Undersøgelse af CFI-400945 fumarat hos patienter med recidiverende eller refraktær AML eller MDS
En åben-label, dosiseskalering, sikkerhed og farmakokinetisk undersøgelse af CFI-400945 fumarat administreret oralt til patienter med recidiverende eller refraktær akut myeloid leukæmi eller myelodysplastisk syndrom
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.
Studieoversigt
Status
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 1
Kontakter og lokationer
Studiesteder
-
-
Ontario
-
Hamilton, Ontario, Canada, L8V 1C3
- Juravinski Hospital and Cancer Centre
-
Toronto, Ontario, Canada, M5G 2M9
- Princess Margaret Cancer Centre
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
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.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: 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. |
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. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Evaluation of the frequency and severity of treatment-emergent adverse events in patients
Tidsramme: 5 years
|
This will be done to assess safety and tolerability of CFI-400945 fumarate |
5 years
|
|
Highest tolerated dose of CFI-400945 fumarate
Tidsramme: 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. |
5 years
|
|
Recommended phase 2 dose of CFI-400945 fumarate
Tidsramme: 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 |
5 years
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Number of participants with response to treatment
Tidsramme: 5 years
|
Patients responses will be assess using the International Working Group response criteria for AML and MDS. |
5 years
|
Samarbejdspartnere og efterforskere
Efterforskere
- Ledende efterforsker: Karen Yee, M.D., Princess Margaret Cancer Centre
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- CFI-400945-CL-002
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
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