Actively Recruiting
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
Led by Hanns Lochmuller · Updated on 2026-02-19
13
Participants Needed
1
Research Sites
66 weeks
Total Duration
On this page
Sponsors
H
Hanns Lochmuller
Lead Sponsor
U
University of Ottawa
Collaborating Sponsor
AI-Summary
What this Trial Is About
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")
CONDITIONS
Official Title
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 6 to 18 years with genetically confirmed congenital or infantile-onset myotonic dystrophy type 1 (DM1)
- Willingness to remain on stable medication from screening through the end of the study
You will not qualify if you...
- Insufficient English skills to complete assessments and questionnaires
- Non-verbal participant
- Lack of signed informed consent by participant or legal representative
- Lack of signed informed consent by parents/caregivers for participation
- Participant not residing in Canada
- Investigator's judgment that participation would be unsafe
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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