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Gilteritinib-Azacitidine-Venetoclax Combination Therapy in Patients With Relapsed/Refractory Leukemia (GAVE001)

5 de agosto de 2026 atualizado por: Florian Kuchenbauer

A Phase 2 Trial of Gilteritinib in Combination With Azacitidine and Venetoclax to Overcome Venetoclax Resistance in Patients With Relapsed/Refractory FLT3-wild Type Acute Myeloid Leukemia

The goal of this clinical trial is to learn if gilteritinib-azacitidine-venetoclax combination drug therapy works to treat adults with FLT3-wt acute myeloid leukemia (AML). It will also learn about the safety and efficacy of this treatment. The main question this clinical trial aims to answer are:

  • Will adding gilteritinib to standard-of-care therapy azacitidine-venetoclax show be more effective in treating AML FLT3-wt patients who have previously been treated with azacitidine and/or venetoclax for their disease and have had their cancer come back (relapsed) or have stopped responding to treatment (refractory)?

All participants in this trial will receive the combination therapy. Participants will be on repeated 28-day cycles, for a minimum of 2 cycles, while on the study

Participants will:

  • Take the gilteritinib once daily, every day
  • Take azacitidine and venetoclax on days 1-7 of each cycle
  • Keep a daily dose diary of the days and times they have taken their treatments
  • Visit the clinic every 4 weeks for checkups and tests

Visão geral do estudo

Status

Ainda não está recrutando

Tipo de estudo

Intervencional

Inscrição (Estimado)

20

Estágio

  • Fase 2

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

    • British Columbia
      • Vancouver, British Columbia, Canadá
        • Vancouver General Hospital

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Descrição

Inclusion Criteria:

Participants must meet all the following inclusion criteria to be eligible for participation in this trial.

  1. Age ≥ 18 years old at the time of informed consent.
  2. Pathologically confirmed diagnosis of AML previously treated with azacitidine and venetoclax that are azacitidine and venetoclax-refractory, defined as:

2a. Primary refractory: Defined as no complete remission (CR) or CR with incomplete recovery (CRi) following at least 2 cycles of azacitidine and venetoclax with AML blasts ≥5% in the bone marrow, or 2b. Relapsed: Defined as recurrence of AML blasts ≥5% in the bone marrow after a previous CR or CRi to azacitidine and venetoclax 3. Confirmed to be negative for FLT3- internal tandem duplication (ITD) and tyrosine kinase domain (TKD) mutations on repeat clinical testing following their last line of therapy 4. Adequate functional status (ECOG ≤ 2) 5. Participant is willing to provide informed consent and comply with trial procedures 6. Participants of either biological sex must be able and willing to use Health-Canada approved effective contraception methods starting 2-weeks prior to trial treatment, throughout the trial, and for 6 months following the last dose of trial drugs 7. For participants of child-bearing potential (menstruation within <2 years): negative serum pregnancy test within 14-days prior to enrollment.

8. Not currently breastfeeding and will not breastfeed while on the trial and for at least 2 months following the last trial dose.

Waivers to the inclusion criteria will NOT be allowed.

Exclusion Criteria:

Participants are excluded from the trial if any of the following criteria apply:

1. Diagnosis of acute promyelocytic leukemia (APL), BCR-ABL positivity, or favorable risk AML 2. Currently considered as eligible for intensive chemotherapy treatment in the opinion of the Investigator 3. Inadequate organ function, defined as: 3a. Liver function: Serum aspartate aminotransferase and alanine aminotransferase ≥ 2.5 x upper limit of normal (ULN) and total bilirubin ≥ 1.5 x ULN 3b. Renal function: estimated glomerular filtration rate of < 30 mL/min as calculated by the Modification of Diet in Renal Disease equation.

3c. Cardiac function: New York Heart Association (NYHA) class 3 or 4 heart failure or known history of left ventricular ejection fraction ≤ 40% or known history of prolonged QTc syndrome 4. Corrected QTc of > 450ms that is not corrected on repeat electrocardiogram (ECG) testing 5. Active and untreated CNS leukemia 6. Active solid organ malignancy requiring treatment in the last 24 months, with the exception of: 6a. Treated nonmelanoma skin cancer 6b. Completely treated breast carcinoma that is considered cured 6c. Completely treated cervical carcinoma that is considered cured 6d. Localized breast or prostate cancer receiving androgen deprivation therapy 6e. A previously treated malignancy that is considered cured with minimal risk of recurrence 7. Extramedullary disease without concomitant marrow based disease (blasts ≤5%) 8. Uncontrolled, active infection or untreated hepatitis B, C, or HIV 9. Known allergy or intolerance to azacitidine, venetoclax, or gilteritinib 10. Any comorbidity that the investigator believes would be incompatible with safe receipt or therapy 11. Inability to comply with requirements of the trial protocol 12. Pregnancy or breastfeeding 13. Unable or unwilling to use Health Canada-approved highly effective methods of contraception (i.e., hormonal contraceptives, vasectomy, tubal ligation, or double-barrier method), or abstinence while on the trial and for at least 6 months following the last dose of trial drugs.

Waivers to the exclusion criteria will NOT be allowed.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

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: All Participants
administration of combination therapy gilteritinib-azacitidine-venetoclax on repeated 28-day cycles for a minimum of 2 cycles. Gilteritinib will be taken daily and azacitidine-venetoclax on days 1-7 of each cycle.
administration of combination therapy gilteritinib-azacitidine-venetoclax on repeated 28-day cycles for a minimum of 2 cycles. Gilteritinib will be taken daily and azacitidine-venetoclax on days 1-7 of each cycle.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Efficacy of triplet regimen on AML FLT3-wt participants
Prazo: From enrollment to the end of treatment at week 8
the ORR up to two cycles of triplet therapy. ORR is defined as the proportion of participants who achieve a CR, CRi, or MLFS after completing two cycles of the combination therapy
From enrollment to the end of treatment at week 8

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
To assess the safety of triplet regimen based on toxicity findings
Prazo: from enrollment to one month after end of treatment at 12 weeks
Safety and toxicity events defined as the frequency of grade > 3 non-hematologic adverse events while receiving triplet-therapy, as per National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) version 6.0
from enrollment to one month after end of treatment at 12 weeks
Impact of triplet regimen on measurable residual disease (MRD) in participants achieving a response
Prazo: from enrollment to one month after the end of treatment at 12 weeks
Rate of MRD negativity amongst participants achieving a response. MRD negativity is defined as the presence of leukemia cells below the threshold of detection by flow cytometry or PCR assay, as defined by ELN 2022 response criteria
from enrollment to one month after the end of treatment at 12 weeks
Impact of study treatment on event-free survival (EFS)
Prazo: from enrollment to end of study closeout at 36 months
EFS, defined as the time from start of therapy until disease persistence after ≥ 2 cycles of triplet therapy, disease relapse of progression while on therapy, or death. EFS is defined as the time from start of treatment until disease progression after > 2 cycles of triplet therapy, disease relapse of progression while on triplet therapy, or death from any cause.
from enrollment to end of study closeout at 36 months
Impact of study treatment on relapse-free survival (RFS)
Prazo: from enrollment to end of study closeout at 36 months
Relapse-free survival (RFS), defined as the time from start of therapy until disease progression or death for participants who achieve a response. RFS is defined as the time from start of treatment until disease progression or death from AML
from enrollment to end of study closeout at 36 months
Impact of study treatment on overall survival (OS) in AML FLT3-wt patients
Prazo: from enrollment to end of study closeout at 36 months
Overall survival (OS), defined as the time from start of therapy until death from any cause.
from enrollment to end of study closeout at 36 months

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

1 de setembro de 2026

Conclusão Primária (Estimado)

1 de outubro de 2029

Conclusão do estudo (Estimado)

1 de maio de 2030

Datas de inscrição no estudo

Enviado pela primeira vez

5 de agosto de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

5 de agosto de 2026

Primeira postagem (Real)

10 de agosto de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

10 de agosto de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

5 de agosto de 2026

Última verificação

1 de agosto de 2026

Mais Informações

Termos relacionados a este estudo

Outros números de identificação do estudo

  • GAVE001

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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