Evaluation of the Clinical Efficacy and Safety of Recombinant Humanized Anti-CD25 Monoclonal Antibody in Preventing Graft-versus-host Disease After Haploidentical / Matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia: a Prospective, Multicenter, Open-label, Randomized Controlled Clinical Trial
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 396
- 试验地点
- 5
- 主要终点
- Grade III-IV aGVHD
研究概览
简要总结
Graft-versus-host disease (GVHD) is a major factor affecting the efficacy and quality of life of alternative donor transplantation in thalassemia major (TM), severely limiting the clinical application of alternative donor transplantation in TM.The purpose of this clinical trial is to evaluate whether recombinant humanized anti-CD25 monoclonal antibody is effective in preventing GVHD and its safety after haploidentical/matched unrelated donor hematopoietic stem cell transplantation. The main questions it aims to answer are:
- Does recombinant humanized anti-CD25 monoclonal antibody reduce the incidence of GVHD disease after haploidentical/matched unrelated donor hematopoietic stem cell transplantation?
- What medical problems will participants experience when using the recombinant humanized anti-CD25 monoclonal antibody? What is the quality of life after 2 years follow-up? In this clinical trail, participants will be randomly assigned to the intervention group or the control group by researchers in a 2:1 ratio. The intervention group will be given recombinant humanized anti-CD25 monoclonal antibody (1mg/Kg) combined with the standard GVHD prophylaxis after transplantation, while the control group will only receive the standard GVHD prophylaxis. The incidence of GVHD after transplantation in the two groups will be observed. The main evaluation is the clinical efficacy of recombinant humanized anti-CD25 monoclonal antibody in preventing aGVHD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Single (Outcomes Assessor)
盲法说明
The researchers and the subjects wil aware of the grouping and the intervention they received. However, this trail will implement a blind method for the outcomes assessors, who objectively evaluated the outcome indicators without knowing the grouping of the subjects.
入排标准
- 年龄范围
- 3 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients with transfusion-dependent thalassemia;
- •patients who are planning to receive matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) or HLA haploidentical donor hematopoietic stem cell transplantation (HID-HSCT);
- •physical condition score (Lansky/Karnofsky score) ≥ 70%;
- •patients (or legal guardians) voluntarily participate in the study and sign the informed consent form
排除标准
- •patients with HLA-matched hematopoietic stem cell donors and willing to receive HLA-matched hematopoietic stem cell transplantation;
- •patients with known infectious diseases such as hepatitis B, hepatitis C, AIDS, syphilis, human T-lymphotropic virus, etc.;
- •patients with serious active bacterial, viral, fungal, malaria or parasitic infections;
- •patients with autoimmune deficiency diseases;
- •patients with a history of malignant tumors or current malignant tumors;
- •patients with important organ diseases or abnormal laboratory tests, including but not limited to: 1) patients with cirrhosis, liver fibrosis or active hepatitis, and/or abnormal liver function tests (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥2.5×ULN; alkaline phosphatase ≥2.5×ULN); 2) patients with heart disease, or left ventricular ejection fraction (LVEF) <60%, or severe iron deposition in the heart; 3) kidney disease, or blood creatinine ≥1.5×ULN with creatinine clearance <30% of normal level; 4) patients with endocrine dysfunction;
- •patients with uncorrected bleeding disease;
- •patients with severe mental illness (such as severe depression, schizophrenia, etc.) or cognitive dysfunction (dementia, delirium, etc.), which are unable to cooperate with the study;
- •peripheral blood white blood cell (WBC) count <3×10^9/L or platelet count <100×10^9/L;
- •patients having received thalidomide treatment within the past 3 months;
- •patients having received any type of gene and/or cell therapy in the past;
- •patients with severe allergies;
- •female patients who are pregnant, breastfeeding, or planning to become pregnant within 1 year of participating in this trial;
- •patients who are participating in other clinical trials;
- •other situations that are not suitable for participation in this clinical trial as assessed by the investigator.
研究组 & 干预措施
intervention group
The intervention group will receive 4 doses of recombinant humanized anti-CD25 monoclonal antibody (Sunshine Guojian Pharmaceutical(Shanghai) Co.,Ltd.) on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg.
干预措施: recombinant humanized anti-CD25 monoclonal antibody (Drug)
结局指标
主要结局
Grade III-IV aGVHD
时间窗: within 100 days after HID-HSCT/MUD-HSCT
the incidence of grade III-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
次要结局
- Grade II-IV aGVHD(within 100 days after HID-HSCT/MUD-HSCT)
- Chronic graft-versus-host disease (cGVHD)(2 years)
- Overall survival (OS)(2 years)
- Thalassemia-free survival (TFS)(2 years)
- Transplantation-related mortality (TRM)(2 years)
- Transplantation-related complications(2 years)
- Infection(2 years)
- Immune reconstitution(2 years)
- Quality of life(2 years)
- Adverse events(2 years)
研究者
Rongrong Liu
consultant; professor
First Affiliated Hospital of Guangxi Medical University