A Study of CFI-400945 Fumarate in Patients With Advanced Cancer

Official Title

An Open Label, Dose Escalation, Safety, and Pharmacokinetic Study of CFI-400945 Fumarate Administered Orally to Patients With Advanced Cancer


This is a phase 1 study to test different doses of a new investigational drug called CFI-400945 to see which dose is safer in people. This study will also look at the safety of CFI-400945 and to study its effects on patients with advanced cancers. This drug has been tested in animals but not yet in people. CFI-400945 is an oral (taken by mouth) drug that works by blocking polo-like kinase 4 (PLK4) from working. PLK4 is a protein that is important in regulating cell growth and division and cell death. Many tumours are shown to make too much PLK4. When there is too much PLK4 produced, it is believed to lead to uncontrolled cancer cell growth and division. Therefore, by blocking this protein from working, it is believed to stop tumours growing or shrink them.

Trial Description

Primary Outcome:

  • Highest dose level that does not lead to unacceptable toxicity in two or more patients in a dosing group over a range of doses and schedules
Secondary Outcome:
  • Pharmacokinetic profile of CFI-400945 fumarate (please see description below) over a range of doses and schedules
  • Number of patients with evidence of benefit over a range of doses and schedules
  • Number of side effects occurring and severity
  • Evaluate the genomic alterations and other molecular features which are associated with response and/or clinical benefit with CFI-400945-CL fumarate treatment
  • to evaluate pharmacodynamics effects relative to CFI-400945 fumarate at MTD
  • Determine evidence of benefit in cancer patients when CFI-400945 fumarate is administered orally at the MTD (expansion)
  • To evaluate possible mechanisms of resistance to CFI-400945 fumarate at MTD
  • Determine recommended Phase 2 dose for CFI-400945 fumarate
All participants will receive CFI-400945. At the beginning of the study, participants are given a low dose of CFI-400945 and are watched very closely to see what side effects they have and to make sure the side effects are not severe. If the side effects are not severe, then more participants are asked to join the study and are given a higher dose of study drug. Participants joining the study later on will get higher doses of study drug than participants who join earlier. This will continue until the highest dose of study drug that can be taken without severe side effects is found (called maximum tolerated dose). Doses higher than that will not be given. After the best dose of study drug is found, additional participants will be asked to join the study and will be given the study drug at the maximum tolerated dose to further test the safety and the drug at that dose.

View this trial on ClinicalTrials.gov

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Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society