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

Trial Summary

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. ^{1 2 3 4 5}

How 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. ^{6 7 8 9 10}

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.

Eligibility 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.

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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]

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 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]

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]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Physical activity and the use of standard and complementary therapies in Duchenne and Becker muscular dystrophies. [2017]

2. England pubmed.ncbi.nlm.nih.gov

Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an external pilot randomised controlled trial. [2023]

3. United States 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. [2015]

4. United States 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. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Singing and Dancing With Neuromuscular Conditions: A Mixed-Methods Study. [2022]

6. Switzerland pubmed.ncbi.nlm.nih.gov

The effectiveness of music-movement integration for vulnerable groups: a systematic literature review. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

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

8. United States pubmed.ncbi.nlm.nih.gov

Music therapy research and applications in pediatric oncology treatment. [2017]

9. Croatia 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. [2021]

10. England 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. [2022]