A Pilot Clinical Trial of Motixafortide for Measurable Residual Disease (MRD) Sensitization in Acute Myeloid Leukemia (AML)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Efficacy of motixafortide on measurable residual disease (MRD) levels
研究概览
简要总结
This is a pilot phase I study evaluating the effect of motixafortide on determination of measurable residual disease (MRD) level in patients with acute myeloid leukemia (AML) who have completed induction treatment. Consenting and eligible patients will undergo standard of care (SOC) bone marrow and peripheral blood assessments with SOC MRD assays, followed by a single injection of motixafortide. Ten to 14 hours after injection, the patient will undergo peripheral blood collection for the same applicable MRD tests
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 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
- •Diagnosed with acute myeloid leukemia (AML), excluding APL, treated with 1-2 cycles of front-line chemotherapy.
- •Achieved CBC parameters compatible with complete remission as defined by ELN
- •Planning to undergo a standard of care blood draw and bone marrow assessment with SOC MRD assays, including morphology, flow cytometry for MRD, NGS panels for MRD, and PCR tests for MRD as applicable.
- •At least 18 years of age.
- •ECOG performance status ≤ 2
- •Life expectancy > 3 months.
- •Adequate organ function as defined below:
- •Total bilirubin ≤ 2.0 x IULN
- •AST(SGOT)/ALT(SGPT) ≤ 5.0 x IULN
- •Creatinine clearance > 30 mL/min by Cockcroft-Gault
- •Ability to understand and willingness to sign an IRB approved written informed consent document.
排除标准
- •Evidence of more than 5% blasts in in the peripheral blood by manual differential within 5 days prior to study enrollment.
- •Prior history of allogeneic stem cell transplant.
- •Prior or concurrent malignancy whose natural history has the potential to interfere with the safety or efficacy assessment of the investigational regimen. Patients with prior or concurrent malignancy that does NOT meet that definition are eligible for this trial
- •Currently receiving any other investigational agents.
- •A history of allergic reactions attributed to compounds of similar chemical or biologic composition to motixafortide.
- •Uncontrolled intercurrent illness including, but not limited to: ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia. Patients with a known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Function Classification; to be eligible for this trial, patients should be a class 2B or better.
- •Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.
- •HIV-infected if not on effective anti-retroviral therapy with undetectable viral load for 6 months. Patients with HIV who are receiving effective anti-retroviral therapy and have had an undetectable viral load for at least 6 months are eligible. HIV testing not required in the absence of known history of infection.
- •Evidence of chronic hepatitis B virus (HBV) that is detectable on suppressive therapy. Patients with evidence of chronic HBV infection with undetectable HBV viral load on suppressive therapy are eligible. HBV testing not required in the absence of known history of infection.
- •History of hepatitis C virus (HCV) infection that has not been cured or that has a detectable viral load. Patients with a history of HCV that has been treated and cured are eligible. Patients with HCV infection who are currently on treatment and have an undetectable HCV viral load are eligible. HCV testing not required in the absence of known history of infection.
研究组 & 干预措施
Motixafortide Injection
Consenting and eligible patients will bone marrow and peripheral blood testing assessments along with measurable residual disease (MRD) assays. Following completion of assessments, patients will receive a single injection of Motixafortide. The following day, 10-14 hours after the injection, patients will repeat the peripheral blood testing for MRD tests. Investigators will follow up with patients every 4 months for 18 months after study treatment.
干预措施: Motixafortide (Drug)
结局指标
主要结局
Efficacy of motixafortide on measurable residual disease (MRD) levels
时间窗: Day 1 before motixafortide and Day 2 (estimated total time is 2 days)
The efficacy of motixafortide on MRD levels will be measured as the proportion of patients who have at least one of the following, as measured in peripheral blood, before versus after motixafortide: MRD negative to MRD positive conversion, or the 5% absolute increase in MRD level by flow cytometry, or leukemia defining polymerase-chain reaction (PCR) transcript (For fusion-driven leukemias or NPM1 and FLT3-ITD-mutated leukemias) or 5% increase in the VAF of recurrent leukemia associated gene mutations by next-generation sequencing (NGS).
次要结局
- Proportion of patients changing from negative to positive MRD levels(Day 1 before motixafortide and Day 2 (estimated total time is 2 days))
- Percentage change in variant allele frequency (VAF) levels by next-generation sequencing (NGS) using error-corrected sequencing (MRD-Seq)(Day 1 before motixafortide and Day 2 (estimated total time is 2 days))
- Percentage change in VAF levels by NGS using MRD-Seq between bone marrow and peripheral blood assessments(Day 1 before motixafortide and Day 2 (estimated total time is 2 days))
- Percentage change in detectable transcript levels by polymerase chain reaction (PCR)(Day 1 before motixafortide and Day 2 (estimated total time is 2 days))
- Relapse-free survival (RFS)(From Day 1 through completion of follow-up (estimated total time is 18 months))
- Time to next line of therapy(From Day 1 through completion of follow-up (estimated total time is 18 months))
- Overall survival (OS)(From Day 1 through completion of follow-up (estimated total time is 18 months))