Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06511882

Discontinuation of Hypomethylating Agent and Venetoclax in Patients With AML MRD

The purpose of this research study is to see if people whose Acute myeloid leukemia (AML) is being successfully treated with azacitidine or decitabine in combination with venetoclax can discontinue this chemotherapy for some period of time after a year of treatment without increasing the likelihood that their AML will return.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Acute Myeloid Leukemia Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location status: Recruiting

Location contact

Alison Walker, MD

SUB_INVESTIGATOR

Andrew Kuykendall, MD

SUB_INVESTIGATOR

David Sallman, MD

SUB_INVESTIGATOR

Eric Padron, MD

SUB_INVESTIGATOR

Gina Bellenger

CONTACT

[email protected]

813-745-6138

Jeffrey Lancet, MD

SUB_INVESTIGATOR

Onyee Chan, MD

PRINCIPAL_INVESTIGATOR

Rami Komrokji, MD

SUB_INVESTIGATOR

Timothy Kubal, MD

SUB_INVESTIGATOR

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adults 18 years of age or older at the time of obtaining informed consent.
  • Diagnosed with Acute Myeloid Leukemia (AML) (non-M3) as defined by 2016 World Health Organization (WHO)
  • Eastern Cooperative Group (ECOG) performance status score ≤ 2
  • Currently on frontline therapy with HMA (azacitidine or decitabine)/VEN and achieved Complete Remission (CR)/Complete Remission with incomplete marrow recovery (CRi) with MRD negativity defined as < 0.1% by Multiparameter Flow Cytometry (MFC)
  • Within 12 months of starting HMA (azacitidine or decitabine)/VEN
  • Ineligible for or declined allogeneic hematopoietic cell transplantation (HCT)
  • Ability to understand and the willingness to sign a written informed consent document
  • Must agree to adhere to the study visit schedule and other protocol requirements
  • Patients must be able to provide adequate Bone Marrow (BM) aspirate and biopsy specimens for histopathological and Measurable Residual Disease analysis during the screening procedure

Exclusion criteria

  • Use of cytotoxic chemotherapeutic agents, or experimental agents (agents that are not commercially available) for the treatment of AML within 28 days, or 5 half-lives, at the start of the study. Only patients who are receiving frontline HMA (azacitidine or decitabine)/VEN are potentially eligible, but if they had received a course of hydroxyurea prior to achieving CR/CRi, this is allowed.
  • Any serious medical condition or uncontrolled current illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements or will place the subject at unacceptable risk if he/she participates in the study. Controlled infections or other medical conditions on long-term therapy is allowed.
  • Patients who harbored TP53 mutation at diagnosis
  • AML with extramedullary involvement including central nervous system (CNS) involvement, myeloid sarcoma, and leukemia cutis requiring directed therapy at the time of enrollment.
  • Patient is pregnant.

Treatment and study plan

Azacitidine

Drug

Standard of Care Intravenous (IV) infusion

Other names: Vidaza

decitabine

Drug

Standard of Care Intravenous (IV) infusion

Other names: DACOGEN

Venetoclax

Drug

Standard of Care PO (By Mouth)

Other names: Venclexta

Primary outcomes

  1. Rates of Complete Response (CR)/Complete Response with incomplete hematologic recovery (CRi)

    Time frame: Up to 18 Months

    Rates of CR/CRi at 18 months from the time of initial CR/CRi in patients who discontinue frontline HMA (azacitidine or decitabine)/VEN (venetoclax) after achieving Measurable Residual Disease (MRD) negativity by MFC within 12 months of starting therapy.

    The Null hypothesis (p0) will be tested against the alternative hypothesis (p1).

    Null hypothesis: p0 ≤ 50% will remain in CR/CRi at 18 months from the time of initial CR/CRi Alternative hypothesis: p1 ≥ 70% will remain in CR/CRi at 18 months from the time of initial CR/CRi

Secondary outcomes

  1. Overall Survival (OS)

    Time frame: Up to 36 Months

    Overall survival will be assessed in MRD-negative patients who discontinue HMA (azacitidine or decitabine)/VEN (venetoclx).

    Overall Survival (OS) is defined as the length of time from the start of treatment until death due to any cause.

  2. Rates to Re-Treatment

    Time frame: Up to 36 Months

    Rates to re-treatment is defined as Measurable Residual Disease (MRD)-negative CR/CRi and CR/CRi and rates to re-treatment following molecular or morphologic relapse duration discontinuation.

  3. Treatment Free Molecular Remission (TFMR)

    Time frame: Up to 36 Months

    Defined as time from Measurable Residual Disease (MRD) negativity to first MRD positivity

  4. European Organization for Research and Treatment of Cancer (EORTC-QLQ-C30)/Quality of Life (QoL)

    Time frame: Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)

    EORTC-QLQ-C30/QoL consists of a 30-item subject self-report questionnaire composed of five functional scales (physical, role, emotional, social, and cognitive), three symptom scales (fatigue, nausea and vomiting, and pain), a global health status/quality of life scale, and six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties).

    Scale scores range from 0-100.

  5. Patient Reported Outcome Measurement Information System (PROMIS)

    Time frame: Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)

    PROMIS scale provides response options 1-5 to indicate patient-reported health status measures for physical, mental, and social well-being.

    It measures concepts such as pain, fatigue, physical function, depression, anxiety, and social function.

  6. EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analog Scale (VAS)

    Time frame: Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)

    EQ-5D-5L VAS is an instrument for measuring the general health status of an individual. It consists of five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.

    Scale scores range from 0-100.

Study contacts

Contact information is provided by the study sponsor or research team.

Gina Bellenger

CONTACT

[email protected]

813-745-6138

Sponsors and collaborators

Lead sponsor

H. Lee Moffitt Cancer Center and Research Institute

Other

Collaborators

  • AbbVie

Registry information

Official study title

Discontinuation of Hypomethylating Agent and Venetoclax in Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) Who Have Achieved Negative Measurable Residual Disease (MRD)

Important dates

Study start
2024
Primary completion
2027
Study completion
2028
First posted
Jul 22, 2024
Registry last updated
Feb 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.