Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Attendance to Each Music Session
研究概览
简要总结
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")
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Music Intervention
All participants will receive weekly music and movement education sessions.
干预措施: Music and movement education (Behavioral)
结局指标
主要结局
Attendance to Each Music Session
时间窗: Over 10-week block of music sessions
Participant attendance rate to 10 music sessions.
Attendance to Block of Music Sessions
时间窗: Over 10-week block of music sessions
The proportion of participants who attend all 10 music sessions.
Qualitative Feedback from Parents/Caregivers Following Each Music Session
时间窗: Following each music session through to the final 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.
次要结局
- Change in Visuospatial Skills Assessed by Rex Complex Figure - Copy(Baseline and 1 week following the final music session.)
- Change in Episodic Memory Assessed by NIH Picture Sequence Memory Test(Baseline and 1 week following the final music session.)
- Change in Receptive Vocabulary Assessed by Peabody Picture Vocabulary Test (PPVT-5)(Baseline and 1 week following the final music session.)
- Change in Mood and Anxiety Assessed by Mood and Feelings Questionnaire (MFQ)(Baseline and 1 week following the final music session.)
- Change in Adaptive Functioning Assessed by Vineland Adaptive Behaviour Scale(Baseline and 1 week following the final music session.)
- Changes in Attention Assessed by Conners Continuous Performance Test Third Edition (CPT3)(Baseline and 1 week following the final music session.)
- Change in Executive Functioning Assessed by Behaviour Rating Inventory of Executive Functioning (BRIEF2)(Baseline and 1 week following the final music session.)
- Change in Physical Activity Assessed by Accelerometer(Baseline and 1 week following the final music session.)
- Change in Affect Assessed by Positive and Negative Affect Scale - Child (PANAS-C)(Baseline and 1 week following the final music session.)
- Change in Anxiety Assessed by State-Trait Anxiety Inventory Scale-5 (STAIS-5)(Baseline and 1 week following the final music session)
- Change in Sleep Quality Assessed by Electroencephalogram (EEG) Headband(Nightly for 7 days at baseline and 1 week following the final music session.)
- Assessment of Heart Rate Variability by Wearable Electrocardiogram (ECG) Chest Strap(Over 7-day period at baseline and following final music intervention.)
- 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.)
- Change in Executive Functioning Assessed by Dimensional Change Card Sort(Baseline and 1 week following the final music session.)
- Change in Quality of Life Assessed by Pediatric Quality of Life Inventory (PedsQL)(Baseline and 1 week following the final music session.)
- Change in Sleep Quality Assessed by Children's Sleep Habits Questionnaire(Baseline and 1 week following the final music session.)
- Assessment of Cardiac Conduction Defects by Electrocardiogram (ECG)(Baseline and 1 week following the final music session.)
- Change in Physical Performance Assessed by the 10-meter walk/run test (10-mW/RT)(Baseline and 1 week following the final music session.)
研究者
Hanns Lochmuller
Senior Scientist
Children's Hospital of Eastern Ontario