Actively Recruiting

Phase Not Applicable
Age: 6Years - 18Years
All Genders
NCT06809049

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

Age: 6Years - 18Years
All Genders

Eligibility Criteria

Eligible

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
Not Eligible

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

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Trial Site Locations

Total: 1 location

1

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada, K1H 8L1

Actively Recruiting

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