A Study of Azacitidine and Venetoclax Versus a Stem Cell Transplant in People 65 Years and Older With Acute Myeloid Leukemia

NCT ID: NCT06903702

Last Updated: 2025-08-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-04

Study Completion Date

2027-06-30

Brief Summary

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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).

Detailed Description

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Conditions

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Acute Myeloid Leukemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a randomized, multi-center, open label, phase II study .
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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maintenance arm Azacitidine and Venetoclax (AZA/VEN)

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.

Group Type EXPERIMENTAL

Azacitidine (AZA)

Intervention Type DRUG

given daily for 7 days starting on Day 1 of each Cycle

Venetoclax

Intervention Type DRUG

orally daily for 28 days

Allogeneic hematopoietic stem cell transplantation (Allo-HCT arm)

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.

Group Type EXPERIMENTAL

Allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

After the conditioning treatment, the HSCT procedure is part of standard care on Day 0.

Interventions

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Azacitidine (AZA)

given daily for 7 days starting on Day 1 of each Cycle

Intervention Type DRUG

Venetoclax

orally daily for 28 days

Intervention Type DRUG

Allogeneic hematopoietic stem cell transplantation

After the conditioning treatment, the HSCT procedure is part of standard care on Day 0.

Intervention Type PROCEDURE

Eligibility Criteria

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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.
Minimum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genentech, Inc.

INDUSTRY

Sponsor Role collaborator

Memorial Sloan Kettering Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Roni Tamari, MD

Role: PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center

Locations

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Memorial Sloan Kettering at Basking Ridge (Limited Protocol Activities)

Basking Ridge, New Jersey, United States

Site Status RECRUITING

Memorial Sloan Kettering Monmouth (Consent and Follow-up)

Middletown, New Jersey, United States

Site Status RECRUITING

Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

Middletown, New Jersey, United States

Site Status RECRUITING

Memorial Sloan Kettering Bergen (Limited Protocol Activities)

Montvale, New Jersey, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center - Suffolk (Limited Protocol Activities)

Commack, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Westchester (Limited Protocol Activities)

Harrison, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Roni Tamari, MD

Role: CONTACT

646-608-3738

Eytan Stein, MD

Role: CONTACT

646-608-3749

Facility Contacts

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Roni Tamari, MD

Role: primary

Roni Tamari, MD

Role: primary

Roni Tamari, MD

Role: primary

Roni Tamari, MD

Role: primary

Roni Tamari, MD

Role: primary

Roni Tamari, MD

Role: primary

Roni Tamari, MD

Role: primary

Related Links

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http://www.mskcc.org/mskcc/html/44.cfm

Memorial Sloan Kettering Cancer Center

Other Identifiers

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25-063

Identifier Type: -

Identifier Source: org_study_id

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