Prospective Randomized Controlled Trial of Novel Therapies for Severe Acute GVHD (Graft-versus-host Disease)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 54
- 试验地点
- 1
- 主要终点
- Overall response rate (ORR) at Day 28
研究概览
简要总结
The purpose of this study is to determine the efficacy and safety of combined Ruxolitinib With Corticosteroids as First Line Therapy for the severe acute GVHD (graft-versus-host disease )
详细描述
Acute graft-versus-host disease (GVHD) is treated with systemic corticosteroid immunosuppression as first line therapy. Many patients with severe acute GVHD do not respond to primary therapy, high-dose systemic corticosteroids; therefore, survival for those patients remains particularly poor. Here we determine the efficacy and safety of combined Ruxolitinib With Corticosteroids as First Line Therapy for the Treatment of severe acute GVHD.
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
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosed with hematological diseases.
- •Have undergone first allogeneic hematopoietic stem cell transplantation (allo-HSCT) from any donor source using bone marrow, peripheral blood stem cells, or cord blood for hematologic malignancies.
- •New onset of severe acute GVHD within 100 days post-transplantation.
排除标准
- •Recipients of second allogeneic stem cell transplant.
- •Acute GVHD induced by donor lymphocyte infusion, interferon.
- •Received first line aGVHD treatment before enrollment.
- •Overlap GVHD syndrome.
- •Pregnant or breast-feeding women.
- •Absolute neutrophil count (ANC) <0.5×10e9/L or platelet count (PLT) < 20×10e9/L.
- •Serum creatinine > 2.0 mg/dL or creatinine clearance < 40 mL/min measured or calculated by Cockroft-Gault equation.
- •Uncontrolled infection.
- •Human immunodeficiency virus infection.
- •Active hepatitis b virus, hepatitis C virus infection and need antivirus treatment.
- •Subjects with evidence of relapsed primary disease, or subjects who have been treated for relapse after the allo-HSCT was performed, or graft rejection.
- •Allergic history to Janus kinase inhibitors.
- •Severe organ dysfunction unrelated to underlying GVHD, including:
- •(1)Cholestatic disorders or unresolved veno-occlusive disease of the liver (defined as persistent bilirubin abnormalities not attributable to GVHD and ongoing organ dysfunction).
- •(2)Clinically significant or uncontrolled cardiac disease including unstable angina, acute myocardial infarction within 6 months from Day 1 of study drug administration, New York Heart Association Class III or IV congestive heart failure, circulatory collapse requiring vasopressor or inotropic support, or arrhythmia that requires therapy.
- •(3)Clinically significant respiratory disease that requires mechanical ventilation support or 50% oxygen.
- •14.Received Janus kinase inhibitor therapy after allo-HSCT for any indication. 15.Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the subject; or interfere with interpretation of study data.
研究组 & 干预措施
Ruxolitinib Arm
Ruxolitinib(5mg/d) combined with Methylprednisolone(1mg/kg)
干预措施: Ruxolitinib and Methylprednisolone (Drug)
Comparator arm
Methylprednisolone (2mg/kg)
干预措施: Methylprednisolone (Drug)
Ruxolitinib Arm
Ruxolitinib(5mg/d) combined with Methylprednisolone(1mg/kg)
干预措施: Methylprednisolone (Drug)
结局指标
主要结局
Overall response rate (ORR) at Day 28
时间窗: Day 28 after treatment
Defined as the proportion of participants demonstrating a complete response (CR), and partial response (PR).
次要结局
- Six-month duration of response(Six-month after treatment)
- Ninety-day duration of response(Day 90 after treatment)
- Nonrelapse mortality (NRM)(1 year after treatment)
- Cumulative incidence of relapse(1 year after treatment)
- Disease-free survival (DFS)(1 year after treatment)
- GVHD-free and relapse-free survival (GRFS)(1 year after treatment)
- recurrence of aGVHD(1 year after treatment)
- Failure-free survival(1 year after treatment)
研究者
Daihong Liu
Chief Physician
Chinese PLA General Hospital