A Prospective, Open Label, Dose-escalation, SAD/MAD, Multicenter, 24weeks, Phase I/IIa Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamics of Investigational Product (VM-001) in Patients With Graft-versus-host Disease(GvHD)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 12
- 主要终点
- Maximum Tolerated Dose (MTD) of VM-001 [ Time Frame: 1 week ]
研究概览
简要总结
The present study is multi center, phase I/IIa clinical trial to evaluate the safety and pharmacodynamics of investigational Product (VM-001) in patients with graft-versus-host disease(GvHD). A total of 12 subjects (Part 1 SAD[3 cohort(2 subjects/cohort)], Part 2 MAD[2 cohort(3subjects/cohort)]) are recruited.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 19 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men and women ≥19 years of age
- •Acute GvHD or chronic GvHD after hematopoietic stem cell transplant
- •Subjects who no longer have available standard treatment.
- •ANC≥1,000cells./mm^3
- •AST, ALT, total bilirubin less than 3 times the upper limit of normal
- •serum creatinine less than 1.5 times the upper limit of normal
排除标准
- •Received an anti-thymocyte globulin(ATG) within 14 days before enrollment
- •FCV or FEV less than 70%
- •Any uncontrolled infection or active infection requiring ongoing systemic treatment
- •Received an investigational agent within 6 months before enrollment.
- •Evidence of bleeding diathesis or coagulopathy.
- •Active hepatitis C virus (HCV) or hepatitis B virus (HBV)
- •Breastfeeding or pregnant.
研究组 & 干预措施
Part 1 Single Ascending Dose, Cohort 1
Cohort 1 : 1X10^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP. If CTCAE grade 3 or higher adverse drug events (ADR) do not occur in the two subjects: Begin enrollment for Cohort 2.
干预措施: VM-001 1X10^6 cells/kg (Biological)
Part 1 Single Ascending Dose, Cohort 2
Cohort 2 : 3X10^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP. If CTCAE grade 3 or higher adverse drug events (ADR) do not occur in the two subjects: Begin enrollment for Cohort 3.
干预措施: VM-001 3X10^6 cells/kg (Biological)
Part 1 Single Ascending Dose, Cohort 3
Cohort 3 : 5X10^6 cells/kg The safety of 2 subjects is evaluated for 1 week after single dose of IP.
干预措施: VM-001 5X10^6 cells/kg (Biological)
Part 2 Multiple Ascending Dose, Cohort 1
Cohort 1 : two doses in total, 1X10^6 cells/kg per dose, weekly The safety of 3 subjects is evaluated for 4 week after two dose of IP. If CTCAE grade 3 or higher adverse drug events (ADR) do not occur in the three subjects: Begin enrollment for Cohort 2.
干预措施: VM-001 1X10^6 cells/kg two dose (Biological)
Part 2 Multiple Ascending Dose, Cohort 2
Cohort 2 : four doses in total, 1X10^6 cells/kg per dose, weekly The safety of 3 subjects is evaluated for 8 week after two dose of IP.
干预措施: VM-001 1X10^6 cells/kg four dose (Biological)
结局指标
主要结局
Maximum Tolerated Dose (MTD) of VM-001 [ Time Frame: 1 week ]
时间窗: 7 day cycle
First 7-day treatment dose limiting toxicities (DLT) graded according to CTCAE in the MTD-determining population in Phase 1 based on the number of participants with adverse effects as measure of tolerability at various dose levels
次要结局
未报告次要终点