A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Overall response rate (ORR)
研究概览
简要总结
A study to evaluate the safety, efficacy, and pharmacokinetics of MegaLT in treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation.
详细描述
A single-center, open-label, dose-escalation study to assess the safety, efficacy, and pharmacokinetics of MegaLT for treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation.
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
- 4 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age between 4 and 75 years (inclusive) at screening; gender is not restricted;
- •Diagnosed with refractory thrombocytopenia following radiotherapy/chemotherapy or transplantation;
- •Adequate organ function：Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) < 2.5*upper limit of normal (ULN); Serum creatinine < 1.5* ULN; Total bilirubin level < 1.5* ULN; Coagulation function: International Normalized Ratio (INR) ≤ 1.5* ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5*ULN; Hemoglobin ≥ 60 g/L, or hemoglobin maintained at this level after transfusion; Absolute neutrophil count (ANC) ≥ 1.0×10^9/L;
- •Eastern Cooperative Oncology Group (ECOG) performance status of 0-2;
- •Voluntarily participating in the clinical trial, with full understanding of the trial details and having signed the informed consent form.
排除标准
- •Patients with any malignant tumor relapse;
- •Pregnant or breastfeeding women;
- •Life expectancy less than 3 months;
- •Severe infections or severe concurrent conditions such as heart, liver, lung, kidney, neurological, or metabolic diseases;
- •History of severe thrombotic events or known thrombotic risk factors. Exceptions: Participants for whom the potential benefits of the study, as determined by the investigator, outweigh the potential risks of thromboembolic events;
- •Uncontrolled infectious or other serious diseases, including but not limited to infections (e.g., HIV positive), congestive heart failure, unstable angina, arrhythmias, psychiatric disorders, or conditions that limit the ability to meet study requirements or pose unpredictable risks as judged by the primary physician;
- •Active hepatitis B or C infection;
- •Refractory thrombocytopenia post-transplant with Grade III-IV acute graft-versus-host disease (GVHD) [according to NIH standards] or severe chronic GVHD (NIH 2014 standards);
- •History of organ transplantation or planned organ transplantation (excluding hematopoietic stem cell transplantation);
- •Participation in another clinical study within 30 days prior to the baseline visit, involving any investigational drug or device; observational studies are allowed;
- •Any other conditions deemed by the investigator to make the participant unsuitable for the clinical trial.
研究组 & 干预措施
MegaLT injection
Dosage Form: ex vivo three-dimensional induced umbilical cord blood mononuclear cells differentiated megakaryocyte injection (MegaLT injection).
Administration Route: Intravenous Infusion Regimen: Single or Multiple Infusions
干预措施: MegaLT injection (Biological)
结局指标
主要结局
Overall response rate (ORR)
时间窗: 4 weeks after initial infusion
Percentage of participants achieved Complete response(CR) or Partial response(PR)
Adverse Event(AE)
时间窗: From the date of initial infusion to 1 year after initial infusion
Number of treatment-related adverse events as assessed by CTCAE v4.0
The probability of GVHD-free, relapse-free survival(GRFS)
时间窗: From the date of initial infusion to 1 year after initial infusion
The composite endpoint of GRFS was defined as the first events occurring after transplantation among Grade III to IV aGVHD, moderate to severe cGVHD, relapse, or death for any reason.
Incidence of bleeding events
时间窗: From the date of initial infusion to 1 year after initial infusion
The incidence of bleeding events after initial infusion
The cumulative incidence and grade of graft-versus-host disease (GVHD) including acute and chronic GVHD
时间窗: From the date of initial infusion to 1 year after initial infusion
The occurrence incidence and grade of GVHD after transplantation
次要结局
- Overall response rate (ORR)(1, 2 and 8 weeks after initial infusion)
- Complete remission (CR) rate(1, 2, 4 and 8 weeks after initial infusion)
- partial response rate (PR)(1, 2, 4 and 8 weeks after initial infusion)
- Changes in platelet counts(before treatment and at 1, 2, 4, and 8 weeks after treatment)
- Median time of platelet count ≥ 50×10^9/L(1 year after initial infusion)
- Median time of platelet count ≥ 100×10^9/L(1 year after initial infusion)
- Total platelet transfusion during 4 weeks of treatment(4 weeks after initial infusion)
- neutrophil reconstitution(1 year after initial infusion)
- erythroid reconstitution(1 year after initial infusion)
- Megakaryocyte levels in bone marrow smears(before treatment and 2 and 4 weeks after treatment)