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

Launched by HANNS LOCHMULLER · Jan 30, 2025

Keywords

ClinConnect Summary

This clinical trial, called the "Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1," is exploring whether music education can help children with a condition called myotonic dystrophy type 1 (DM1), which affects their muscles and can impact their brain and heart. The study aims to see if weekly music sessions are easy for these children to attend and whether they can handle the sessions well. Over 10 weeks, participants will join 45-minute music sessions once a week, undergo physical and mental assessments, and provide biological samples to help researchers understand more about DM1.

To be eligible, children aged 6 to 17 with a confirmed diagnosis of congenital or infantile-onset DM1 can participate, as long as they maintain stable medication during the study. Participants will also wear devices that track their movements and heart activity, and caregivers will be asked to share their thoughts through questionnaires and focus groups. This trial is currently recruiting participants, and the findings could help shape future research and treatments for children with DM1.

Gender

ALL

Eligibility criteria

• 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.