An Open-Label, Dose Escalation, Safety and Pharmacokinetic Study of CFI-400945 Fumarate Administered Orally to Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- Evaluation of the frequency and severity of treatment-emergent adverse events in patients
研究概览
简要总结
This is a phase 1 study of investigational drug CFI-400945 in patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome. The purpose of this phase 1 study is to see how safe and tolerable the study drug is and to determine the best dose (maximum tolerated dose or recommended phase 2 dose) that can be given in this patient population.
详细描述
Participants will be screened prior to the start of the study drug for eligibility.
Eligible participants will take CFI-400945 by mouth, once a day, every day of each 28 day cycle. Participants will be asked to keep a study drug diary.
While receiving the study drug, participants will have standard tests and procedures done for safety purposes.
Procedures for research purposes include bone marrow aspirate and additional blood collection for biomarker research, and additional blood samples for pharmacokinetic research.
When participants stop the study drug permanently for any reason, they will be asked to have an End of Treatment Visit and be followed for safety purposes.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 years of age or older
- •Relapsed and/or refractory myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that meets certain criteria for prior treatments
- •Have acceptable circulating blasts count
- •Have clinically acceptable laboratory blood and urine test results
- •Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
- •Be able to swallow oral medications
- •Have a life expectancy of 3 months or more
- •Agree to use highly effective means of contraception during a defined period
- •Negative serum pregnancy test before the start of the study drugs
- •Have the ability to understand the requirements of the study, provide written informed consent which includes authorization for release of protected health information, abide by the study restrictions, provide a blood and bone marrow sample for genetic testing and agree to return for the required assessments
排除标准
- •Have received cancer therapies within 14 days or 5 half-lives (whichever is shorter) prior to first dose of study drug or have not recovered from toxicities from prior treatments
- •Not recovered from toxicities related to allogeneic transplant
- •Known active extramedullary central nervous system (CNS) AML
- •Secondary cancer needing therapy with exceptions
- •Known active human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) infection
- •Known significant mental illness or other condition that may affect the ability to follow the requirements of the study
- •Have a chronic infection
- •Have uncontrolled severe hypertension
- •Have symptomatic congestive heart failure
- •Have active angina pectoris or recent myocardial infarction
- •Have chronic atrial fibrillation or unacceptable QTc
- •Have had major surgery within 21 days of starting therapy
- •Have additional uncontrolled serious medical or psychiatric illness
- •Have any medical condition that would affect taking the study drug
- •Receiving treatment with full dose warfarin
- •Receiving treatment with drugs not allowed in this study
- •Women who are pregnant or lactating.
研究组 & 干预措施
CFI-400945
CFI-400945 will be given by mouth at 64,96,128,160,192 or 224 mg/day, everyday until intolerable side effects or disease progression.
干预措施: CFI-400945 Fumarate (Drug)
结局指标
主要结局
Evaluation of the frequency and severity of treatment-emergent adverse events in patients
时间窗: 5 years
This will be done to assess safety and tolerability of CFI-400945 fumarate
Highest tolerated dose of CFI-400945 fumarate
时间窗: 5 years
The Maximum Tolerated Dose MTD is defined as the highest dose level that does not lead to unacceptable toxicity in two or more patients in a dosing cohort.
Recommended phase 2 dose of CFI-400945 fumarate
时间窗: 5 years
Following completion of dosing of at least 1 cycle for all patients enrolled the Recommended Phase 2 Dose (RP2D) will be determined.It will be based upon the MTD established during dose escalation and its comprehensive outcome
次要结局
- Number of participants with response to treatment(5 years)