Clinical trial NCT06511882: Discontinuation of Hypomethylating Agent and Venetoclax in Patients With AML MRD
Official title: Discontinuation of Hypomethylating Agent and Venetoclax in Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) Who Have Achieved Negative Measurable Residual Disease (MRD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Keywords: AML
Lead sponsor: H. Lee Moffitt Cancer Center and Research Institute
Sponsor class: OTHER
Collaborators: name: AbbVie
First posted: 2024-07-22
Enrollment: 37
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Rates of Complete Response (CR)/Complete Response with incomplete hematologic recovery (CRi)
timeFrame: Up to 18 Months
Secondary outcomes:
measure: Overall Survival (OS)
timeFrame: Up to 36 Months
measure: Rates to Re-Treatment
timeFrame: Up to 36 Months
measure: Treatment Free Molecular Remission (TFMR)
timeFrame: Up to 36 Months
measure: European Organization for Research and Treatment of Cancer (EORTC-QLQ-C30)/Quality of Life (QoL)
timeFrame: Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)
measure: Patient Reported Outcome Measurement Information System (PROMIS)
timeFrame: Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)
measure: EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analog Scale (VAS)
timeFrame: Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)
Other outcomes:
Locations:
city: Tampa
country: United States
facility: Moffitt Cancer Center
state: Florida
Study officials:
affiliation: Moffitt Cancer Center
name: Onyee Chan, MD
role: PRINCIPAL_INVESTIGATOR
References: