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

NCT06809049 | Recruiting

• 3 other identifiers: 24/143XCFREF-2022-00007BHI 23-118
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief 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:
• The feasibility of attendance, as assessed by attendance rate to 10 music sessions (≥ 60%)
• Feasibility of attendance, as rated by parents/caregivers of participants (≥60% rate "extremely" or "very" practical to attend)
• Attrition rate of the study, as determined by percentage of participants who complete the study (≥ 60%)
• Overall satisfaction, as rated by parents/caregivers of participants (≥60% rate "very satisfied" or "satisfied")

Conditions

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

Keywords

Music intervention; Myotonic dystrophy; DM1; Rare disease

Outcome Measures

Primary Outcomes (3)

• Attendance to Each Music Session

  Participant attendance rate to 10 music sessions.

  Over 10-week block of music sessions

• Attendance to Block of Music Sessions

  The proportion of participants who attend all 10 music sessions.

  Over 10-week block of music sessions

• Qualitative Feedback from Parents/Caregivers Following Each Music Session

  Parents/Caregivers will complete short questionnaires following each of the 10 music sessions. Questions will focus on recall of participant mood, tiredness and engagement in the 24 hours immediately following each music session.

  Following each music session through to the final music session.

Secondary Outcomes (22)

• Change in Physical Performance Assessed by the 6-Minute Walk Test (6MWT)

  Baseline and 1 week following the final music session.

• Change from Baseline in Clinical Phenotype Assessed by the Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

  Baseline and 1 week following the final music session.

• Change in Global IQ Assessed by Wechsler Abbreviated Scale of Intelligence Second Edition (WASI-II)

  Baseline and 1 week following the final music session.

• Change in Attention Assessed by Bells Assessment

  Baseline and 1 week following the final music session.

• Change in Working Memory Assessed by NIH List Sorting Working Memory Test

  Baseline and 1 week following the final music session.

Study Arms (1)

Music Intervention

EXPERIMENTAL

All participants will receive weekly music and movement education sessions.

Behavioral: Music and movement education

Interventions

Music and movement education BEHAVIORAL

The music and movement intervention is partially inspired by an adaptive music-education method called Dalcroze eurhythmics. It 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 this music and movement education to employ a holistic approach to improve the brain-heart symptoms of children with congenital DM1.

Music Intervention

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Hanns Lochmuller: lead
• University of Ottawa: collaborator

Study Sites (1)

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy; Rare Diseases

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Senior Scientist

Model Details: Participants will be grouped with 3-7 other participants of similar age and ability for the music education sessions. All participants will receive the music intervention.

Study Record Dates

• First Submitted: January 27, 2025
• First Posted: February 5, 2025
• Study Start: March 25, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: February 19, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Access Criteria: IPD will be stored within the ARCHIMEDES health data portal and accessible to qualified researchers with a research plan approved by a data access committee. Supporting information (protocol and informed consent forms) will be available as a publication.

Locations

CA (1)