A Phase 1/2a, Open-Label, Dose Escalation, and Dose Expansion Study to Assess the Safety and Efficacy of GDX012 in Patients With Relapsed or Refractory Acute Myeloid Leukemia
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 16
- 试验地点
- 20
- 主要终点
- Number of Participants With Adverse Events (AEs)
研究概览
简要总结
GDX012 is a novel cell therapy developed for the treatment of certain types of cancer, including Acute Myeloid Leukemia (AML). The main aims of the study are to learn how safe GDX012 is, how treatment with GDX012 is tolerated and to determine the best dose of GDX012.
详细描述
The drug being tested in this study is called GDX012. GDX012 is being tested to evaluate the safety and tolerability in adult participants with AML.
The study will enroll approximately 53 patients in two phases, dose escalation and dose expansion.
During Phase 1 (sequential dose escalation), participants will be assigned to one of the following treatment groups each consisting of 3 to 6 participants to receive GDX012 at one of the three dose levels:
- GDX012 Dose 1
- GDX012 Dose 2
- GDX012 Dose 3
Upon completion of Phase 1, 1 to 2 dose levels will be selected for Phase 2a of the study. At the completion of Phase 2a of the study a single dose may be selected by the sponsor and investigators as the recommended phase 2 dose (RP2D) for future study.
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
- •Total body weight of ≥40 kg.
- •Must have pathologically confirmed relapsed or refractory acute myeloid leukemia (R/R AML) including:
- •Relapsed AML is defined as ≥5% blasts in the bone marrow (BM) or peripheral blood at any time after achieving a CR, CRh, Cri, or MLFS.
- •Refractory AML is defined as failure to achieve a CR, CRh, Cri, or MLFS after 1 of the following regimens:
- •i. Two courses of intensive induction chemotherapy. ii. At least 2 cycles of hypomethylating agent (HMA) or low-dose, cytarabine-based combination regimen.
- •iii. At least 4 cycles of HMA monotherapy.
- •During dose escalation, participants must be ineligible for hematopoietic stem cell transplantation (HSCT).
- •Must have an anticipated life expectancy of >3 months before lymphodepletion.
- •Eastern Cooperative Oncology Group (ECOG) performance status of 0 to
- •Participants must have adequate renal, cardiac, hepatic, pulmonary and bone marrow function as defined by the protocol.
排除标准
- •Diagnosis of acute promyelocytic leukemia.
- •Has received or plans to receive any of the excluded therapy/treatment within the specified timeframe before lymphodepleting chemotherapy as defined by the protocol.
- •Prior allogeneic HSCT within 3 months of signing informed consent form (ICF) or with ongoing requirement for systemic graft-versus-host therapy.
- •Active central nervous system (CNS) involvement.
- •History of malignancy other than non-melanoma skin cancer or carcinoma in situ (eg. cervix, bladder, breast) low grade prostate cancer without treatment requirement unless in remission without treatment for ≥2 years.
研究组 & 干预措施
Phase 1: Dose Escalation of GDX012
Participants will receive GDX012 weight-based dose as intravenous (IV) infusion on Day 1 of Phase 1 after lymphodepleting chemotherapy. Three dose levels of GDX012 will be tested in Phase 1. Some participants may be eligible for a second dose.
干预措施: GDX012 (Drug)
Phase 1: Dose Escalation of GDX012
Participants will receive GDX012 weight-based dose as intravenous (IV) infusion on Day 1 of Phase 1 after lymphodepleting chemotherapy. Three dose levels of GDX012 will be tested in Phase 1. Some participants may be eligible for a second dose.
干预措施: Chemotherapy Agents (Drug)
Phase 2a: GDX012
Participants will receive GDX012 (weight-based) IV infusion at pre-selected one or two dose levels from Phase 1, on Day 1 after lymphodepleting chemotherapy. Some participants may be eligible for a second dose.
干预措施: GDX012 (Drug)
Phase 2a: GDX012
Participants will receive GDX012 (weight-based) IV infusion at pre-selected one or two dose levels from Phase 1, on Day 1 after lymphodepleting chemotherapy. Some participants may be eligible for a second dose.
干预措施: Chemotherapy Agents (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs)
时间窗: Up to 14 months
An adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug.
Number of Participants With Dose Limiting Toxicities (DLTs)
时间窗: Up to 1 month
Maximum Tolerated Dose (MTD) of GDX012
时间窗: Up to 1 month
次要结局
- Number of Participants With Measurable Residual Disease (MRD) Negative Status as Determined by Flow Cytometry(Up to 14 months)
- Duration of Response (DOR)(Up to 14 months)
- Number of Participants With Disease Response(Up to 14 months)
- Overall Survival (OS)(Up to 14 months)
- Event-free Survival (EFS)(Up to 14 months)