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

5 de agosto de 2026 actualizado 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

Descripción general del estudio

Estado

Aún no reclutando

Tipo de estudio

Intervencionista

Inscripción (Estimado)

20

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

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

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Descripción

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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: N / A
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
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.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Efficacy of triplet regimen on AML FLT3-wt participants
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
To assess the safety of triplet regimen based on toxicity findings
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

1 de septiembre de 2026

Finalización primaria (Estimado)

1 de octubre de 2029

Finalización del estudio (Estimado)

1 de mayo de 2030

Fechas de registro del estudio

Enviado por primera vez

5 de agosto de 2026

Primero enviado que cumplió con los criterios de control de calidad

5 de agosto de 2026

Publicado por primera vez (Actual)

10 de agosto de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

10 de agosto de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

5 de agosto de 2026

Última verificación

1 de agosto de 2026

Más información

Términos relacionados con este estudio

Otros números de identificación del estudio

  • GAVE001

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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