Assessing Molecular Mechanisms and Effects of Music Therapy in Youth With Sickle Cell Disease Using Single-cell RNA-sequencing
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.
調査の概要
詳細な説明
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.
研究の種類
入学 (推定)
段階
- 適用できない
連絡先と場所
研究連絡先
- 名前：Manoj Bhasin, PhD
- 電話番号：404-712-9849
- メール：manoj.bhasin@emory.edu
研究場所
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Georgia
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Atlanta、Georgia、アメリカ、30324
- 募集
- Children's Healthcare of Atlanta
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コンタクト：
- Soumitri Sil, PhD
- 電話番号：404-727-2712
- メール：soumitri.sil@emory.edu
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副調査官：
- Soumitri Sil, PhD
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-
参加基準
適格基準
就学可能な年齢
- 子
- 大人
健康ボランティアの受け入れ
説明
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:
- Persistent or recurrent for ≥ 3 months
- 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).
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：支持療法
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：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).
|
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).
他の名前：
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偽コンパレータ：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)
|
Routinely available educational material on sickle cell disease management and a healthy lifestyle.
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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change in Pain characteristics
時間枠：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
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Change in Health-related quality of life (HRQoL)
時間枠：Baseline, Week 5
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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
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change in Depressive Symptoms
時間枠：Baseline, Week 5
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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
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Change in General Anxiety Disorder scale scores
時間枠：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
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Change in Psychological Stress
時間枠：Baseline, Week 5
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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
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Change in Physical stress
時間枠：Baseline, Week 5
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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
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Hospital utilization
時間枠：4 weeks
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The number of hospitalizations or pain-related treatments during the 4-week study will be tracked through medical chart review and participant report.
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4 weeks
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その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Gene expression changes in immune cells
時間枠：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
|
協力者と研究者
スポンサー
捜査官
- 主任研究者：Manoj Bhasin、Emory University
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- 2025P012013
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
IPD 共有時間枠
IPD 共有アクセス基準
IPD 共有サポート情報タイプ
- STUDY_PROTOCOL
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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