Acute Myeloid LeukemiaPhase 2GAVE001NCT07755202
Gilteritinib-Azacitidine-Venetoclax Combination Therapy in Patients With Relapsed/Refractory Leukemia
A Phase 2 study testing gilteritinib-azacitidine-venetoclax in people with Acute Myeloid Leukemia and AML With FLT3 Mutation, run by Florian Kuchenbauer at 1 site in Canada.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, AML With FLT3 Mutation
- What is being tested?
- gilteritinib-azacitidine-venetoclax
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteCanada
- Study dates
- 1 Sep 2026to 1 May 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must meet all the following inclusion criteria to be eligible for participation in this trial.
- Age ≥ 18 years old at the time of informed consent.
- Pathologically confirmed diagnosis of AML previously treated with azacitidine and venetoclax that are azacitidine and venetoclax-refractory, defined as:
- 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
- Relapsed: Defined as recurrence of AML blasts ≥5% in the bone marrow after a previous CR or CRi to azacitidine and venetoclax
- 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
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- Adequate functional status (ECOG ≤ 2)
- Participant is willing to provide informed consent and comply with trial procedures
- 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
- For participants of child-bearing potential (menstruation within <2 years): negative serum pregnancy test within 14-days prior to enrollment.
- 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.
Who cannot join
- Participants are excluded from the trial if any of the following criteria apply:
- Diagnosis of acute promyelocytic leukemia (APL), BCR-ABL positivity, or favorable risk AML
- Currently considered as eligible for intensive chemotherapy treatment in the opinion of the Investigator
- Inadequate organ function, defined as:
- Liver function: Serum aspartate aminotransferase and alanine aminotransferase ≥ 2.5 x upper limit of normal (ULN) and total bilirubin ≥ 1.5 x ULN
- Renal function: estimated glomerular filtration rate of < 30 mL/min as calculated by the Modification of Diet in Renal Disease equation.
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- 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
- Corrected QTc of > 450ms that is not corrected on repeat electrocardiogram (ECG) testing
- Active and untreated CNS leukemia
- Active solid organ malignancy requiring treatment in the last 24 months, with the exception of:
- Treated nonmelanoma skin cancer
- Completely treated breast carcinoma that is considered cured
- Completely treated cervical carcinoma that is considered cured
- Localized breast or prostate cancer receiving androgen deprivation therapy
- A previously treated malignancy that is considered cured with minimal risk of recurrence
- Extramedullary disease without concomitant marrow based disease (blasts ≤5%)
- Uncontrolled, active infection or untreated hepatitis B, C, or HIV
- Known allergy or intolerance to azacitidine, venetoclax, or gilteritinib
- Any comorbidity that the investigator believes would be incompatible with safe receipt or therapy
- Inability to comply with requirements of the trial protocol
- Pregnancy or breastfeeding
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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)?
Read more
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
- Conditions
- Acute Myeloid Leukemia, AML With FLT3 Mutation
- Being tested
- gilteritinib-azacitidine-venetoclax
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gilteritinib-azacitidine-venetoclaxCombination product
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.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Experimental | 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. Receives: Combination Product: gilteritinib-azacitidine-venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of triplet regimen on AML FLT3-wt participants
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
Measured: From enrollment to the end of treatment at week 8
Secondary outcomes (5)
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
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
Measured: 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
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
Measured: from enrollment to one month after the end of treatment at 12 weeks
Impact of study treatment on event-free survival (EFS)
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.
Measured: from enrollment to end of study closeout at 36 months
Impact of study treatment on relapse-free survival (RFS)
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
Measured: from enrollment to end of study closeout at 36 months
Impact of study treatment on overall survival (OS) in AML FLT3-wt patients
Overall survival (OS), defined as the time from start of therapy until death from any cause.
Measured: from enrollment to end of study closeout at 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2026
- Study start1 Sep 2026 (estimated)
- Primary completion1 Oct 2029 (estimated)
- Study completion1 May 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Florian Kuchenbauer
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Florian Kuchenbauer, British Columbia Cancer Agency
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07755202
- Sponsor's protocol ID
- GAVE001
- Acronym
- GAVE001
- Status last verified
- Sep 2026
- First submitted
- 5 Aug 2026
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07755202
- Acronym / sponsor ID
- GAVE001 / GAVE001
- Conditions
- Acute Myeloid Leukemia, AML With FLT3 Mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Florian KuchenbauerUniversity, hospital or other
- Study start
- 1 Sep 2026 (estimated)
- Primary completion
- 1 Oct 2029 (estimated)
- Study completion
- 1 May 2030 (estimated)
- First posted
- 10 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Participants | Experimental | Combination Product: gilteritinib-azacitidine-venetoclaxadministration 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Participants must meet all the following inclusion criteria to be eligible for participation in this trial.
- Age ≥ 18 years old at the time of informed consent.
- Pathologically confirmed diagnosis of AML previously treated with azacitidine and venetoclax that are azacitidine and venetoclax-refractory, defined as:
- 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
- Relapsed: Defined as recurrence of AML blasts ≥5% in the bone marrow after a previous CR or CRi to azacitidine and venetoclax
Key exclusion criteria (23 listed)
- Diagnosis of acute promyelocytic leukemia (APL), BCR-ABL positivity, or favorable risk AML
- Currently considered as eligible for intensive chemotherapy treatment in the opinion of the Investigator
- Inadequate organ function, defined as:
- Liver function: Serum aspartate aminotransferase and alanine aminotransferase ≥ 2.5 x upper limit of normal (ULN) and total bilirubin ≥ 1.5 x ULN
- Renal function: estimated glomerular filtration rate of < 30 mL/min as calculated by the Modification of Diet in Renal Disease equation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| gilteritinib-azacitidine-venetoclaxadministration 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. | Combination product | All Participants |
Primary outcome
Efficacy of triplet regimen on AML FLT3-wt participants
Time frame: 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
5 secondary outcomes
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 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
Time frame: from enrollment to one month after the end of treatment at 12 weeks
Impact of study treatment on event-free survival (EFS)
Time frame: from enrollment to end of study closeout at 36 months
Impact of study treatment on relapse-free survival (RFS)
Time frame: from enrollment to end of study closeout at 36 months
Impact of study treatment on overall survival (OS) in AML FLT3-wt patients
Time frame: from enrollment to end of study closeout at 36 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing gilteritinib-azacitidine-venetoclax in people with Acute Myeloid Leukemia and AML With FLT3 Mutation, run by Florian Kuchenbauer at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of triplet regimen on AML FLT3-wt participants (time frame: From enrollment to the end of treatment at week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.