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

NCT03187288 | Completed Phase 1 DMC

• 1 other identifier: CFI-400945-CL-002
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 2

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.

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndromes; Relapsed Cancer; Refractory Cancer

Outcome Measures

Primary Outcomes (3)

• 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

  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.

  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

  5 years

Secondary Outcomes (1)

• Number of participants with response to treatment

  5 years

Study Arms (1)

CFI-400945

EXPERIMENTAL

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.

Drug: CFI-400945 Fumarate

Interventions

CFI-400945 Fumarate DRUG

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.

CFI-400945

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Health Network, Toronto: lead

Study Sites (2)

Juravinski Hospital and Cancer Centre

Hamilton, Ontario, L8V 1C3, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Recurrence; Neoplasms

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Karen Yee, M.D.

  Princess Margaret Cancer Centre

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2017
• First Posted: June 14, 2017
• Study Start: May 25, 2018
• Primary Completion: June 30, 2021
• Study Completion: June 30, 2021
• Last Updated: August 22, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2)