Clinical trial NCT06809049: Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
Official title: Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Myotonic Dystrophy, Congenital
Myotonic Dystrophy, Type 1 (DM1)
Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1)
Keywords: Music intervention
Myotonic dystrophy
DM1
Rare disease
Dalcroze music education
Lead sponsor: Children's Hospital of Eastern Ontario
Sponsor class: OTHER
Collaborators: name: University of Ottawa
First posted: 2025-02-05
Enrollment: 16
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: 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 purpose: TREATMENT

Brief summary: 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.


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.

Primary outcomes:
measure: Attendance to Each Music Session
timeFrame: Over 10-week block of music sessions
measure: Attendance to Block of Music Sessions
timeFrame: Over 10-week block of music sessions
measure: Qualitative Feedback from Parents/Caregivers Following Each Music Session
timeFrame: Following each music session through to the final music session.
Secondary outcomes:
measure: Change in Physical Performance Assessed by the 6-Minute Walk Test (6MWT)
timeFrame: Baseline and 1 week following the final music session.
measure: Change from Baseline in Clinical Phenotype Assessed by the Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Global IQ Assessed by Wechsler Abbreviated Scale of Intelligence Second Edition (WASI-II)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Attention Assessed by Bells Assessment
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Working Memory Assessed by NIH List Sorting Working Memory Test
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Executive Functioning Assessed by Dimensional Change Card Sort
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Visuospatial Skills Assessed by Rex Complex Figure - Copy
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Episodic Memory Assessed by NIH Picture Sequence Memory Test
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Receptive Vocabulary Assessed by Peabody Picture Vocabulary Test (PPVT-5)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Mood and Anxiety Assessed by Mood and Feelings Questionnaire (MFQ)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Adaptive Functioning Assessed by Vineland Adaptive Behaviour Scale
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Quality of Life Assessed by Pediatric Quality of Life Inventory (PedsQL)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Sleep Quality Assessed by Children's Sleep Habits Questionnaire
timeFrame: Baseline and 1 week following the final music session.
measure: Assessment of Cardiac Conduction Defects by Electrocardiogram (ECG)
timeFrame: Baseline and 1 week following the final music session.
measure: Changes in Attention Assessed by Conners Continuous Performance Test Third Edition (CPT3)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Executive Functioning Assessed by Behaviour Rating Inventory of Executive Functioning (BRIEF2)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Physical Performance Assessed by the 10-meter walk/run test (10-mW/RT)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Physical Activity Assessed by Accelerometer
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Affect Assessed by Positive and Negative Affect Scale - Child (PANAS-C)
timeFrame: Baseline and 1 week following the final music session.
measure: Change in Anxiety Assessed by State-Trait Anxiety Inventory Scale-5 (STAIS-5)
timeFrame: Baseline and 1 week following the final music session
measure: Change in Sleep Quality Assessed by Electroencephalogram (EEG) Headband
timeFrame: Nightly for 7 days at baseline and 1 week following the final music session.
measure: Assessment of Heart Rate Variability by Wearable Electrocardiogram (ECG) Chest Strap
timeFrame: Over 7-day period at baseline and following final music intervention.
Other outcomes:
Locations:
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
Study officials:
References: