Home › Trials › NCT06903702

Azacitidine plus venetoclax versus stem cell transplant in adults 65 and older with AML

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

Phase 2 Interventional Memorial Sloan Kettering Cancer Center · NCT06903702

This study will test whether maintenance azacitidine with venetoclax or an allogeneic stem cell transplant better prevents AML from coming back in people aged 65 and older.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment100 (estimated)
Ages65 Years and up
SexAll
SponsorMemorial Sloan Kettering Cancer Center Academic / other
Locations7 sites (Basking Ridge, New Jersey and 6 other locations)
Trial IDNCT06903702 on ClinicalTrials.gov

What this trial studies

This Phase 2 interventional study compares allogeneic hematopoietic stem cell transplant (allo‑HSCT) to maintenance therapy with azacitidine plus venetoclax in patients age 65 and older who have AML and have been treated with azacitidine/venetoclax. Eligible participants must have a confirmed ELN 2017 diagnosis of AML, have received standard azacitidine and venetoclax, and meet organ‑function criteria to be considered transplant candidates. Participants will either proceed to allo‑HSCT (with permitted bridging AZA/VEN if transplant timing requires) or continue on azacitidine plus venetoclax maintenance, with treatment activities and follow‑up conducted at Memorial Sloan Kettering sites in New Jersey. The study aims to compare relapse rates and durability of remission between the transplant and maintenance approaches.

Who should consider this trial

Good fit: Ideal candidates are adults aged 65 or older with ELN 2017–confirmed AML who have already been treated with azacitidine and venetoclax and have adequate cardiac, renal, and pulmonary function to be considered for allo‑HSCT.

Not a fit: Patients who are too frail for transplant, have inadequate organ function, or who have not received azacitidine plus venetoclax as their AML treatment are unlikely to qualify or benefit from the options compared here.

Why it matters

Potential benefit: If successful, this could identify a treatment approach that lowers relapse risk and improves long‑term outcomes for older adults with AML while clarifying who most benefits from transplant versus maintenance therapy.

How similar studies have performed: Azacitidine plus venetoclax has shown high response rates in older AML patients and allo‑HSCT is an established curative option, but direct comparisons of transplant versus maintenance AZA/VEN in this age group are limited.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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.
  * 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.

Exclusion Criteria:

* 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.

Where this trial is running

Basking Ridge, New Jersey and 6 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Myeloid LeukemiaAzacitidineVenetoclaxAllogeneic Stem Cell Transplant
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.