Music Intervention for Muscular Dystrophy

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Hanns Lochmuller

Disqualifiers: Non-verbal, Insufficient English, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 12

Duration: 10 weeks

16 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test whether Dalcroze music education can help children with a specific type of muscular dystrophy called DM1. It examines whether weekly music sessions are feasible and comfortable for these children and assesses if the music can improve their brain and heart symptoms. Children aged 6 to 18 with confirmed congenital or infantile-onset DM1 who can maintain their current medication might be suitable candidates. Participants will attend music sessions, participate in assessments, and wear devices to track their progress. The trial will also gather feedback from parents to evaluate the practicality of this music approach. As an unphased trial, this study provides a unique opportunity for children to explore the potential benefits of music education in managing DM1 symptoms.

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.

What prior data suggests that this music intervention is safe for children with DM1?

Studies have shown that Dalcroze music education is generally easy for participants to handle, including those with special educational needs. Research indicates that this music-and-movement approach can lead to positive results, with most studies reporting benefits and only a few noting issues like low participation rates. Reports of serious side effects linked to this method are absent, suggesting it is safe for use in this trial. Participants in the study will engage in music sessions designed to be both fun and easy to manage, further supporting its ease of use. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike standard treatments for muscular dystrophy, which often focus on medication and physical therapy, the Dalcroze music education intervention takes a unique approach by integrating music and movement. This method is believed to enhance motor skills and coordination through rhythm and improvisation, offering a holistic benefit that traditional therapies might not address. Researchers are excited about this treatment because it could provide a non-invasive, enjoyable, and supportive therapy option that complements existing care, potentially improving quality of life for individuals with muscular dystrophy.

What evidence suggests that Dalcroze music education is effective for improving brain and heart symptoms in children with DM1?

Research has shown that activities combining music and movement, such as Dalcroze music education, can benefit individuals with brain-heart disorders. Most studies report positive results, although some faced challenges with participant adherence. Dalcroze music education uses body movement to connect with music, enhancing coordination and cognitive skills. While limited data exists on children with congenital myotonic dystrophy (DM1), this method has proven beneficial for other groups. Early findings suggest it might alleviate both brain and heart symptoms in DM1 by engaging the whole body and mind. In this trial, participants will attend weekly Dalcroze music education sessions to explore its potential benefits for DM1. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Dalcroze music education

Trial Overview

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.

How Is the Trial Designed?

Group I: Music Intervention Experimental Treatment 1 Intervention

All participants will receive weekly Dalcroze music education sessions.

Who Is Running the Clinical Trial?

Hanns Lochmuller

Lead Sponsor

Trials

1

Recruited

20+

University of Ottawa

Collaborator

Trials

231

Recruited

267,000+

Published Research Related to This Trial

A systematic review of 20 studies on Dalcroze-based music-movement interventions showed positive outcomes for vulnerable groups, including older adults and individuals with special educational needs, enhancing cognitive, physical, social, and emotional well-being.

In older adults, benefits included improved balance, mobility confidence, and quality of life, while individuals with special educational needs experienced enhancements in social interaction, cognitive abilities, and self-regulation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effectiveness of music-movement integration for vulnerable groups: a systematic literature review. Juntunen, ML., Sutela, K.[2023]

A survey of 125 parents of boys with Duchenne or Becker muscular dystrophy revealed that 73% utilized allied health services, with physiotherapy being the most common, indicating a significant reliance on these services for support.

Despite the frequent use of complementary therapies and nutritional supplements, many families reported a lack of proven efficacy, highlighting the need for further studies to provide evidence-based guidance for these treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Physical activity and the use of standard and complementary therapies in Duchenne and Becker muscular dystrophies. de Valle, KL., Davidson, ZE., Kennedy, RA., et al.[2017]

The pilot study aimed to assess the feasibility of recruiting boys with Duchenne muscular dystrophy (DMD) for a trial comparing aquatic therapy (AT) with standard land-based exercises, but only 12 out of 40 targeted participants were successfully randomized, indicating significant recruitment challenges.

While the study found that boys receiving aquatic therapy experienced less decline in motor function (NSAA score change of -2.8) compared to those receiving only land-based exercises (-5.5), the overall feasibility of conducting such a trial in the UK was deemed insufficient, suggesting a need for better community-based delivery of AT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an external pilot randomised controlled trial. Hind, D., Parkin, J., Whitworth, V., et al.[2023]

Music therapy can significantly help pediatric oncology patients by addressing physical, social, and psychological needs, such as reducing pain and anxiety, and decreasing reliance on pain medication.

It promotes social interaction and emotional expression, enhancing relationships with healthcare providers and family, which is crucial for patients undergoing long-term treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Music therapy research and applications in pediatric oncology treatment. Standley, JM., Hanser, SB.[2017]

Music interventions significantly improve various physical outcomes for adults undergoing haemodialysis, including breath rate, oxygen saturation, pain related to arteriovenous fistula puncture, and sleep quality, as well as psychological outcomes like anxiety and stress, based on a meta-analysis of 16 randomized controlled trials.

The analysis suggests that 4-6 music sessions are more effective for lowering blood pressure and heart rate, while shorter music durations of 20 minutes or less are better for reducing anxiety, indicating that both frequency and duration of music interventions are important for maximizing benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of music intervention on physical and psychological problems in adults receiving haemodialysis treatment: A systematic review and meta-analysis. Yangöz, ŞT., Özer, Z.[2022]

Music therapy, including listening to classical music and group singing, has shown benefits for patients with anorexia nervosa (AN), such as increased food consumption and reduced anxiety after meals.

While music can be therapeutic for AN and bulimia nervosa (BN), certain music-related activities, like watching music videos, may negatively impact body image and promote unhealthy body ideals, highlighting the need for careful application in treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Systematic Review of Scientific Studies on the Effects of Music in People with or at Risk for Eating Disorders. Testa, F., Arunachalam, S., Heiderscheit, A., et al.[2021]

A 15-week recreational dance and singing program for six individuals with neuromuscular conditions showed no safety concerns and led to respiratory improvements, as well as enhancements in swallowing and speech.

Participants reported significant quality-of-life benefits, including better relationships and leisure activities, highlighting the program's role in providing social connection and enjoyable physical activity for those affected by neuromuscular conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Singing and Dancing With Neuromuscular Conditions: A Mixed-Methods Study. Peers, D., Eales, L., Jones, K., et al.[2022]

In a study of 80 adults with Duchenne muscular dystrophy (DMD), high prevalence rates were found for fatigue (40.5%), pain (73.4%), anxiety (24%), and depression (19%), indicating that these symptoms are common in this population.

These symptoms significantly impact health-related quality of life, with fatigue affecting physical health and environment domains, while anxiety influences the psychological domain, suggesting that addressing these issues could improve overall well-being.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prevalence of fatigue, pain, and affective disorders in adults with duchenne muscular dystrophy and their associations with quality of life. Pangalila, RF., van den Bos, GA., Bartels, B., et al.[2015]

In a study of 1,057 males with Duchenne muscular dystrophy (DMD), current steroid use was found to be the most significant predictor of delayed loss of ambulation, indicating that corticosteroids can improve wheelchair-free survival.

Among commonly used steroids, deflazacort was associated with a longer duration of ambulation compared to prednisone, with median delays of 14 years and 13 years, respectively.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits. Wang, RT., Silverstein Fadlon, CA., Ulm, JW., et al.[2020]

Music therapy is a beneficial intervention for hospice patients, helping to improve their social relationships and potentially enhancing trust and belief during challenging times.

Research indicates positive outcomes from music therapy, supported by feedback from nurses, music therapists, families, and patients, suggesting it should be more widely implemented and studied in hospice care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The patients can't wait, and why should they? Gifford, EJ.[2009]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06809049?cond=Congenital%20myotonic%20dystrophy%20OR%20%22%22&aggFilters=status:not%20rec&viewType=Table&rank=6

Music Intervention for Brain-Heart Disease in Myotonic ...

The goal of this interventional study is to demonstrate the feasibility and tolerability of Dalcroze music intervention for children with congenital DM1, ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10513045/

The effectiveness of music–movement integration for ...

All studies showed beneficial outcomes for music–movement intervention except one that suffered from low adherence rates.

3.

researchgate.net

researchgate.net/publication/396800474_Community-dwelling_older_adults_music_education_and_performance_self-efficacy_Comparing_two_improvisational_methods

Community-dwelling older adults, music education, and ...

Based on the criteria of the Asian Working Group on Muscular Dystrophy, participants were classified into a sarcopenia group (31 cases) and a ...

4.

hrmars.com

hrmars.com/papers_submitted/24387/positive-impact-of-dalcroze-eurhythmics-a-systematic-review.pdf

Positive Impact of Dalcroze Eurhythmics: A Systematic ...

This review seeks to explore the three core impacts of DE in the field of music: performance skills (eight papers), musical theory knowledge ( ...

5.

eric.ed.gov

eric.ed.gov/?id=ED503006

The Quantifying Analysis of Effectiveness of Music ...

This study aims at investigating how children can be motivated and inspired rhythmically by body movement through Dalcroze approach. In this research, the ...

6.

researchgate.net

researchgate.net/publication/313422778_Inclusive_music_education_The_potential_of_the_Dalcroze_approach_for_students_with_special_educational_needs

(PDF) Inclusive music education: The potential of ...

In this article, we discuss the potential of Dalcroze-inspired music education for students with special educational needs (SEN), based on preliminary findings ...

7.

oulurepo.oulu.fi

oulurepo.oulu.fi/bitstream/handle/10024/36691/isbn978-952-62-2587-6.pdf?sequence=1

Exploring the possibilities of Dalcroze-based music education ...

For this purpose, the study examined the possibilities of Dalcroze-based music-and-movement teaching to foster agency, especially the bodily ...

8.

ijea.org

ijea.org/v24si2/v24si2.6/v24si2.6.pdf

v24si2.6.pdf

The purpose of this article is to explore the extent to which the PERMA model of well-being. (Seligman, 2011) can help us understand Dalcroze ...