A Prospective Study of Madecassoside Tablets in Sclerotic Chronic Graft-versus-Host Disease After Allo-HSCT
A Prospective, Multicenter, Single-Arm Phase 2 Study of Madecassoside Tablets Combined With Standard Therapy for Sclerotic Chronic Graft-versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
Sclerotic chronic graft-versus-host disease (scl-cGVHD) is a severe subtype of chronic GVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT), characterized by skin sclerosis, fascial involvement, and restricted joint mobility, leading to substantial functional impairment and reduced quality of life. Current standard therapies, including corticosteroids and other immunosuppressive agents, have limited efficacy in patients with refractory or steroid-dependent disease.
Madecassoside tablets, a standardized extract of Centella asiatica, have demonstrated anti-inflammatory and anti-fibrotic properties in previous studies and may provide therapeutic benefit in fibrotic diseases.
This study is a prospective, multicenter, single-arm phase 2 clinical trial designed to evaluate the efficacy and safety of madecassoside tablets combined with standard therapy in patients with scl-cGVHD after allo-HSCT. Eligible participants will receive oral madecassoside tablets (0.2 g three times daily) in addition to standard treatment for 6 months. Clinical assessments will be performed at baseline and at weeks 4, 8, 12, and 24.
The primary endpoint is the improvement rate of skin and/or joint/fascia NIH cGVHD scores at 6 months according to the 2014 NIH consensus criteria. Secondary endpoints include overall cGVHD response rate, failure-free survival, non-relapse mortality, corticosteroid dose reduction, patient-reported outcomes, and safety assessments. Peripheral blood samples will also be collected to explore changes in inflammatory cytokines and lymphocyte subsets during treatment.
研究概览
研究类型
注册 (估计的)
阶段
- 阶段2
联系人和位置
学习联系方式
- 姓名：wei MD, PhD
- 电话号码：+86 027-85726003
- 邮箱：shiwei076@hust.edu.cn
学习地点
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Hubei
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Wuhan、Hubei、中国、430022
- 招聘中
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
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接触：
- Wei Shi, M.D.
- 电话号码：+86 027-85726003
- 邮箱：shiwei076@hust.edu.cn
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参与标准
资格标准
适合学习的年龄
- 孩子
- 成人
- 年长者
接受健康志愿者
描述
Inclusion Criteria:
- Ability to understand and willingness to sign written informed consent;
- Age 14 to 65 years;
- Diagnosis of sclerotic chronic graft-versus-host disease (scl-cGVHD) according to the 2014 NIH consensus criteria, including superficial or deep skin sclerosis, fasciitis, or restricted joint mobility;
Prior treatment history meeting at least one of the following:
- Received systemic corticosteroid therapy for ≥12 months and at least 1 additional systemic therapy; OR
- Received corticosteroid therapy and at least 2 additional systemic therapies;
- Karnofsky Performance Status (KPS) score ≥60;
- Absolute neutrophil count >1,000/μL;
- Platelet count ≥50,000/μL.
Exclusion Criteria:
- 1. Total bilirubin >1.5 times the upper limit of normal; 2. Creatinine clearance <30 mL/min; 3. Uncontrolled infection; 4. Uncontrolled cardiovascular or pulmonary disease; 5. Any clinical condition that, in the investigator's judgment, makes the participant unsuitable for study participation.
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的：Experimental: Madecassoside Tablets Plus Standard Therapy
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Madecassoside tablets will be administered orally at a dose of 0.2 g (2 tablets) three times daily for 6 months in combination with standard therapy.
Standard therapy may include corticosteroids, calcineurin inhibitors, ruxolitinib, belumosudil, topical medications, and supportive care according to institutional guidelines and physician discretion.
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Change in Skin and/or Joint/Fascia NIH cGVHD Scores
大体时间：6 months
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The proportion of participants achieving complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) in skin and/or joint/fascia manifestations according to the 2014 NIH chronic graft-versus-host disease response criteria after 6 months of treatment.
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6 months
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Overall cGVHD Response Rate
大体时间：6 months
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Overall response rate of chronic graft-versus-host disease based on the 2014 NIH response criteria.
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6 months
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Failure-Free Survival
大体时间：2 years
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Failure-free survival defined as survival without relapse, non-relapse mortality, or addition of new systemic therapy.
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2 years
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Non-Relapse Mortality
大体时间：2 years
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Incidence of death without relapse of the underlying disease during follow-up.
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2 years
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Corticosteroid Dose Reduction
大体时间：6 months
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Proportion of participants achieving at least 50% reduction or discontinuation of corticosteroid therapy.
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6 months
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Patient-Reported Outcomes
大体时间：Baseline to 6 months
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Changes in patient-reported outcomes including Lee Symptom Scale (LSS), Scleroderma Health Assessment Questionnaire (SHAQ), and Photographic Range of Motion (P-ROM) scores.
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Baseline to 6 months
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Traditional Chinese Medicine Symptom Score
大体时间：Baseline to 6 months
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Changes in Traditional Chinese Medicine symptom scores during treatment.
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Baseline to 6 months
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Safety and Adverse Events
大体时间：Up to 2 years
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Incidence and severity of adverse events assessed according to CTCAE version 5.0.
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Up to 2 years
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合作者和调查者
赞助
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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