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

NCT ID: NCT06809049

Last Updated: 2025-04-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-25

Study Completion Date

2025-12-31

Brief Summary

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The goal of this interventional study is to demonstrate the feasibility and tolerability of Dalcroze music 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.

Detailed Description

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Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Participants will be grouped with 5-7 other participants of similar age and ability for the music education sessions. All participants will receive the music intervention.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Music Intervention

All participants will receive weekly Dalcroze music education sessions.

Group Type EXPERIMENTAL

Dalcroze music education

Intervention Type BEHAVIORAL

Dalcroze is a type of music education which 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 Dalcroze music education to employ a holistic approach to improve the brain-heart symptoms of children with congenital DM1.

Interventions

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Dalcroze music education

Dalcroze is a type of music education which 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 Dalcroze music education to employ a holistic approach to improve the brain-heart symptoms of children with congenital DM1.

Intervention Type BEHAVIORAL

Eligibility Criteria

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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.
Minimum Eligible Age

6 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Ottawa

OTHER

Sponsor Role collaborator

Hanns Lochmuller

OTHER

Sponsor Role lead

Responsible Party

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Hanns Lochmuller

Senior Scientist

Responsibility Role SPONSOR_INVESTIGATOR

Locations

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Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status RECRUITING

Countries

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Canada

Facility Contacts

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Dr. Hanns Lochmuller

Role: primary

613-737-7600 ext. 4014

Tamara Burgess

Role: backup

613-737-7600 ext. 6651

Other Identifiers

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CFREF-2022-00007

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

BHI 23-118

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

24/143X

Identifier Type: -

Identifier Source: org_study_id

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