Acute Myeloid Leukemia in RemissionPhase 2NCT04062266
AZA + Venetoclax as Maintenance Therapy in Patients With AML in Remission
A Phase 2 study testing Azacitidine and Venetoclax in people with Acute Myeloid Leukemia in Remission and FLT3 Gene Mutation, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase II trial studies how well azacitidine and venetoclax work in treating patients with acute myeloid leukemia that is in remission. Drugs used in chemotherapy, such as azacitidine and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia in Remission, FLT3 Gene Mutation, Hematologic and Lymphocytic Disorder
- What is being tested?
- Azacitidine and Venetoclax
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Sep 2019to 12 Oct 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Patients aged >/= 18 years AML who have achieved their FIRST CR or CRi and are not immediately candidates for allogeneic stem cell transplant.
- Patients who have received intensive therapy (defined as receiving standard or higher dose cytarabine-based therapy) to achieve remission (CR/CRi) should have received remission induction therapy and at least 1 consolidation cycle. These patients are eligible as long as they are not greater than 2 months from their last consolidation therapy and will be enrolled in COHORT 1.
- Patients who have received lower intensity therapy (defined as receiving low-dose cytarabine (LDAC) or hypomethylating agent (HMA)-based therapy) to achieve remission should have received at least 2 cycles of lower intensity therapy between the time they have achieved CR/CRi and enrollment on this protocol. They will be treated on COHORT 2.
- For either subgroup (lower or higher intensity), patients who have measurable residual disease may be enrolled on their respective cohort at any time without maximum 'time from consolidation' requirement.
- ECOG performance status of < or = 3
- Adequate organ function as follows:
Show all 16Show fewer
- Serum total bilirubin < or = to 1.5 X the Upper Limit of Normal (ULN)
- Serum creatinine < or = to 2.5 x ULN
- Adequate BM reserve:
- Absolute neutrophil count (ANC) > 0.5 x k/uL
- Platelet count > or = 30 x k/uL
- For females of childbearing age, they may participate if they:
- Have a negative serum or urine pregnancy test within 10 to 14 days of enrolling
- Agree to either abstinence or 2 effective contraceptive methods (such as barrier methods or hormonal contraception) throughout the treatment period and up to 30 days after discontinuing treatment.
- For male patients with a female partner of childbearing age, they may participate if they agree to either abstinence or 2 effective contraceptive methods throughout the treatment period and up to 30 days after discontinuing treatment.
- Ability to understand and sign informed consent.
Who cannot join
- Diagnosis of acute promyelocytic leukemia (APL), AML - M3 by FAB classification based on morphology, immunophenotype, molecular, or cytogenetic s studies.
- Diagnosis of AML associated t(15;17) or APL variant. Patients with t(9;22) are also ineligible unless they are unable or unwilling to receive therapy with a tyrosine kinase inhibitor.
- Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Patients with active CNS (central nervous system) disease.
- Patients with documented hypersensitivity to any components of the study program.
- Females who are pregnant or lactating or intending to become pregnant during the study.
- Patients with history of extramedullary AML, except for CNS involvement that is currently controlled, will not be eligible for enrollment.
- Patient should be removed from current trial if they wish to participate and get treatment on another trial.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Study of 5-Azacytidine (AZA) + Venetoclax as Maintenance Therapy in Patients With AML in Remission
This phase II trial studies how well azacitidine and venetoclax work in treating patients with acute myeloid leukemia that is in remission. Drugs used in chemotherapy, such as azacitidine and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
Read the detailed description
PRIMARY OBJECTIVES:
- To assess relapse-free survival (RFS) of patients (pts) with acute myeloid leukemia (AML) treated with 5-azacytidine (azacitidine; AZA) combined with venetoclax (VEN) as maintenance therapy after achieving remission.
SECONDARY OBJECTIVES:
- To assess modified RFS of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.
II. To assess overall survival (OS) of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.
III. To assess event-free survival (EFS) of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.
IV. To assess the duration of remission (CRd) of pts with AML treated 5-azacytidine combined with venetoclax as maintenance therapy.
- To assess toxicity and safety of 5-azacytidine combined with venetoclax as maintenance therapy in pts with AML.
VI. To assess the effects of 5-azacytidine combined with venetoclax on dynamics of minimal residual disease (MRD) and their relationship to outcomes.
OUTLINE:
Patients receive azacitidine subcutaneously (SC) or intravenously (IV) over 1 hour daily on days 1-5, and venetoclax orally (PO) daily on days 1-14. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days and then every 6-12 months thereafter.
- Conditions
- Acute Myeloid Leukemia in Remission, FLT3 Gene Mutation, Hematologic and Lymphocytic Disorder, Acute Myeloid Leukemia, Minimal Residual Disease Persistence, Therapy-Related Acute Myeloid Leukemia
- Being tested
- Azacitidine, Venetoclax
- Medical subject headings
- Hematologic Diseases, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
Also called 5 AZC, 5-AC, 5-Azacytidine, 5-AZC
Given SC or IV
VenetoclaxDrug
Also called ABT-0199, ABT-199, ABT199, GDC-0199
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (azacytidine, venetoclax) | Experimental | Patients receive azacitidine SC or IV over 1 hour daily on days 1-5, and venetoclax PO daily on days 1-14. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Azacitidine; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse-free survival (RFS)
The study will be continuously monitored for the primary endpoint, RFS, using the method of Thall, Wooten, and Tannir (2005). Will also summarize the posterior distribution of lambda-E (i.e., median RFS) assuming posterior mean and 95% credible interval.
Measured: From date of complete remission (CR) or complete remission with incomplete count recovery (CRi), assessed for up to 10 years
Secondary outcomes (5)
Incidence of toxicity
Toxicities are defined as any treatment -related clinically significant grade 3 or 4 non-hematologic adverse events occurred during cycles 1-2 of the trial. Will continuously monitor treatment-related toxicities using the Bayesian approach of Thall, Simon, Estey (1995).
Measured: Up to 10 years
Modified RFS
Distribution assessed using Kaplan-Meier method.
Measured: Time from enrollment on study until date of first objective documentation of relapse or death, assessed for up to 10 years
Overall survival (OS)
Distribution assessed using Kaplan-Meier method.
Measured: From the start of study treatment until date of death due to any cause, assessed for up to 10 years
Event free survival (EFS)
Distribution assessed using Kaplan-Meier method.
Measured: From the start of study treatment until date of first confirmed relapse, withdrawal from study due to adverse event, or death due to any cause, assessed for up to 10 years
Complete remission duration (CRd)
Distribution assessed using Kaplan-Meier method.
Measured: Time from CR or CRi until date of first objective documentation of confirmed relapse, assessed for up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2019
- Study start13 Sep 2019
- Primary completion12 Oct 2026
- Study completion12 Oct 2026
Why it stopped: \<75% participation
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia in RemissionStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI), Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Tapan M Kadia, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04062266
- Sponsor's protocol ID
- 2019-0226
- Other IDs
- NCI-2019-04987, 2019-0226
- Status last verified
- Oct 2026
- First submitted
- 16 Aug 2019
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Bazinet A, Kantarjian H, Bataller A, Pemmaraju N, Borthakur G, Chien K, Alvarado Y, Bose P, Jabbour E, Yilmaz M, DiNardo C, Issa G, Montalban-Bravo G, Short N, Sasaki K, Bull-Linderman D, Daver N, Garcia-Manero G, Ravandi F, Kadia T. Reduced dose azacitidine plus venetoclax as maintenance therapy in acute myeloid leukaemia following intensive or low-intensity induction: a single-centre, single-arm, phase 2 trial. Lancet Haematol. 2024 Apr;11(4):e287-e298. doi: 10.1016/S2352-3026(24)00034-6.
PubMed 38548404
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of 5-Azacytidine (AZA) + Venetoclax as Maintenance Therapy in Patients With AML in Remission
- ClinicalTrials.gov ID
- NCT04062266
- Acronym / sponsor ID
- 2019-0226
- Conditions
- Acute Myeloid Leukemia in Remission, FLT3 Gene Mutation, Hematologic and Lymphocytic Disorder, Acute Myeloid Leukemia, Minimal Residual Disease Persistence, Therapy-Related Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: \<75% participation
- Enrolment
- 36 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), Genentech, Inc.
- Study start
- 13 Sep 2019 (actual)
- Primary completion
- 12 Oct 2026 (actual)
- Study completion
- 12 Oct 2026 (actual)
- First posted
- 20 Aug 2019 (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.
This phase II trial studies how well azacitidine and venetoclax work in treating patients with acute myeloid leukemia that is in remission. Drugs used in chemotherapy, such as azacitidine and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
Detailed description
PRIMARY OBJECTIVES:
- To assess relapse-free survival (RFS) of patients (pts) with acute myeloid leukemia (AML) treated with 5-azacytidine (azacitidine; AZA) combined with venetoclax (VEN) as maintenance therapy after achieving remission.
SECONDARY OBJECTIVES:
- To assess modified RFS of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.
II. To assess overall survival (OS) of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.
III. To assess event-free survival (EFS) of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.
IV. To assess the duration of remission (CRd) of pts with AML treated 5-azacytidine combined with venetoclax as maintenance therapy.
- To assess toxicity and safety of 5-azacytidine combined with venetoclax as maintenance therapy in pts with AML.
VI. To assess the effects of 5-azacytidine combined with venetoclax on dynamics of minimal residual disease (MRD) and their relationship to outcomes.
OUTLINE:
Patients receive azacitidine subcutaneously (SC) or intravenously (IV) over 1 hour daily on days 1-5, and venetoclax orally (PO) daily on days 1-14. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days and then every 6-12 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (azacytidine, venetoclax) | Experimental | Drug: Azacitidine, Drug: VenetoclaxPatients receive azacitidine SC or IV over 1 hour daily on days 1-5, and venetoclax PO daily on days 1-14. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Patients aged >/= 18 years AML who have achieved their FIRST CR or CRi and are not immediately candidates for allogeneic stem cell transplant.
- Patients who have received intensive therapy (defined as receiving standard or higher dose cytarabine-based therapy) to achieve remission (CR/CRi) should have received remission induction therapy and at least 1 consolidation cycle. These patients are eligible as long as they are not greater than 2 months from their last consolidation therapy and will be enrolled in COHORT 1.
- Patients who have received lower intensity therapy (defined as receiving low-dose cytarabine (LDAC) or hypomethylating agent (HMA)-based therapy) to achieve remission should have received at least 2 cycles of lower intensity therapy between the time they have achieved CR/CRi and enrollment on this protocol. They will be treated on COHORT 2.
- For either subgroup (lower or higher intensity), patients who have measurable residual disease may be enrolled on their respective cohort at any time without maximum 'time from consolidation' requirement.
- ECOG performance status of < or = 3
Key exclusion criteria (8 listed)
- Diagnosis of acute promyelocytic leukemia (APL), AML - M3 by FAB classification based on morphology, immunophenotype, molecular, or cytogenetic s studies.
- Diagnosis of AML associated t(15;17) or APL variant. Patients with t(9;22) are also ineligible unless they are unable or unwilling to receive therapy with a tyrosine kinase inhibitor.
- Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Patients with active CNS (central nervous system) disease.
- Patients with documented hypersensitivity to any components of the study program.
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 |
|---|---|---|
| AzacitidineAlso: 5 AZC, 5-AC, 5-Azacytidine, 5-AZCGiven SC or IV | Drug | Treatment (azacytidine, venetoclax) |
| VenetoclaxAlso: ABT-0199, ABT-199, ABT199, GDC-0199Given PO | Drug | Treatment (azacytidine, venetoclax) |
Primary outcome
Relapse-free survival (RFS)
Time frame: From date of complete remission (CR) or complete remission with incomplete count recovery (CRi), assessed for up to 10 years
The study will be continuously monitored for the primary endpoint, RFS, using the method of Thall, Wooten, and Tannir (2005). Will also summarize the posterior distribution of lambda-E (i.e., median RFS) assuming posterior mean and 95% credible interval.
5 secondary outcomes
Incidence of toxicity
Time frame: Up to 10 years
Modified RFS
Time frame: Time from enrollment on study until date of first objective documentation of relapse or death, assessed for up to 10 years
Overall survival (OS)
Time frame: From the start of study treatment until date of death due to any cause, assessed for up to 10 years
Event free survival (EFS)
Time frame: From the start of study treatment until date of first confirmed relapse, withdrawal from study due to adverse event, or death due to any cause, assessed for up to 10 years
Complete remission duration (CRd)
Time frame: Time from CR or CRi until date of first objective documentation of confirmed relapse, assessed for up to 10 years
Results and safety
The study was stopped early (reason given: \<75% participation). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Derived from this study: PubMed 38548404Bazinet A, Kantarjian H, Bataller A, Pemmaraju N, Borthakur G, Chien K, Alvarado Y, Bose P, Jabbour E, Yilmaz M, DiNardo C, Issa G, Montalban-Bravo G, Short N, Sasaki K, Bull-Linderman D, Daver N, Garcia-Manero G, Ravandi F, Kadia T. Reduced dose azacitidine plus venetoclax as maintenance therapy in acute myeloid leukaemia following intensive or low-intensity induction: a single-centre, single-arm, phase 2 trial. Lancet Haematol. 2024 Apr;11(4):e287-e298. doi: 10.1016/S2352-3026(24)00034-6.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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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. This study is not recruiting at the moment.
Tapan M Kadia principal investigator
M.D. Anderson Cancer Center
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 Azacitidine and Venetoclax in people with Acute Myeloid Leukemia in Remission and FLT3 Gene Mutation, run by M.D. Anderson Cancer Center at 1 site in United States.
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 16 inclusion and 8 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: stopped early, as of the sponsor's last update on 16 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 United States. 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?
The study was stopped early (reason given: \<75% participation). Results are not available in the source record.
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?
Relapse-free survival (RFS) (time frame: From date of complete remission (CR) or complete remission with incomplete count recovery (CRi), assessed for up to 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04062266, which also lists 1 linked publication.
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 16 Oct 2026.