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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
CFI-400945 is an investigational drug that is being look at for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). CFI-400945 is an oral (taken by mouth) drug that blocks Polo-like kinase 4 (PLK4) activity. PLK4 is a protein that is important in regulating cell growth and division and cell death. Many tumors are shown to make too much PLK4. When there is too much PLK4 produced, it is believed to contribute to uncontrolled cancer cell growth and division. Therefore, by blocking this protein from working, it is believed to stop tumors from growing or shrink them.
Juravinski Hospital and Cancer Centre
Hamilton, Ontario, Canada
Princess Margaret Cancer Centre
Toronto, Ontario, Canada
Evaluation of the frequency and severity of treatment-emergent adverse events in patients
This will be done to assess safety and tolerability of CFI-400945 fumarate
Time frame: 5 years
Highest tolerated dose of CFI-400945 fumarate
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.
Time frame: 5 years
Recommended phase 2 dose of CFI-400945 fumarate
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
Time frame: 5 years
Number of participants with response to treatment
Patients responses will be assess using the International Working Group response criteria for AML and MDS.
Time frame: 5 years
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