Estudo do fumarato CFI-400945 em pacientes com LMA ou SMD recidivante ou refratária
Um estudo aberto, escalonamento de dose, segurança e farmacocinética do fumarato CFI-400945 administrado por via oral a pacientes com leucemia mieloide aguda recidivante ou refratária ou síndrome mielodisplásica
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.
Visão geral do estudo
Status
Intervenção / Tratamento
Descrição detalhada
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.
Tipo de estudo
Inscrição (Real)
Estágio
- Fase 1
Contactos e Locais
Locais de estudo
-
-
Ontario
-
Hamilton, Ontario, Canadá, L8V 1C3
- Juravinski Hospital and Cancer Centre
-
Toronto, Ontario, Canadá, M5G 2M9
- Princess Margaret Cancer Centre
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-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
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.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
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. |
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. |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Evaluation of the frequency and severity of treatment-emergent adverse events in patients
Prazo: 5 years
|
This will be done to assess safety and tolerability of CFI-400945 fumarate |
5 years
|
|
Highest tolerated dose of CFI-400945 fumarate
Prazo: 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
Prazo: 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
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Number of participants with response to treatment
Prazo: 5 years
|
Patients responses will be assess using the International Working Group response criteria for AML and MDS. |
5 years
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Investigador principal: Karen Yee, M.D., Princess Margaret Cancer Centre
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Real)
Conclusão do estudo (Real)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- CFI-400945-CL-002
Plano para dados de participantes individuais (IPD)
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Informações sobre medicamentos e dispositivos, documentos de estudo
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