NCT06809049

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

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 13 (estimated)
• Sponsor: Hanns Lochmuller · Academic / Other
• Sex: All
• Age: 6 Years – 18 Years
• Healthy volunteers: Not accepted

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. 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

• Myotonic Dystrophy, Congenital
• Myotonic Dystrophy, Type 1 (DM1)
• Myotonic Dystrophy Type 1
• Myotonic Dystrophy Type 1 (DM1)

Interventions

Type	Name	Description
BEHAVIORAL	Music and movement education	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.

Timeline

• Start date: 2025-03-25
• Primary completion: 2026-07-01
• Completion: 2026-07-01
• First posted: 2025-02-05
• Last updated: 2026-02-19

Locations

1 site across 1 country: Canada