This phase II trial tests how well venetoclax with cladribine and cytarabine alternating with azacitidine and venetoclax works in treating patients with newly diagnosed monocytic acute myeloid leukemia (AML) and active signaling mutated AML. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking BCL-2, a protein needed for cancer cell survival. Chemotherapy drugs, such as cladribine, cytarabine and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving venetoclax with cladribine and cytarabine alternating with azacitidine and venetoclax may kill more cancer cells in patients with newly diagnosed monocytic AML and active signaling mutated AML.
PRIMARY OBJECTIVE: I. Assess efficacy of the investigational treatment based on disease remission. SECONDARY OBJECTIVES: I. Assess efficacy of the investigational treatment based on clinical response. II. Assess any-cause survival after treatment. III. Assess survival in the absence of treatment failure, hematologic relapse, or progressive disease. IV. Assess duration of response, based on morphological assessments. V. Assess the safety and tolerability of the regimen. EXPLORATORY OBJECTIVES: I. Assess response based on measurable residual disease (MRD). II. Identify markers of clonal or clinical response and resistance to treatment. III. Assess participant quality of life (QoL) using Patient Reported Outcomes Common Terminology Criteria for Adverse Events (PRO CTCAE). OUTLINE: INDUCTION: Patients receive cladribine intravenously (IV) over 1-2 hours on days 1-5, cytarabine subcutaneously (SC) twice daily (BID) or once daily (QD) on days 1-10, and venetoclax orally (PO) on days 1-21 of cycle 1. RE-INDUCTION: Patients with \> 5% blasts after cycle 1 receive cladribine IV over 1-2 hours on days 1-5, cytarabine SC BID or QDon days 1-10, and venetoclax PO QD on days 1-21 of cycle 2. REMISSION AFTER INDUCTION: Patients who achieve complete remission (CR)/CR with partial hematologic recovery/CR with incomplete blood count recovery/morphologic leukemia-free state (MLFS) after cycle 1 of induction, receive cladribine IV over 1-2 hours on days 1-3, cytarabine SC BID or QD on days 1-10 and venetoclax PO QD on days 1-21 of cycle 2. CONTINUING THERAPY: Patients who achieve MLFS receive venetoclax PO QD on days 1-21 and azacitidine IV or SC QD on days 1-7 for 2 cycles then cladribine IV over 1-2 hours on days 1-3, cytarabine SQ BID or QD on days 1-10 and venetoclax PO QD on days 1-21 for 2 cycles. Cycles repeat every 28 days and continue to alternate every 2 cycles for up to cycle 18 in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo echocardiography (ECHO) or multigated acquisition scan (MUGA) as clinically indicated on study. Patients also undergo bone marrow aspiration and biopsy and blood sample collection throughout the study. At completion of study treatment, patients are followed up for 2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Given IV or SC
Undergo blood sample collection
Undergo bone marrow aspiration and biopsy
Undergo bone marrow aspiration and biopsy
Given IV
Given SC
Undergo ECHO
Undergo LP
Undergo MUGA
Ancillary studies
Given PO
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, United States
RECRUITINGOHSU Knight Cancer Institute
Portland, Oregon, United States
RECRUITINGComposite complete remission (CRc) rate
CRc is defined as achievement of complete remission (CR), CR with partial hematologic recovery (CRh) or CR with incomplete blood count recovery (CRi) after induction (or re-induction). The CRc rate will be computed on the safety set and presented with a point-estimate and exact binomial 95% confidence interval (CI). CRc will also be modeled with univariable logistic regressions, applied to the efficacy set, to determine if any baseline patient or disease feature is correlated with clinical response.
Time frame: At start of treatment to post-induction disease assessment (Cycle 1 Day 21 or Cycle 2 Day 21. Each cycle is 28 days)
Overall response rate (ORR)
ORR will be defined as the proportion of evaluable participants who attain a partial remission (PR) or better (i.e., CR, CRh, CRi, morphologic leukemia-free state \[MLFS\], or PR) while on investigational treatment. ORR will be computed for both the safety set and the efficacy set and presented with a point estimate and exact binomial CI. ORR will also be modeled with univariable logistic regressions with baseline patient and disease features as predictors and results considered hypothesis-generating.
Time frame: At start of treatment to post-induction disease assessment (Cycle 1 Day 21 or Cycle 2 Day 21. Each cycle is 28 days)
Overall survival (OS)
OS will be computed for both the safety set and the efficacy set, and estimated with the Kaplan-Meier method. Median OS and OS rates at common landmark times (e.g., 1 year, 2 years) will be estimated with 95% log-log CIs. For both sets, univariable Cox models will be fit to OS to assess the impact of baseline patient and disease features with results (i.e., unadjusted hazard ratios and Wald test p-values) considered hypothesis-generating. Median follow-up will utilize a patient's OS measurement and censoring status and be estimated using the reverse Kaplan-Meier method.
Time frame: At start of treatment to death up to 2 years
Event-free survival (EFS)
EFS will be measured from start of treatment to the earliest date of treatment failure, progressive disease, or death from any cause. EFS will be estimated for the efficacy set using the Kaplan-Meier method, with median EFS and EFS rates at common landmark times presented with 05% log-log CIs. EFS will be alternatively estimated upon assigning 1-day event times to participants who never achieve MLFS or better. Univariable Cox models will be fit to EFS to assess the impact of baseline patient and disease features with model results considered hypothesis-generating.
Time frame: At start of treatment to primary refractory disease, hematologic relapse, discontinuation of treatment due to toxicity, disease progression or death up to 2 years
Duration of response (DOR)
DOR will be measured from the first date of documented hematologic response to the earliest of the following occurrences: hematologic relapse, disease progression (clinical or morphologic), or any-cause death. DOR will be estimated by the Kaplan-Meier method. Median DOR will be presented with a 95% log-log CI.
Time frame: At first date of documented ≥ MLFS or better to primary refractory disease, hematologic relapse, discontinuation of treatment due to toxicity, disease progression or death up to 2 years
Incidence of grade ≥ 3 adverse events (AEs)
AEs severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
Time frame: At start of treatment up to 30 days after last dose of any study drug
Percentage of patients achieving 80% relative dose intensity
The percentage of participants achieving 80% relative dose intensity will be reported.
Time frame: At start of treatment to end of treatment up to 1 year and 5 months
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