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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 septiembre de 2026 actualizado 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.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

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 estudio

Intervencionista

Inscripción (Estimado)

25

Fase

  • No aplica

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Ubicaciones de estudio

  • Estados Unidos
    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30324
        • Reclutamiento
        • Children's Healthcare of Atlanta
        • Contacto:
        • Sub-Investigador:
          • Soumitri Sil, PhD

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Niño
  • Adulto

Acepta Voluntarios Saludables

No

Descripción

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).

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Cuidados de apoyo
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
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).
Otro: 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).
Otros nombres:
  • MONTE
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)
Otro: Standard of Care
Routinely available educational material on sickle cell disease management and a healthy lifestyle.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Change in Pain characteristics
Periodo de tiempo: 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)
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Change in Depressive Symptoms
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Otras medidas de resultado

Medida de resultado
Medida Descripción
Periodo de tiempo
Gene expression changes in immune cells
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Emory University

Colaboradores

National Institutes of Health (NIH)

Investigadores

  • Investigador principal: Manoj Bhasin, Emory University

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

1 de septiembre de 2026

Finalización primaria (Estimado)

1 de abril de 2028

Finalización del estudio (Estimado)

1 de abril de 2028

Fechas de registro del estudio

Enviado por primera vez

27 de abril de 2026

Primero enviado que cumplió con los criterios de control de calidad

27 de abril de 2026

Publicado por primera vez (Actual)

1 de mayo de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

10 de septiembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

8 de septiembre de 2026

Última verificación

1 de agosto de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 2025P012013

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

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

Marco de tiempo para compartir IPD

Beginning 9 months and ending 36 months following publication

Criterios de acceso compartido de 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 información de apoyo para compartir IPD

  • PROTOCOLO DE ESTUDIO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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