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Study of CFI-400945 Fumarate in Patients With Relapsed or Refractory AML or MDS

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ClinicalTrials.gov Identifier: NCT03187288
Recruitment Status : Recruiting
First Posted : June 14, 2017
Last Update Posted : August 15, 2018
Sponsor:
Information provided by (Responsible Party):
University Health Network, Toronto

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.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Myelodysplastic Syndromes Relapsed Cancer Refractory Cancer Drug: CFI-400945 Fumarate Phase 1

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : May 25, 2018
Estimated Primary Completion Date : April 2, 2021
Estimated Study Completion Date : April 4, 2022


Arm Intervention/treatment
Experimental: CFI-400945
CFI-400945 will be given by mouth at 64, 72, 96, 128, 176 or 224 mg/day, everyday until intolerable side effects or disease progression.
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.




Primary Outcome Measures :
  1. Incidence of treatment emergent adverse events [ Time Frame: 5 years ]
  2. Highest tolerated dose of CFI-400945 fumarate [ Time Frame: 5 years ]
  3. Recommended phase 2 dose of CFI-400945 fumarate [ Time Frame: 5 years ]

Secondary Outcome Measures :
  1. Number of participants with response to treatment [ Time Frame: 5 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03187288


Contacts
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Contact: Karen Yee, M.D. 416-946-4495 karen.yee@uhn.ca

Locations
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Canada, Ontario
Juravinski Hospital and Cancer Centre Not yet recruiting
Hamilton, Ontario, Canada, L8V 1C3
Contact: Brian Leber, M.D.    905-521-2100 ext 76384      
Princess Margaret Cancer Centre Recruiting
Toronto, Ontario, Canada, M5G 2M9
Contact: Karen Yee, M.D.    416-946-4495      
Sponsors and Collaborators
University Health Network, Toronto
Investigators
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Principal Investigator: Karen Yee, M.D. Princess Margaret Cancer Centre

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Responsible Party: University Health Network, Toronto
ClinicalTrials.gov Identifier: NCT03187288     History of Changes
Other Study ID Numbers: CFI-400945-CL-002
First Posted: June 14, 2017    Key Record Dates
Last Update Posted: August 15, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Neoplasms by Histologic Type
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions