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Assessing Molecular Mechanisms and Effects of Music Therapy in Youth With Sickle Cell Disease Using Single-cell RNA-sequencing

8 de setembro de 2026 atualizado por: Manoj Bhasin, Emory University

The goal of this clinical trial is to evaluate whether a 4-week music therapy (MT) intervention can reduce chronic pain and improve psychosocial outcomes in youth with sickle cell disease (SCD).

The main questions it aims to answer are:

  • Does MT reduce pain intensity, frequency of pain episodes, and improve health-related quality of life (HRQoL)?
  • Does MT alter immune cell composition and gene expression in inflammatory pathways, as measured by single-cell RNA sequencing (scRNA-seq)?

Researchers will compare participants randomized to music therapy versus a control condition to see if MT produces superior improvements in pain and psychosocial outcomes, and distinct molecular changes.

Visão geral do estudo

Status

Recrutamento

Condições

Intervenção / Tratamento

Descrição detalhada

Sickle cell disease (SCD) is an inherited blood disorder that affects approximately 100,000 individuals in the United States, with higher prevalence among people of African and Latin-American descent. Children and adolescents with SCD often experience recurrent vaso-occlusive episodes (VOE) and chronic pain, which contribute to emotional distress, impaired academic performance, reduced daily functioning, and increased hospital utilization. Chronic pain also imposes a substantial financial burden and is associated with anxiety, depression, and diminished quality of life. Current pharmacologic treatments, such as hydroxyurea and blood transfusions, can reduce pain crises but are limited by side effects that interfere with school attendance and social participation, underscoring the need for safe, nonpharmacologic alternatives. Integrative approaches, such as cognitive behavioral therapy (CBT) and music therapy (MT), have been recognized as effective non-pharmacological pain management strategies, with evidence showing improvements in pain, mood, fatigue, and overall quality of life. However, the molecular mechanisms underlying these benefits remain poorly understood, particularly in SCD.

This study will evaluate the effects of a 4-week MT intervention in youth with SCD, focusing on both psychosocial outcomes and molecular mechanisms. The intervention will be delivered using Music Care©, a virtual platform that employs a structured "U" algorithm, beginning with stimulating music, transitioning into relaxation, and concluding with a gentle reintroduction of rhythm and energy. The primary aim is to determine whether MT reduces pain intensity, frequency of pain episodes, stress, anxiety, depression, and improves health-related quality of life (HRQoL). The secondary aim is to investigate changes in immune cell composition and gene expression using single-cell RNA sequencing (scRNA-seq), which will enable detailed profiling of immune cells, including T cells, B cells, monocytes, neutrophils, and granulocytes, as well as their transcriptional states and regulatory networks. Samples will be collected at baseline and endpoint to identify pathways altered by MT and to correlate molecular findings with clinical outcomes.

By integrating psychosocial assessments with single-cell transcriptomic profiling, this study seeks to provide novel insights into how MT modulates immune and inflammatory pathways in pediatric SCD. Understanding these mechanisms may reveal new therapeutic targets, improve patient stratification, and support broader adoption of MT as an evidence-based, nonpharmacologic intervention for chronic pain management in children and adolescents living with SCD.

Tipo de estudo

Intervencional

Inscrição (Estimado)

25

Estágio

  • Não aplicável

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Locais de estudo

  • Estados Unidos
    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30324
        • Recrutamento
        • Children's Healthcare of Atlanta
        • Contato:
        • Subinvestigador:
          • Soumitri Sil, PhD

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Filho
  • Adulto

Aceita Voluntários Saudáveis

Não

Descrição

Inclusion Criteria:

  • Diagnosed with SCD
  • English fluency (for survey completion)
  • Have access to a personal electronic device (e.g., phone, tablet) with access to the internet

  • Meet the criteria for chronic pain defined by the International Association for the Study of Pain (IASP). IASP defines chronic pain as pain that is both:

    1. Persistent or recurrent for ≥ 3 months
    2. is associated with significant emotional distress or functional disability.30

Exclusion Criteria:

  • Major hearing deficiency or medical condition in which listening to music may be contraindicated (e.g., reflex epilepsy).

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Cuidados de suporte
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Music Therapy group
The MT group will engage in 20-minute daily virtual music therapy sessions for 4 weeks, using the freely available Music Care© app (manufactured by MUSIC CARE) to independently participate in electronic music therapy following the U sequence. This app is voluntarily downloaded onto a participant's personal device, and participants will be instructed to complete daily MT sessions under optimal conditions (i.e., lying down, eyes closed, low lighting).
Outro: Music Therapy
The MT group will engage in 20-minute daily virtual music therapy sessions for 4 weeks, using the freely available Music Care© app (manufactured by MUSIC CARE) to independently participate in electronic music therapy following the U sequence. This app is voluntarily downloaded onto a participants personal device and participants will be instructed to complete daily MT sessions under optimal conditions (i.e., lying down, eyes closed, low lighting).
Outros nomes:
  • MT
Comparador Falso: Control group
The control group will be asked to engage with educational material for 20 minutes daily. There will be no restrictions for research subjects in regards to listening to music under other conditions (e.g., phone, radio, concert, etc)
Outro: Standard of Care
Routinely available educational material on sickle cell disease management and a healthy lifestyle.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Change in Pain characteristics
Prazo: Baseline, Week 5

The Brief Pain Inventory (BPI) is a validated self-report questionnaire that measures both the severity of pain and the degree to which pain interferes with daily activities.

Pain intensity is rated on a 0-10 numeric scale (0 = no pain, 10 = worst pain imaginable). Pain interference items are also scored 0-10 (0 = does not interfere, 10 = completely interferes). Greater scores indicate more severe pain and greater interference with daily activities.
Baseline, Week 5
Change in Health-related quality of life (HRQoL)
Prazo: Baseline, Week 5

Parents will complete the Pediatric Quality of Life Inventory (PedsQL) at baseline and Week 5. This parent-proxy measure assesses physical, emotional, social, and school functioning. Changes in HRQoL scores will be analyzed to determine whether music therapy improves overall well-being compared to control.

Scoring: Items are scored on a 0-100 scale, with higher scores reflecting better quality of life (less impairment). Lower scores indicate poorer functioning across physical, emotional, social, and school domains.
Baseline, Week 5

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Change in Depressive Symptoms
Prazo: Baseline, Week 5

Youth participants will complete the Patient Health Questionnaire (PHQ-8) at baseline and Week 5. This validated tool measures the frequency and severity of depressive symptoms.

Each item scored 0-3 (0 = not at all, 3 = nearly every day). Total score range: 0-24. Higher scores indicate greater severity of depressive symptoms.
Baseline, Week 5
Change in General Anxiety Disorder scale scores
Prazo: Baseline, Week 5

The General Anxiety Disorder scale (GAD-2) will be administered at baseline and Week 5. This brief self-report tool screens for generalized anxiety symptoms.

Each item scored 0-3, total score range: 0-6. Higher scores indicate greater anxiety symptoms.
Baseline, Week 5
Change in Psychological Stress
Prazo: Baseline, Week 5

The PROMIS Pediatric Short Form - Psychological Stress will be collected at baseline and Week 5 to assess perceived psychological stress.

Items scored on a 5-point Likert scale. Raw scores are converted to T-scores (mean = 50, SD = 10). Higher T-scores indicate greater psychological stress.
Baseline, Week 5
Change in Physical stress
Prazo: Baseline, Week 5

The PROMIS Pediatric Short Form - Physical Stress will be collected at baseline and Week 5 to measure somatic stress symptoms.

Items scored on a 5-point Likert scale. Raw scores are converted to T-scores (mean = 50, SD = 10). Higher T-scores indicate greater psychological stress.
Baseline, Week 5
Hospital utilization
Prazo: 4 weeks
The number of hospitalizations or pain-related treatments during the 4-week study will be tracked through medical chart review and participant report.
4 weeks

Outras medidas de resultado

Medida de resultado
Descrição da medida
Prazo
Gene expression changes in immune cells
Prazo: Baseline, Week 5
gene expression changes in immune cells (T cells, B cells, monocytes, neutrophils, granulocytes). Assessed via single-cell RNA sequencing (scRNA-seq)
Baseline, Week 5

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Emory University

Colaboradores

National Institutes of Health (NIH)

Investigadores

  • Investigador principal: Manoj Bhasin, Emory University

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

1 de setembro de 2026

Conclusão Primária (Estimado)

1 de abril de 2028

Conclusão do estudo (Estimado)

1 de abril de 2028

Datas de inscrição no estudo

Enviado pela primeira vez

27 de abril de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

27 de abril de 2026

Primeira postagem (Real)

1 de maio de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

10 de setembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

8 de setembro de 2026

Última verificação

1 de agosto de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • 2025P012013

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

SIM

Descrição do plano IPD

Individual participant data that underlie the results reported in the publication will be shared after de-identification

Prazo de Compartilhamento de IPD

Beginning 9 months and ending 36 months following publication

Critérios de acesso de compartilhamento IPD

Investigators whose proposed use of the data is provided in a formal written request, has been approved by the IRB, and receives explicit PI approval. Proposals may be submitted up to 36 months following publication. After 36 months the data will be available in our University's data warehouse but without investigator support other than deposited metadata.

Tipo de informação de suporte de compartilhamento de IPD

  • PROTOCOLO DE ESTUDO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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