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Trial record 1 of 1 for:    NCT03187288
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Study of CFI-400945 Fumarate in Patients With Relapsed or Refractory AML or MDS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03187288
Recruitment Status : Recruiting
First Posted : June 14, 2017
Last Update Posted : April 2, 2021
Information provided by (Responsible Party):
University Health Network, Toronto

Tracking Information
First Submitted Date  ICMJE June 12, 2017
First Posted Date  ICMJE June 14, 2017
Last Update Posted Date April 2, 2021
Actual Study Start Date  ICMJE May 25, 2018
Estimated Primary Completion Date April 2, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 30, 2021)
  • Evaluation of the frequency and severity of treatment-emergent adverse events in patients [ Time Frame: 5 years ]
    This will be done to assess safety and tolerability of CFI-400945 fumarate
  • Highest tolerated dose of CFI-400945 fumarate [ Time Frame: 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 [ Time Frame: 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
Original Primary Outcome Measures  ICMJE
 (submitted: June 12, 2017)
  • Incidence of treatment emergent adverse events [ Time Frame: 5 years ]
  • Highest tolerated dose of CFI-400945 fumarate [ Time Frame: 5 years ]
  • Recommended phase 2 dose of CFI-400945 fumarate [ Time Frame: 5 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 12, 2019)
Number of participants with response to treatment [ Time Frame: 5 years ]
Patients responses will be assess using the International Working Group response criteria for AML and MDS.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 12, 2017)
Number of participants with response to treatment [ Time Frame: 5 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Study of CFI-400945 Fumarate in Patients With Relapsed or Refractory AML or MDS
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description

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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Acute Myeloid Leukemia
  • Myelodysplastic Syndromes
  • Relapsed Cancer
  • Refractory Cancer
Intervention  ICMJE Drug: CFI-400945 Fumarate
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.
Study Arms  ICMJE Experimental: 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.
Intervention: Drug: CFI-400945 Fumarate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 12, 2017)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 4, 2022
Estimated Primary Completion Date April 2, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Karen Yee, M.D. 416-946-4495
Listed Location Countries  ICMJE Canada
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT03187288
Other Study ID Numbers  ICMJE CFI-400945-CL-002
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party University Health Network, Toronto
Study Sponsor  ICMJE University Health Network, Toronto
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Karen Yee, M.D. Princess Margaret Cancer Centre
PRS Account University Health Network, Toronto
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP