A Prospective, Single-arm Clinical Trial of Prevention of Severe Acute Graft-versus-host Disease After Pediatric Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation Using a daGOAT Model
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Severe aGVHD during 100 days after transplantation according to the MAGIC criteria
研究概览
简要总结
To evaluate the efficacy and safety of ruxolitinib for prophylactic therapy of child patients who are predicted to have a high risk for developing severe acute graftversus-host disease (aGVHD) by the dynamic aGVHD Onset Anticipation Tianjin (daGOAT) model.
详细描述
This study aims to prospectively evaluate the use of the daGOAT model in real-world clinical settings at the Institute of Hematology, Chinese Academy of Medical Sciences (IHCAMS).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- — 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must be ≤ 16 years of age;
- •Patients receiving human leukocyte antigen mismatched and non-cord blood allogeneic hematopoietic stem cell transplantation;
- •Patients who can take oral medication;
- •Patients or their guardians have to sign an informed consent form before the start of the research procedure.
排除标准
- •Tandem transplantation or multiple transplantations;
- •Patients who are allergic to or cannot tolerate ruxolitinib;
- •Mental or other medical conditions that make the patients unable to comply with the research treatment and monitoring requirements;
- •Patients who are pregnant or cannot take appropriate contraceptive measures during treatment;
- •Patients who are ineligible for the study due to other factors, or will bear great risk if participating in the study.
研究组 & 干预措施
The group of daGOAT model prevention
Model-predicted high-risk patients: weight ≤ 25 kg, ruxolitinib, 2.5mg bid po until at least day 60 post-transplant and terminated after day 100; weight > 25 kg, ruxolitinib, 5mg bid po until at least day 60 post-transplant and terminated after day 100. If 'azoles' are taken concomitantly, ruxolitinib will start at half dose. If the patient tolerates ruxolitinib, the dose can be increased to 10mg bid po.
Model-predicted low risk: regular aGVHD prophylactic regimens.
干预措施: Ruxolitinib (Drug)
结局指标
主要结局
Severe aGVHD during 100 days after transplantation according to the MAGIC criteria
时间窗: 100 days after transplantation
Incidence of severe aGVHD after transplantation within 100 days. The medical records for each case wil be reviewed by two or three physicians to confirm the aGVHD diagnosis and grading (according to the MAGIC criteria).
次要结局
- aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria(100 days after transplantation)
- Relapse-free survival rate and relapse rate during 1.5 year after transplantation(Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation)
- Incidence of infections during 1.5 year after transplantation(1.5 year after transplantation)
- Total cost of treatment during 1.5 year after transplantation(1.5 year after transplantation)
- Overall survival during 1.5 year after transplantation(Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation)
- Safety of treatment during 100 days after transplantation according to the Common Terminology Criteria for Adverse Events version 5.0(100 days after transplantation)