Acute Myeloid LeukemiaPhase 2NCT06903702
A Study of Azacitidine and Venetoclax Versus a Stem Cell Transplant in People 65 Years and Older With Acute Myeloid Leukemia
A Phase 2 study testing Azacitidine (AZA), Venetoclax and Allogeneic hematopoietic stem cell transplantation in people with Acute Myeloid Leukemia, run by Memorial Sloan Kettering Cancer Center.
Trial summary
The researchers are doing this study to find out if an allogeneic hematopoietic stem cell transplant (HSCT) or maintenance therapy with azacitidine and venetoclax is more effective at keeping AML from coming back (relapsing). Read more about this study
- Who is it for?
- Ages 65 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Azacitidine (AZA), Venetoclax and Allogeneic hematopoietic stem cell transplantation
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 4 Aug 2026to Jun 2027
- 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 65 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Adult patients ≥65 years of age at the time of signing the informed consent form.
- Confirmed diagnosis of acute myeloid leukemia according to the ELN 2017 criteria
- Treatment with azacitidine and venetoclax for the diagnosis of AML
- o The first cycle of study treatment will start 28-42 days after the start of the second cycle of SOC AZA/VEN. In the event that patients can't be admitted for allo-HCT until after Day 42 due to donor related issues, an additional cycle of AZA/VEN will be allowed as a bridge to the transplant, and then initiation of conditioning will start no later than day 42 after the start of the third cycle.
- Patients with adequate organ function to be considered as candidates for allo-HCT:
- Cardiac: asymptomatic or if symptomatic, then LVEF at rest must be >40% and must improve with exercise.
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- Renal: CrCl ≥50 ml/min (measured or calculated/estimated).
- Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted (corrected for hemoglobin)
- Hepatic: < 5x ULN liver function tests and < 2x ULN total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
- KPS of ≥ 70
- Patients with suitable donor for allo-HCT
- Patients must achieve a morphologic remission <5% blast with MRD negative status by flow cytometry (defined as one or less residual leukemic blasts per 1000 leukocytes (or 10^3)) meeting one of the below:
- Complete remission (CR) defined as: <5% blasts with ANC> 1000 AND Plt >100K
- CRh defined as <5% blasts with ANC > 500 AND Plt >50K
- CRi defined as <5% blasts with ANC< 1000 OR Plt < 100K
- Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
Who cannot join
- Patients who are not considered to be transplant eligible (either due to lack of suitable donor or due to comorbidities/performance status). The reason the patient is not considered transplant eligible will be documented in the eCRF.
- History of prior allo-HCT
- Patients who underwent prior leukemia directed treatment (other than aza/ven)
- Patients with CNS involvement at any time point prior to enrollment.
- Patients with previous exposure to venetoclax or an HMA for the treatment of a myeloid malignancy
- Patients who are planned for treatment other than AZA/VEN
- Patients who are planned for treatment with HMA/VEN with another agent (e.g. a "triplet")
- Presence of any other condition that may increase the risk associated with study participation, and in the opinion of the investigator, would make the patient inappropriate for entry into the study.
About this study
Official title: A Randomized, Multicenter, Phase II Study of Maintenance Azacitidine and Venetoclax Versus an Allogeneic Stem Cell Transplant in Older Patients (65 Years and Older) With Acute Myeloid Leukemia Who Achieve an MRD Negative Complete Remission After Induction With Azacitidine and Venetoclax
The researchers are doing this study to find out if an allogeneic hematopoietic stem cell transplant (HSCT) or maintenance therapy with azacitidine and venetoclax is more effective at keeping AML from coming back (relapsing).
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Azacitidine (AZA), Venetoclax, Allogeneic hematopoietic stem cell transplantation
- Keywords
- Azacitidine, Venetoclax, Allogeneic Stem Cell Transplant
- 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.
Azacitidine (AZA)Drug
given daily for 7 days starting on Day 1 of each Cycle
VenetoclaxDrug
orally daily for 28 days
Allogeneic hematopoietic stem cell transplantationProcedure or surgery
After the conditioning treatment, the HSCT procedure is part of standard care on Day 0.
| Study group | Role | What they receive |
|---|---|---|
| maintenance arm Azacitidine and Venetoclax (AZA/VEN) | Experimental | Patients randomized to the maintenance arm (AZA/VEN) will be treated with azacitidine given daily for 7 days starting on Day 1 of each Cycle (7 consecutive days or a total of 7 days with a 2 day break due to weekends or holidays is permissible). Venetoclax will be taken orally daily for 28 days. A cycle will be considered 28 days. Receives: Drug: Azacitidine (AZA); Drug: Venetoclax |
| Allogeneic hematopoietic stem cell transplantation (Allo-HCT arm) | Experimental | Patients who are randomized to the transplant arm will start the conditioning regimen no later than 6 weeks (day 42) after C2D1 of AZA/VEN. Receives: Procedure: Allogeneic hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
1-year Relapse free survival (RFS)
Relapse free survival (RFS)- will be defined with patients being alive and without any evidence of disease. i.e. no morphologic relapse and no emergence of minimal residual disease. In the event of emergence of MRD without a morphologic relapse, this will be defined as relapse only if documented on 2 separate time points. For patients alive and in remission at the data cut-off, RFS will be censored at the last assessment date.
Measured: 1 year
Secondary outcomes (1)
Overall survival (OS)
will be defined with patients being alive and non-relapse mortality (NRM) will be defined as death in the absence of disease recurrence.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2025
- Study start4 Aug 2026 (estimated)
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Why it stopped: Lack of funding
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.
- More trials run by Memorial Sloan Kettering 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a randomized, multi-center, open label, phase II study .
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Roni Tamari, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06903702
- Sponsor's protocol ID
- 25-063
- Status last verified
- Aug 2026
- First submitted
- 25 Mar 2025
- Last updated
- 5 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Multicenter, Phase II Study of Maintenance Azacitidine and Venetoclax Versus an Allogeneic Stem Cell Transplant in Older Patients (65 Years and Older) With Acute Myeloid Leukemia Who Achieve an MRD Negative Complete Remission After Induction With Azacitidine and Venetoclax
- ClinicalTrials.gov ID
- NCT06903702
- Acronym / sponsor ID
- 25-063
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Azacitidine, Venetoclax, Allogeneic Stem Cell Transplant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 4 Aug 2026 (estimated)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 1 Apr 2025 (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 researchers are doing this study to find out if an allogeneic hematopoietic stem cell transplant (HSCT) or maintenance therapy with azacitidine and venetoclax is more effective at keeping AML from coming back (relapsing).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This is a randomized, multi-center, open label, phase II study .
| Arm | Type | Receives |
|---|---|---|
| maintenance arm Azacitidine and Venetoclax (AZA/VEN) | Experimental | Drug: Azacitidine (AZA), Drug: VenetoclaxPatients randomized to the maintenance arm (AZA/VEN) will be treated with azacitidine given daily for 7 days starting on Day 1 of each Cycle (7 consecutive days or a total of 7 days with a 2 day break due to weekends or holidays is permissible). Venetoclax will be taken orally daily for 28 days. A cycle will be considered 28 days. |
| Allogeneic hematopoietic stem cell transplantation (Allo-HCT arm) | Experimental | Procedure: Allogeneic hematopoietic stem cell transplantationPatients who are randomized to the transplant arm will start the conditioning regimen no later than 6 weeks (day 42) after C2D1 of AZA/VEN. |
Who may qualify
- Ages
- 65 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (16 listed)
- Adult patients ≥65 years of age at the time of signing the informed consent form.
- Confirmed diagnosis of acute myeloid leukemia according to the ELN 2017 criteria
- Treatment with azacitidine and venetoclax for the diagnosis of AML
- o The first cycle of study treatment will start 28-42 days after the start of the second cycle of SOC AZA/VEN. In the event that patients can't be admitted for allo-HCT until after Day 42 due to donor related issues, an additional cycle of AZA/VEN will be allowed as a bridge to the transplant, and then initiation of conditioning will start no later than day 42 after the start of the third cycle.
- Patients with adequate organ function to be considered as candidates for allo-HCT:
Key exclusion criteria (8 listed)
- Patients who are not considered to be transplant eligible (either due to lack of suitable donor or due to comorbidities/performance status). The reason the patient is not considered transplant eligible will be documented in the eCRF.
- History of prior allo-HCT
- Patients who underwent prior leukemia directed treatment (other than aza/ven)
- Patients with CNS involvement at any time point prior to enrollment.
- Patients with previous exposure to venetoclax or an HMA for the treatment of a myeloid malignancy
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 |
|---|---|---|
| Azacitidine (AZA)given daily for 7 days starting on Day 1 of each Cycle | Drug | maintenance arm Azacitidine and Venetoclax (AZA/VEN) |
| Venetoclaxorally daily for 28 days | Drug | maintenance arm Azacitidine and Venetoclax (AZA/VEN) |
| Allogeneic hematopoietic stem cell transplantationAfter the conditioning treatment, the HSCT procedure is part of standard care on Day 0. | Procedure or surgery | Allogeneic hematopoietic stem cell transplantation (Allo-HCT arm) |
Primary outcome
1-year Relapse free survival (RFS)
Time frame: 1 year
Relapse free survival (RFS)- will be defined with patients being alive and without any evidence of disease. i.e. no morphologic relapse and no emergence of minimal residual disease. In the event of emergence of MRD without a morphologic relapse, this will be defined as relapse only if documented on 2 separate time points. For patients alive and in remission at the data cut-off, RFS will be censored at the last assessment date.
1 secondary outcome
Overall survival (OS)
Time frame: 3 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org
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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.
Roni Tamari, MD principal investigator
Memorial Sloan Kettering Cancer Center
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azacitidine (AZA), Venetoclax and Allogeneic hematopoietic stem cell transplantation in people with Acute Myeloid Leukemia, run by Memorial Sloan Kettering Cancer Center.
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: withdrawn, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
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 withdrawn before enrolling participants, so there are no results.
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?
1-year Relapse free survival (RFS) (time frame: 1 year). 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 5 Aug 2026.