A Single-Center, Randomized, Controlled Trial of Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Vitiligo
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 96
- 试验地点
- 1
- 主要终点
- VASI 50 Response Rate at Week 24
研究概览
简要总结
This study evaluates whether exosomes derived from human umbilical cord mesenchymal stem cells (hUMSCs-Exo) are safe and effective for treating vitiligo in adults.
Vitiligo is a skin condition that causes white patches due to loss of pigment-producing cells. Current treatments have limitations, especially for patients with active disease who require oral steroids with significant side effects.
This study is a single-center, randomized, controlled trial enrolling 96 adults aged 18 to 65 years with non-segmental vitiligo. Participants are divided into two groups based on disease activity: progressive vitiligo (new patches appearing or existing patches expanding in the past 3 months) and stable vitiligo (no changes in the past year).
For progressive vitiligo, participants are randomly assigned to either:
Experimental group: hUMSCs-Exo given by intravenous infusion every 2 weeks for 5 sessions, plus tacrolimus ointment and narrowband UVB light therapy, or Control group: oral prednisone (a standard steroid treatment) plus tacrolimus ointment and narrowband UVB light therapy
For stable vitiligo, participants are randomly assigned to either:
Experimental group: hUMSCs-Exo given by local injection into the white patches every 2 weeks for 5 sessions, plus tacrolimus ointment and narrowband UVB light therapy, or Control group: tacrolimus ointment plus narrowband UVB light therapy alone The treatment period lasts 12 weeks, with follow-up visits continuing to 24 weeks. The main outcome measures include improvement in skin repigmentation measured by the Vitiligo Area Scoring Index (VASI), changes in quality of life, and safety monitoring throughout the study.
This study aims to establish a standardized approach for using hUMSCs-Exo in vitiligo treatment and to explore how exosomes may work by reducing oxidative stress and regulating immune responses.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
The study is an open-label trial due to the nature of the cellular exosome intervention, which makes blinding of participants, investigators, and outcome assessors infeasible
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinically diagnosed non-segmental vitiligo
- •Progressive vitiligo: new lesions or enlargement of existing lesions within the past 3 months (VIDA score ≥ 3); Stable vitiligo: no new lesions or enlargement of existing lesions within the past 1 year (VIDA score = 0)
- •Total body surface area (BSA) of vitiligo lesions between 1% and 30%
- •Age 18 to 65 years, male or female
- •Willing to participate and provide written informed consent
排除标准
- •Pregnancy or breastfeeding
- •Known allergy to mesenchymal stem cells or exosome components
- •Severe cardiac, hepatic, or renal dysfunction, or severe immunocompromised status
- •For progressive vitiligo: use of systemic immunosuppressants, corticosteroids, phototherapy, or photochemotherapy within the past 3 months
- •For stable vitiligo: use of phototherapy, topical immunosuppressants, or corticosteroids within the past 1 month
- •Concurrent autoimmune diseases (e.g., systemic lupus erythematosus, rheumatoid arthritis, psoriasis) or severe infectious diseases
- •History of malignancy or hematologic disorders
- •History of psychiatric disorders or inability to comply with study procedures
- •Any other condition that, in the investigator's judgment, may increase the risk to the participant or interfere with the conduct of the trial
研究组 & 干预措施
Progressive Stage Experimental Group
Patients in progressive stage receive intravenous infusion of human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) at a fixed dose of 2×10¹¹ particles per infusion, administered once every 2 weeks for a total of 5 infusions, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes (hUC-MSC-Exos) Intravenous Infusion (Biological)
Progressive Stage Experimental Group
Patients in progressive stage receive intravenous infusion of human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) at a fixed dose of 2×10¹¹ particles per infusion, administered once every 2 weeks for a total of 5 infusions, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Topical Tacrolimus Ointment (Drug)
Progressive Stage Experimental Group
Patients in progressive stage receive intravenous infusion of human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) at a fixed dose of 2×10¹¹ particles per infusion, administered once every 2 weeks for a total of 5 infusions, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Narrow-Band Ultraviolet B (NB-UVB) Phototherapy (Procedure)
Progressive Stage Control Group
Patients in progressive stage receive oral prednisone 0.5 mg/kg daily for 4 weeks followed by gradual tapering, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Topical Tacrolimus Ointment (Drug)
Progressive Stage Control Group
Patients in progressive stage receive oral prednisone 0.5 mg/kg daily for 4 weeks followed by gradual tapering, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Narrow-Band Ultraviolet B (NB-UVB) Phototherapy (Procedure)
Stable Stage Experimental Group
Patients in stable stage receive local injection of human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) at a fixed dose of 1×10¹⁰ particles per cm² of lesion area per infusion, administered once every 2 weeks for a total of 5 infusions, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes (hUC-MSC-Exos) Local Injection (Biological)
Stable Stage Experimental Group
Patients in stable stage receive local injection of human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) at a fixed dose of 1×10¹⁰ particles per cm² of lesion area per infusion, administered once every 2 weeks for a total of 5 infusions, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Topical Tacrolimus Ointment (Drug)
Stable Stage Experimental Group
Patients in stable stage receive local injection of human umbilical cord mesenchymal stem cell-derived exosomes (hUC-MSC-Exos) at a fixed dose of 1×10¹⁰ particles per cm² of lesion area per infusion, administered once every 2 weeks for a total of 5 infusions, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Narrow-Band Ultraviolet B (NB-UVB) Phototherapy (Procedure)
Stable Stage Control Group
Patients in stable stage receive topical tacrolimus ointment combined with NB-UVB phototherapy, following the routine clinical regimen.
干预措施: Topical Tacrolimus Ointment (Drug)
Stable Stage Control Group
Patients in stable stage receive topical tacrolimus ointment combined with NB-UVB phototherapy, following the routine clinical regimen.
干预措施: Narrow-Band Ultraviolet B (NB-UVB) Phototherapy (Procedure)
Progressive Stage Control Group
Patients in progressive stage receive oral prednisone 0.5 mg/kg daily for 4 weeks followed by gradual tapering, combined with topical tacrolimus ointment and NB-UVB phototherapy.
干预措施: Oral Prednisone (Drug)
结局指标
主要结局
VASI 50 Response Rate at Week 24
时间窗: Baseline to 24 weeks
Proportion of participants achieving at least 50% improvement in Vitiligo Area Scoring Index (VASI) from baseline to week 24. VASI is calculated by summing the product of body surface area affected and degree of depigmentation across all body regions. VASI 50 response is defined as (baseline VASI - week 24 VASI) / baseline VASI × 100% ≥ 50%.
次要结局
- Percent Change in VASI From Baseline(Baseline, weeks 4, 8, 12, 16, and 24)