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

8 settembre 2026 aggiornato da: 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.

Panoramica dello studio

Stato

Reclutamento

Condizioni

Intervento / Trattamento

Descrizione dettagliata

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 di studio

Interventistico

Iscrizione (Stimato)

25

Fase

  • Non applicabile

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

Luoghi di studio

  • Stati Uniti
    • Georgia
      • Atlanta, Georgia, Stati Uniti, 30324
        • Reclutamento
        • Children's Healthcare of Atlanta
        • Contatto:
        • Sub-investigatore:
          • Soumitri Sil, PhD

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino
  • Adulto

Accetta volontari sani

No

Descrizione

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).

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Terapia di supporto
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: 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).
Altro: 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).
Altri nomi:
  • MT
Comparatore fittizio: 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)
Altro: Standard of Care
Routinely available educational material on sickle cell disease management and a healthy lifestyle.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Change in Pain characteristics
Lasso di tempo: 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)
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Change in Depressive Symptoms
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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

Altre misure di risultato

Misura del risultato
Misura Descrizione
Lasso di tempo
Gene expression changes in immune cells
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Emory University

Collaboratori

National Institutes of Health (NIH)

Investigatori

  • Investigatore principale: Manoj Bhasin, Emory University

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

1 settembre 2026

Completamento primario (Stimato)

1 aprile 2028

Completamento dello studio (Stimato)

1 aprile 2028

Date di iscrizione allo studio

Primo inviato

27 aprile 2026

Primo inviato che soddisfa i criteri di controllo qualità

27 aprile 2026

Primo Inserito (Effettivo)

1 maggio 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

10 settembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

8 settembre 2026

Ultimo verificato

1 agosto 2026

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • 2025P012013

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

SÌ

Descrizione del piano IPD

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

Periodo di condivisione IPD

Beginning 9 months and ending 36 months following publication

Criteri di accesso alla condivisione 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 di informazioni di supporto alla condivisione IPD

  • STUDIO_PROTOCOLLO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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