A Phase 2a, Open-Label, Two Stage Study: Stage A: Dose-Range Finder Study to Assess the Safety and Efficacy of Two Doses of Nerofe and Two Doses of Nerofe in Combination With Doxorubicin in Subjects With Acute Myelogenous Leukemia or High Risk Myelodysplastic Syndrome (AML/High Risk MDS). Stage B: Dose Confirmation Study to Assess the Safety and Efficacy of Nerofe or Nerofe in Combination With Doxorubicin in Subjects With AML/ High Risk MDS
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 2
- 主要终点
- Assessing change in IWG Criteria to evaluate response to Nerofe treatment (with or without Doxorubicin) for AML subjects
研究概览
简要总结
This is a Phase 2a, Open-label, one arm study in which the eligible patients will be treated with IV Nerofe, three times a week in 28 days cycles (up to 12 cycles).
Evaluation will include safety procedures, blood level of study drug in certain time points, immune system response and tests checking the mechanism of the drug action.
详细描述
Nerofe is a first-in-class hormone peptide with cancer suppressive properties. It works in three mechanisms of action. The purpose of this research is to study Nerofe's effect in patients diagnosed with AML and MDS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Nerofe 48mg/m2
48mg/m2 IV Nerofe - three times a week
干预措施: Nerofe (Drug)
Nerofe 96mg/m2
96mg/m2 IV Nerofe - three times a week
干预措施: Nerofe (Drug)
Nerofe 48mg/m2 + Doxorubicin 10mg/m2
48mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week
干预措施: Nerofe (Drug)
Nerofe 48mg/m2 + Doxorubicin 10mg/m2
48mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week
干预措施: Doxorubicin (Drug)
Nerofe 96mg/m2 + Doxorubicin 10mg/m2
96mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week
干预措施: Nerofe (Drug)
Nerofe 96mg/m2 + Doxorubicin 10mg/m2
96mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week
干预措施: Doxorubicin (Drug)
结局指标
主要结局
Assessing change in IWG Criteria to evaluate response to Nerofe treatment (with or without Doxorubicin) for AML subjects
时间窗: At end of Cycles 2, 4, 6, 8, 10, 12 (Cycle length 28 Days)
Bone Marrow samples and CBC will be done every 2 cycles
Assessing changes in R-IPSS (Revised International Prognostic Scoring System) Score to evaluate response to Nerofe treatment (with or without Doxorubicin) for MDS patients. A calculation of several variables.
时间窗: At end of Cycles 2, 4, 6, 8, 10, 12 (Cycle length 28 Days)
Measuring cytogenetic abnormalities. Range from very good (0) to very poor (4)
Safety as determined by frequency, nature and severity of adverse events
时间窗: 13 months
Per CTCAE v4.0
次要结局
- Pharmacodynamic analysis of changes from baseline in PBMCs' T1/ST2 receptor expression(Every cycle (Cycle length 28 days))
- Pharmacokinetic behavior of Nerofe: Maximum Plasma Concentration (Cmax)(At cycles 1 and 2 (Cycle length 28 days))
- Pharmacokinetic behavior of Nerofe: Tmax(At cycles 1 and 2 (Cycle length 28 days))
- Pharmacodynamic analysis of changes from baseline in levels of circulating cytokines(Every cycle (Cycle length 28 days))
- Pharmacodynamic analysis of changes from baseline in levels of soluble T1/ST2 receptor(Every cycle (Cycle length 28 days))
- Pharmacokinetic behavior of Nerofe: Minimum Plasma Concentration (Cmin)(At cycles 1 and 2 (Cycle length 28 days))
- Pharmacokinetic behavior of Nerofe: Area Under the Curve (AUC)(At cycles 1 and 2 (Cycle length 28 days))