Music Intervention for Muscular Dystrophy
Recruiting in Palo Alto (17 mi)
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Academic
Recruiting
Sponsor: Hanns Lochmuller
Disqualifiers: Non-verbal, Insufficient English, others
No Placebo Group
Trial Summary
What is the purpose of this trial?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.
Will I have to stop taking my current medications?
No, you will not have to stop taking your current medications. Participants are required to stay on stable medication from the day of screening to the end of the study.
Is music intervention safe for people with neuromuscular conditions?
A study on a 15-week recreational dance and singing program for people with neuromuscular conditions found no evidence of safety concerns, suggesting that such activities can be safe and meaningful.
12345How does the Dalcroze music education treatment differ from other treatments for muscular dystrophy?
Dalcroze music education is unique because it combines music and movement to improve physical, cognitive, and social abilities, which is different from traditional treatments that may focus solely on physical therapy or medication. This approach has shown benefits in improving balance, mobility, and social interaction, making it a novel option for enhancing quality of life in individuals with muscular dystrophy.
678910Eligibility Criteria
This trial is for children with congenital Myotonic Dystrophy Type 1 (DM1) who have brain and heart symptoms. They must be able to attend weekly music sessions for 10 weeks, provide biological samples, and use wearable devices for monitoring. Parents should also participate in questionnaires and focus groups.Inclusion Criteria
I agree to not change my medications during the study.
I am aged 6-17 with a genetic diagnosis of early-onset DM1.
Exclusion Criteria
Patients for whom - in the opinion of the investigator - it would not be safe to participate in the study
Insufficient English language skills to complete required assessments and questionnaires
I cannot speak.
+3 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants attend 45-minute-long music sessions once weekly for 10 weeks and provide biological samples
10 weeks
10 visits (in-person)
Follow-up
Participants are monitored for changes in physical and cognitive assessments, and qualitative feedback is collected
1 week
2 visits (in-person)
Extension
Participants may continue to use wearable devices for extended monitoring of brain-heart biomarkers
Long-term
Participant Groups
The study tests the feasibility of Dalcroze music education as a treatment to improve brain-heart symptoms in DM1 patients. It involves weekly music sessions, assessments at baseline and after the intervention, along with collecting data from wearables and biological samples.
1Treatment groups
Experimental Treatment
Group I: Music InterventionExperimental Treatment1 Intervention
All participants will receive weekly Dalcroze music education sessions.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Children's Hospital of Eastern OntarioOttawa, Canada
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Who Is Running the Clinical Trial?
Hanns LochmullerLead Sponsor
University of OttawaCollaborator
References
Physical activity and the use of standard and complementary therapies in Duchenne and Becker muscular dystrophies. [2017]To record the use and perceived benefits of mainstream allied health services, complementary therapies, nutritional supplements and structured physical activity in a paediatric population of males with Duchenne or Becker muscular dystrophy.
Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an external pilot randomised controlled trial. [2023]Standard treatment of Duchenne muscular dystrophy (DMD) includes regular physiotherapy. There are no data to show whether adding aquatic therapy (AT) to land-based exercises helps maintain motor function. We assessed the feasibility of recruiting and collecting data from boys with DMD in a parallel-group pilot randomised trial (primary objective), also assessing how intervention and trial procedures work.
Prevalence of fatigue, pain, and affective disorders in adults with duchenne muscular dystrophy and their associations with quality of life. [2015]To assess the prevalence of fatigue, pain, anxiety, and depression in adults with Duchenne muscular dystrophy (DMD), and to analyze their relationship with health-related quality of life.
Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits. [2020]To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and age at loss of ambulation. Because registrants reported differences in steroid and other medication usage, as well as age and ambulation status, we could explore these data for correlations with age at loss of ambulation. Using multivariate analysis, current steroid usage was the most significant and largest independent predictor of improved wheelchair-free survival. Thus, these online self-report data were sufficient to retrospectively observe that current steroid use by patients with DMD is associated with a delay in loss of ambulation. Comparing commonly used steroid drugs, deflazacort prolonged ambulation longer than prednisone (median 14 years and 13 years, respectively). Further, use of Vitamin D and Coenzyme Q10, insurance status, and age at diagnosis after 4 years were also significant, but smaller, independent predictors of longer wheelchair-free survival. Nine other common supplements were also individually tested but had lower study power. This study demonstrates the utility of DuchenneConnect data to observe therapeutic differences, and highlights needs for improvement in quality and quantity of patient-report data, which may allow exploration of drug/therapeutic practice combinations impractical to study in clinical trial settings. Further, with the low barrier to participation, we anticipate substantial growth in the dataset in the coming years.
Singing and Dancing With Neuromuscular Conditions: A Mixed-Methods Study. [2022]The purpose of this study was to assess the safety and meaningfulness of a 15-week recreational dance and singing program for people with neuromuscular conditions. Within a transformative mixed-methods design, pulmonary function tests, plethysmography through wearable technology (Hexoskin vests), individualized neuromuscular quality-of-life assessments (version 2.0), and semistructured interviews were used. The interviews were analyzed through inductive, semantic thematic analysis. Although the sample sizes were small (six people with neuromuscular conditions), the authors found no evidence of safety concerns. There was evidence of respiratory improvements and reported improvements in swallowing and speech. The most notable quality-of-life changes included improvements related to weakness, swallowing, relationships, and leisure. The participants shared that the program offered meaningful social connection and embodied skills and safe and pleasurable physical exertion. The authors learned that recreational singing and dancing programs could be a safe and deeply meaningful activity for those with neuromuscular conditions that impact respiration.
The effectiveness of music-movement integration for vulnerable groups: a systematic literature review. [2023]This systematic review synthesized the outcomes of previous intervention studies published from January 2000-October 2022 to evaluate the effectiveness of Dalcroze-based or similar music-movement integration among groups of individuals considered vulnerable (in relation to their abilities and health/wellbeing). The target groups addressed in previous intervention studies included individuals with special educational needs (such as disorders, disabilities, or impairments) or with a (risk of) decline in health and/or physical strength. Twenty articles met the review inclusion criteria. All studies showed beneficial outcomes for music-movement intervention except one that suffered from low adherence rates. In older adults, the benefits were cognitive, physical, social, and/or emotional, including improved postural stability, balance, gait safety, confidence in mobility, metamemory skills, dual-task performance, social and physical pleasure, autotelic/flow experience, enjoyment, health, and quality of life. In individuals with special educational needs, improvement was seen in relation to inclusion, reductions in compulsive and other problematic behaviors, self-regulation, perceptual and cognitive abilities and functions, linguistic and learning skills, auditory attention and phonological awareness, social interaction, engagement, and agency.
The patients can't wait, and why should they? [2009]The three Ds of disease, disability, and discomfort negatively affect hospice patient's energy, intelligence, and organization. Music therapy is a studied, learned, credentialed behavioral intervention. It is specific, prescribed, and delivered therapeutically. At a time when trust and belief are compromised by the three Ds, the utilization of the complementary alternative medical intervention of music therapy, with its positive effect on social relationships, may enhance weakened trust and belief abilities. The research to date regarding music therapy presents positive results from studies and surveys of nurses, music therapists, families, and patients. Recommendations for using music therapy and music therapists will establish the use of this intervention, giving increased numbers to sample sizes for further study.
Music therapy research and applications in pediatric oncology treatment. [2017]Music therapy is a profession which meets multiple physical, social, and psychological needs. Music therapists can facilitate health objectives by reducing the intensity or duration of pain, alleviating anxiety, and decreasing the amount of analgesic medication needed. Rehabilitative objectives can include activities which incorporate exercise, range of motion therapy, or gait training. Reduction of fear, anxiety, stress, or grief are common psychological objectives. Music therapy is particularly effective in promoting social objectives such as increased interaction, verbalization, independence, and cooperation; enhanced relationships with health care personnel and family members; and increased stimulation during long-term hospitalization or isolation. Counseling techniques are often paired with music to achieve emotional objectives such as expression, adjustment, stability, or locus of control. The purpose of this article is to synthesize the extant music/medical research literature and clarify how music therapy can provide a quintessential combination of physical, social, and psychological benefits to enhance the health care of pediatric oncology patients.
A Systematic Review of Scientific Studies on the Effects of Music in People with or at Risk for Eating Disorders. [2021]The prevalence of the three main eating disorders (EDs) anorexia nervosa (AN), bulimia nervosa (BN) and binge eating disorder (BED) is increasing, and a growing number of patients with EDs is seeking professional help. Thus, there is a need for additional treatment strategies in EDs. The aim of this review was to summarize the literature on the benefits and risks of music as well as the evidence for its therapeutic application in people with EDs.
Effects of music intervention on physical and psychological problems in adults receiving haemodialysis treatment: A systematic review and meta-analysis. [2022]To synthesise the effects of music intervention on the physical and psychological problems of adults receiving haemodialysis.