A Clinical Study to Evaluate the Safety and Efficacy of Unrelated Umbilical Cord Blood Microtransplantation in Patients With Higher-risk Myelodysplastic Syndromes
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Complete response (CR) rate
研究概览
简要总结
This study aimed to evaluate the safety and efficacy of unrelated umbilical cord blood microtransplantation in the treatment of above-mentioned MDS patients by observing the factors related to the efficacy and adverse reactions.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with MDS with WHO criteria through bone marrow morphology, histochemistry, immunophenotyping, pathological testing, etc. Patients can be newly diagnosed, recurrent or unresponsive, with International prognostic scoring system (IPSS-R) score> 3.5
- •Patients aged 14-80, gender and race are not limited;
- •Karnofsky score ≥ 60%, Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;
- •Expected survival time ≥ 3 months;
- •The examination results meet the following requirements:
- •ALT and AST ≤ 3 × Upper limit of normal value (ULN); Total bilirubin ≤ 3 × ULN; Creatinine ≤ 2 × ULN or creatinine clearance rate ≥ 40mL/min; The left ventricular ejection fraction (LVEF) measured by echocardiography or multigated acquisition (MUGA) scanning is within the normal range (>50%);
- •The recipient and selected donor should be 0-3/10 HLA-matched with same blood type;
- •Patients who voluntarily participate in this clinical study and have signed an informed consent.
排除标准
- •Patients who have suffered from malignant tumors;
- •Patients have suffered from hematopoietic failure after chemotherapy, and have undergone ineffective blood transfusion with unknown cause;
- •Patients who have undergone Class II or above surgery within 4 weeks prior to enrollment;
- •Suffering from life-threatening diseases other than MDS;
- •Allergic to the drugs in the research;
- •Patient with severe cardiac insufficiency, including uncontrolled or symptomatic arrhythmia, congestive heart failure, myocardial infarction within 6 months, or any grade 3 (moderate) or grade 4 (severe) heart disease;
- •Patients with test positive for HIV, HCV or HBV;
- •Stroke or intracranial hemorrhage occurred within 6 months prior to enrollment;
- •Warfarin or vitamin K antagonists (such as phenprocoumarin) is necessary for anticoagulation;
- •Patients with mental illnesses or cognitive impairments;
- •Patients have participated within the month prior to enrollment or patients are currently participating in other clinical trials;
- •There are other conditions that the investigators consider inappropriate for inclusion.
研究组 & 干预措施
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation
Intermittent infusion of HLA mismatched or incompletely matched granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells (G-PBSC) during routine chemotherapy without the use of immunosuppressants, abbreviated as "microtransplantation"
干预措施: Unrelated Umbilical Cord Blood (Biological)
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation
Intermittent infusion of HLA mismatched or incompletely matched granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells (G-PBSC) during routine chemotherapy without the use of immunosuppressants, abbreviated as "microtransplantation"
干预措施: Venetoclax (Drug)
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation
Intermittent infusion of HLA mismatched or incompletely matched granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells (G-PBSC) during routine chemotherapy without the use of immunosuppressants, abbreviated as "microtransplantation"
干预措施: Decetabine (Drug)
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation
Intermittent infusion of HLA mismatched or incompletely matched granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells (G-PBSC) during routine chemotherapy without the use of immunosuppressants, abbreviated as "microtransplantation"
干预措施: Azacitidine (Drug)
结局指标
主要结局
Complete response (CR) rate
时间窗: 28±7 days
CR rate is defined as the percentage of patients who met the following conditions: Myelogram: naïve cells less than 5% (at least 200 nucleated cells); Hemogram: absolute neutrophil count \> 1.0×10\^9/L, platelet count \> 100×10\^9/L; Clinical diagnosis: no symptoms and signs caused by leukemia infiltration, and patients do not rely on blood transfusion.
Hematopoietic recovery time
时间窗: 28±7 days
The time of absolute neutrophil count\>0.5×10\^9/L and platelet count \>30×10\^9/L for 3 consecutive days.
次要结局
- Time to Progression（TTP）(1 year)
- Disease Free Survival（DFS）(1 year)
- Overall Survival（OS）(1 year)
- Early mortality rate(3 months)
研究者
Xiaoyu Zhu
Principal Investigator
Anhui Provincial Hospital