Myotonic Dystrophy Type 2 Clinical Trials

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

Status: Recruiting
Location: See location...
Intervention Type: Behavioral
Study Type: Interventional
Study Phase: Not Applicable
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)

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 18
Healthy Volunteers: f
View:

• 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.

Locations
Other Locations
Canada
Children's Hospital of Eastern Ontario
RECRUITING
Ottawa
Time Frame
Start Date: 2025-03-25
Estimated Completion Date: 2026-07
Participants
Target number of participants: 13
Treatments
Experimental: Music Intervention
All participants will receive weekly music and movement education sessions.
Sponsors
Collaborators: University of Ottawa
Leads: Hanns Lochmuller

This content was sourced from clinicaltrials.gov