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

University Health Network, Toronto

Status and phase

Completed

Phase 1

Conditions

Relapsed Cancer

Refractory Cancer

Myelodysplastic Syndromes

Acute Myeloid Leukemia

Treatments

Drug: CFI-400945 Fumarate

Study type

Interventional

Funder types

Other

Identifiers

NCT03187288

CFI-400945-CL-002

Details and patient eligibility

About

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.

Full 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.

Enrollment

13 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.