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")
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.
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.
Single group
Music and movement education
BehavioralThe 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.
Attendance to Each Music Session
Participant attendance rate to 10 music sessions.
Attendance to Block of Music Sessions
The proportion of participants who attend all 10 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.
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.
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).
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.
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.
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Hanns Lochmuller
Lead sponsor
Children's Hospital of Eastern Ontario
Sponsor institution
University of Ottawa
Collaborator
