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

Hanns Lochmuller

Status

Enrolling

Conditions

Myotonic Dystrophy Type 1

Myotonic Dystrophy Type 1 (DM1)

Myotonic Dystrophy, Type 1 (DM1)

Myotonic Dystrophy, Congenital

Treatments

Behavioral: Dalcroze music education

Study type

Interventional

Funder types

Other

Identifiers

NCT06809049

BHI 23-118 (Other Grant/Funding Number)

CFREF-2022-00007 (Other Grant/Funding Number)

24/143X

Details and patient eligibility

About

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.

Enrollment

16 estimated patients

Sex

All

Ages

6 to 18 years old

Volunteers

No Healthy Volunteers

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.