorting Working Memory Test

    Time frame: Baseline and 1 week following the final music session.

  6. Change in Executive Functioning Assessed by Dimensional Change Card Sort

    Time frame: Baseline and 1 week following the final music session.

    The participant is asked to match a series of picture pairs to a target picture.

  7. Change in Visuospatial Skills Assessed by Rex Complex Figure - Copy

    Time frame: Baseline and 1 week following the final music session.

  8. Change in Episodic Memory Assessed by NIH Picture Sequence Memory Test

    Time frame: Baseline and 1 week following the final music session.

  9. Change in Receptive Vocabulary Assessed by Peabody Picture Vocabulary Test (PPVT-5)

    Time frame: Baseline and 1 week following the final music session.

    A measure of receptive vocabulary with scores ranging from 0 (lower receptive vocabulary skills) to 175 (higher receptive vocabulary skills).

  10. Change in Mood and Anxiety Assessed by Mood and Feelings Questionnaire (MFQ)

    Time frame: Baseline and 1 week following the final music session.

    Participants self-report the applicability of phrases as true, sometimes true, or not true.

  11. Change in Adaptive Functioning Assessed by Vineland Adaptive Behaviour Scale

    Time frame: Baseline and 1 week following the final music session.

  12. Change in Quality of Life Assessed by Pediatric Quality of Life Inventory (PedsQL)

    Time frame: Baseline and 1 week following the final music session.

    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.

  13. Change in Sleep Quality Assessed by Children's Sleep Habits Questionnaire

    Time frame: Baseline and 1 week following the final music session.

    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.

  14. Assessment of Cardiac Conduction Defects by Electrocardiogram (ECG)

    Time frame: Baseline and 1 week following the final music session.

    Participants will receive clinical ECG testing to assess for the presence of cardiac conduction defects.

  15. Changes in Attention Assessed by Conners Continuous Performance Test Third Edition (CPT3)

    Time frame: Baseline and 1 week following the final music session.

    Participants are instructed to press the spacebar of a computer each time the letter X appears. A higher score indicates worse performance.

  16. Change in Executive Functioning Assessed by Behaviour Rating Inventory of Executive Functioning (BRIEF2)

    Time frame: Baseline and 1 week following the final music session.

    The score ranges from 80-240, with a higher score indicating greater executive dysfunction.

  17. 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.

    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.

  18. Change in Physical Activity Assessed by Accelerometer

    Time frame: Baseline and 1 week following the final music session.

    Actigraph wGT3X-BT accelerometer will be worn for continuous tracking of activity for two 7-day periods.

  19. Change in Affect Assessed by Positive and Negative Affect Scale - Child (PANAS-C)

    Time frame: Baseline and 1 week following the final music session.

    Participants self-report the frequency each feeling or emotion was experienced on a 5-point Likert scale.

  20. Change in Anxiety Assessed by State-Trait Anxiety Inventory Scale-5 (STAIS-5)

    Time frame: Baseline and 1 week following the final music session

  21. 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.

    Participants will wear a Muse-EEG headband overnight. Measures of sleep architecture will inform overall sleep quality.

  22. Assessment of Heart Rate Variability by Wearable Electrocardiogram (ECG) Chest Strap

    Time frame: Over 7-day period at baseline and following final music intervention.

    Participants will wear a Polar H10 ECG chest strap for at-home monitoring of heart rate.

Sponsors and collaborators

Lead sponsor

Hanns Lochmuller

Other

Collaborators

  • University of Ottawa

Registry information

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Feb 5, 2025
Registry last updated
Jul 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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