RECRUITING

A Study of Azacitidine and Venetoclax in People With Acute Myeloid Leukemia (AML)

Conditions

Acute Myeloid Leukemia (AML)Azacitidine Venetoclax Allogeneic Stem Cell Transplant 24-347

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to find out if azacitidine and venetoclax are an effective treatment approach to get rid of or lower measurable residual disease (MRD) in people with acute myeloid leukemia (AML) who have received standard chemotherapy and are planning to have an allogeneic hematopoietic stem cell transplant (HSCT). Allogeneic HSCT, sometimes called a bone marrow transplant, involves receiving healthy blood-forming cells (stem cells) from a donor in order to replace the patient's immune system and lower the chances of the disease returning (relapse).

Official Title

A Phase 2 Study of Azacitidine and Venetoclax to Treat Acute Myeloid Leukemia Patients With Measurable Residual Disease Before an Allogeneic Stem Cell Transplant

Quick Facts

Study Start:2025-01-07

Study Completion:2027-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06773208

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* 1. Adult patient ≥18 years of age at the time of signing the informed consent form (ICF). Legal Authorized Representatives (LAR) are permitted. 2. Patient is willing and able to adhere to the study visit schedule and other protocol requirements. 3. Patient has a confirmed diagnosis of de-novo AML (non-APL) as per World Health Organization (2022) guidelines. All (non-APL) subtypes of AML are permitted, irrespective of ELN risk category or mutational status. 4. Patient has received 2 cycles of intensive chemotherapy (either induction + consolidation or 2 induction cycles). 5. Patient is in a morphologic remission, defined as less than 5% percent blasts seen by aspirate differential (or immunohistochemistry if no aspirate available) from bone marrow biopsy. 6. Patient and is either in CR, or CR with partial count recovery, either CRi/CRh\\\^1. 7. Patient has positive measurable residual disease (MRD) at or above a level of 0.1%, by flow cytometry or in molecular cases (NPM1 mutated or one of the CBF translocations) RT-qPCR reported at or above 0.01%, as described above (see section 3.6). 8. Patient is eligible for intensive chemotherapy and immediate allogeneic transplant, with intention to proceed to transplant after trial intervention. 9. Patient has an ECOG performance status of ≤3 10. Patient has adequate organ function defined as: 1. Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN 2. Serum total bilirubin \< 1.5 x ULN (or direct bilirubin normal in subjects with total bilirubin \> 1.5 ULN). Except in cases of Gilbert's disease. 3. Creatinine clearance greater than 30 mL/min based on the Cockroft-Gault glomerular filtration rate (GFR) estimation. 11. Absence of active uncontrolled infection, heart failure or severe psychiatric or neurological disease. 12. Females of childbearing potential may participate provided they have a negative serum pregnancy test at screening and a negative serum OR urine pregnancy test within two weeks of starting on treatment. 13. Females of reproductive potential should use effective contraception during the study, and for 6 months after last dose of azacitidine. Males with female partners of reproductive potential should use effective contraception during treatment and for 3 months after.	1. Patients with acute promyelocytic leukemia (APL) or relapsed/refractory AML 2. Blast crisis of chronic myeloid leukemia 3. Patient with 5% blasts or more by flow or bone marrow aspirate differential (or IHC if no aspirate available) 4. Patient without count recovery (CBC less than CRh) 5. Patient has received previous therapy with a venetoclax containing regimen. 6. Patient has 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. 7. Patient has active uncontrolled systemic fungal, bacterial, or viral infection. 8. Patient had recent, significant venous or arterial thrombotic event that would necessitate full anticoagulation or dual anti-platelet therapy, including PE within 30 days prior to start of treatment or insertion of drug eluting stent within 6 months prior to start of treatment. Chronic indications for anticoagulation such as atrial fibrillation, can be included if CHADS2 score below 4. 9. Patient has mechanical heart valve. 10. Patient had recent significant hemorrhagic episode, at the discretion of investigator. 11. Patient has significant active cardiac disease within 6 months prior to start of study treatment. 12. Patient is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. 13. Female subject who is pregnant or lactating.

Inclusion Criteria

Exclusion Criteria

* 1. Adult patient ≥18 years of age at the time of signing the informed consent form (ICF). Legal Authorized Representatives (LAR) are permitted.
2. Patient is willing and able to adhere to the study visit schedule and other protocol requirements.
3. Patient has a confirmed diagnosis of de-novo AML (non-APL) as per World Health Organization (2022) guidelines. All (non-APL) subtypes of AML are permitted, irrespective of ELN risk category or mutational status.
4. Patient has received 2 cycles of intensive chemotherapy (either induction + consolidation or 2 induction cycles).
5. Patient is in a morphologic remission, defined as less than 5% percent blasts seen by aspirate differential (or immunohistochemistry if no aspirate available) from bone marrow biopsy.
6. Patient and is either in CR, or CR with partial count recovery, either CRi/CRh\\\^1.
7. Patient has positive measurable residual disease (MRD) at or above a level of 0.1%, by flow cytometry or in molecular cases (NPM1 mutated or one of the CBF translocations) RT-qPCR reported at or above 0.01%, as described above (see section 3.6).
8. Patient is eligible for intensive chemotherapy and immediate allogeneic transplant, with intention to proceed to transplant after trial intervention.
9. Patient has an ECOG performance status of ≤3 10. Patient has adequate organ function defined as:
1. Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN
2. Serum total bilirubin \< 1.5 x ULN (or direct bilirubin normal in subjects with total bilirubin \> 1.5 ULN). Except in cases of Gilbert's disease.
3. Creatinine clearance greater than 30 mL/min based on the Cockroft-Gault glomerular filtration rate (GFR) estimation.
11. Absence of active uncontrolled infection, heart failure or severe psychiatric or neurological disease.
12. Females of childbearing potential may participate provided they have a negative serum pregnancy test at screening and a negative serum OR urine pregnancy test within two weeks of starting on treatment.
13. Females of reproductive potential should use effective contraception during the study, and for 6 months after last dose of azacitidine. Males with female partners of reproductive potential should use effective contraception during treatment and for 3 months after.

1. Patients with acute promyelocytic leukemia (APL) or relapsed/refractory AML 2. Blast crisis of chronic myeloid leukemia 3. Patient with 5% blasts or more by flow or bone marrow aspirate differential (or IHC if no aspirate available) 4. Patient without count recovery (CBC less than CRh) 5. Patient has received previous therapy with a venetoclax containing regimen. 6. Patient has 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.
7. Patient has active uncontrolled systemic fungal, bacterial, or viral infection.
8. Patient had recent, significant venous or arterial thrombotic event that would necessitate full anticoagulation or dual anti-platelet therapy, including PE within 30 days prior to start of treatment or insertion of drug eluting stent within 6 months prior to start of treatment. Chronic indications for anticoagulation such as atrial fibrillation, can be included if CHADS2 score below 4.
9. Patient has mechanical heart valve. 10. Patient had recent significant hemorrhagic episode, at the discretion of investigator.
11. Patient has significant active cardiac disease within 6 months prior to start of study treatment.
12. Patient is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
13. Female subject who is pregnant or lactating.

Contacts and Locations

Study Contact

Eytan Stein, MD

CONTACT

646-608-3749

steine@mskcc.org

Aaron Goldberg, MD,PhD

CONTACT

646-608-3752

Principal Investigator

Eytan Stein, MD

PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center

Study Locations (Sites)

Memorial Sloan Kettering at Basking Ridge (All Protocol Activities)

Basking Ridge, New Jersey, 07920

United States

Memorial Sloan Kettering Monmouth (All Protocol Activities)

Middletown, New Jersey, 07748

United States

Memorial Sloan Kettering Bergen (All Protocol Activities)

Montvale, New Jersey, 07645

United States

Memorial Sloan Kettering Suffolk-Commack (All Protocol Activities)

Commack, New York, 11725

United States

Memorial Sloan Kettering Westchester (Limited Protocol Activities)

Harrison, New York, 10604

United States

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

New York, New York, 10065

United States

Memorial Sloan Kettering Nassau (All Protocol Activities)

Rockville Centre, New York, 11553

United States

Collaborators and Investigators

Sponsor: Memorial Sloan Kettering Cancer Center

Eytan Stein, MD, PRINCIPAL_INVESTIGATOR, Memorial Sloan Kettering Cancer Center

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-01-07

Study Completion Date2027-01

Study Record Updates

Study Start Date2025-01-07

Study Completion Date2027-01

Terms related to this study

Keywords Provided by Researchers

Azacitidine
Venetoclax
Allogeneic Stem Cell Transplant
24-347

Additional Relevant MeSH Terms

Acute Myeloid Leukemia (AML)