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

8. september 2026 opdateret af: 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.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

25

Fase

  • Ikke anvendelig

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

  • Forenede Stater
    • Georgia
      • Atlanta, Georgia, Forenede Stater, 30324
        • Rekruttering
        • Children's Healthcare of Atlanta
        • Kontakt:
        • Underforsker:
          • Soumitri Sil, PhD

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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).

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Støttende pleje
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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).
Andet: 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).
Andre navne:
  • MT
Sham-komparator: 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)
Andet: Standard of Care
Routinely available educational material on sickle cell disease management and a healthy lifestyle.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change in Pain characteristics
Tidsramme: 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)
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change in Depressive Symptoms
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Andre resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Gene expression changes in immune cells
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Emory University

Samarbejdspartnere

National Institutes of Health (NIH)

Efterforskere

  • Ledende efterforsker: Manoj Bhasin, Emory University

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

1. september 2026

Primær færdiggørelse (Anslået)

1. april 2028

Studieafslutning (Anslået)

1. april 2028

Datoer for studieregistrering

Først indsendt

27. april 2026

Først indsendt, der opfyldte QC-kriterier

27. april 2026

Først opslået (Faktiske)

1. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

10. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

8. september 2026

Sidst verificeret

1. august 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • 2025P012013

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

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

IPD-delingstidsramme

Beginning 9 months and ending 36 months following publication

IPD-delingsadgangskriterier

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.

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL

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