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Azacitidine, Mitoxantrone Hydrochloride, and Etoposide in Treating Older Patients With Poor-Prognosis Acute Myeloid Leukemia

Phase 1
Terminated
Conditions
Adult Acute Eosinophilic Leukemia
Adult Acute Monoblastic Leukemia
Adult Acute Myeloid Leukemia in Remission
Adult Acute Myelomonocytic Leukemia
Adult Acute Basophilic Leukemia
Untreated Adult Acute Myeloid Leukemia
Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11
Adult Acute Myeloid Leukemia With Maturation
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1
Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL
Interventions
Registration Number
NCT01260714
Lead Sponsor
National Cancer Institute (NCI)
Brief Summary

This phase I trial studies the best dose of azacitidine and to see how well it works with mitoxantrone hydrochloride and etoposide in treating older patients with acute myeloid leukemia that has a lower chance of responding to treatment or higher risk of returning (poor prognosis). Drugs used in chemotherapy, such as azacitidine, mitoxantrone hydrochloride, and etoposide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells.

Detailed Description

PRIMARY OBJECTIVES:

I. To determine the highest tolerated dose of two dosing schedules of azacitidine when combined with mitoxantrone (mitoxantrone hydrochloride) and etoposide (A-NOVE) chemotherapy in poor prognosis older patients with acute myeloid leukemia (AML).

II. To evaluate the toxicity of this regimen.

SECONDARY OBJECTIVES:

I. To determine the complete response (CR) rate and using this regimen. II. To evaluate changes in topoisomerase II activity, deoxyribonucleic acid (DNA) methylation and DNA expression arrays in leukemia cells during azacitidine treatment, and to correlate these changes with responses to A-NOVE chemotherapy.

III. To evaluate relapse-free survival (RFS) and overall survival (OS) in patients receiving post-remission consolidation with A-NOVE in patients achieving CR. (OS follow-up discontinued as of 08/07/2014)

OUTLINE: This is a dose-escalation study of azacitidine.

Patients receive induction therapy comprising azacitidine subcutaneously (SC) once daily (QD) on days 1-7, mitoxantrone hydrochloride IV over 30 minutes, and etoposide IV over 1 hour on days 4-8. Patients may receive up to 2 additional courses of the same treatment as re-induction or consolidation therapy beginning 35-60 days from the start of the previous course.

After completion of study treatment, patients are followed up every 3 months.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
13
Inclusion Criteria
  • Acute myeloid leukemia (AML) as defined by World Health Organization (WHO) criteria, any subtype, de novo or secondary, except acute promyelocytic leukemia (APL)

  • One of the following:

    • Previously untreated, with adverse-risk cytogenetics, including any one of the following:

      • Complete or partial deletion of chromosome 7
      • Complete or partial deletion of chromosome 5
      • At least 3 numerical or structural abnormalities, other than t(15;17), t(8;21) or inv(16) or variant
      • 11q23 abnormalities
      • Inv(3) or variant such as t(3:3)
    • Previously untreated, transformed from prior myelodysplastic syndrome (MDS) or myeloproliferative disorder (MPD) other than CML

    • Persistent leukemia following one cycle of 3+7 induction therapy (cytarabine plus either daunorubicin or idarubicin), any cytogenetic risk group

  • Left ventricular ejection fraction (LVEF) > 50% based on multi gated acquisition scan (MUGA) scan or 2-dimensional (2-D) echocardiogram

  • Serum creatinine =< 1.5 x upper limit of normal (ULN)

  • Serum bilirubin =< 1.5 x ULN

  • Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2.5 x ULN

  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2 (Karnofsky >= 60%)

  • Patients with high initial white blood cell (WBC) should have the WBC reduced to below 50 x 10^9/L with hydroxyurea, to minimize the risk of leukostasis related-complications; hydroxyurea is permitted up to 24 hours prior to starting azacitidine

  • Men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; men should not father a child while participating in this study

  • Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria
  • Patients who have had chemotherapy, radiotherapy or investigational agents within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
  • Patients who have received prior radiation greater than 3000 cGy to marrow producing areas
  • Patients may not be receiving any other investigational agents
  • Patients with active central nervous system (CNS) leukemia; prior CNS leukemia is permitted provided the cerebrospinal fluid has cleared and there is no other evidence of active CNS leukemia
  • Prior therapy for AML with decitabine, azacitidine, mitoxantrone, or etoposide
  • Prior therapy with azacitidine or decitabine for pre-existing MDS
  • History of allergic reactions attributed to decitabine, azacitidine, etoposide, mitoxantrone, or compounds of similar chemical or biologic composition
  • Uncontrolled intercurrent illness including, but not limited to, active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Human immunodeficiency virus (HIV)-positive patients with cluster of differentiation (CD) counts less than 500/mm^3 and/or a history of HIV/acquired immune deficiency syndrome (AIDS)-related complications will be excluded from the study

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (azacitidine, mitoxantrone hydrochloride, etoposide)Mitoxantrone HydrochloridePatients receive induction therapy comprising azacitidine SC QD on days 1-7, mitoxantrone hydrochloride IV over 30 minutes, and etoposide IV over 1 hour on days 4-8. Patients may receive up to 2 additional courses of the same treatment as re-induction or consolidation therapy beginning 35-60 days from the start of the previous course.
Treatment (azacitidine, mitoxantrone hydrochloride, etoposide)Laboratory Biomarker AnalysisPatients receive induction therapy comprising azacitidine SC QD on days 1-7, mitoxantrone hydrochloride IV over 30 minutes, and etoposide IV over 1 hour on days 4-8. Patients may receive up to 2 additional courses of the same treatment as re-induction or consolidation therapy beginning 35-60 days from the start of the previous course.
Treatment (azacitidine, mitoxantrone hydrochloride, etoposide)AzacitidinePatients receive induction therapy comprising azacitidine SC QD on days 1-7, mitoxantrone hydrochloride IV over 30 minutes, and etoposide IV over 1 hour on days 4-8. Patients may receive up to 2 additional courses of the same treatment as re-induction or consolidation therapy beginning 35-60 days from the start of the previous course.
Treatment (azacitidine, mitoxantrone hydrochloride, etoposide)EtoposidePatients receive induction therapy comprising azacitidine SC QD on days 1-7, mitoxantrone hydrochloride IV over 30 minutes, and etoposide IV over 1 hour on days 4-8. Patients may receive up to 2 additional courses of the same treatment as re-induction or consolidation therapy beginning 35-60 days from the start of the previous course.
Primary Outcome Measures
NameTimeMethod
Maximum-tolerated dose of azacitidine that can be safely combined with mitoxantrone hydrochloride and etoposide chemotherapyUp to 2 courses of treatment

The descriptions and grading scales found in the revised National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting.

Secondary Outcome Measures
NameTimeMethod
Complete response rateUp to 4 years
Changes in DNA methylationBaseline to day 4

Nonquantitative comparisons will be made between responders and non-responders with respect to changes in DNA methylation.

Overall survivalFrom the start of study treatment until death from any cause or last follow up, assessed up to 4 years
Relapse-free survivalFrom documentation of CR or MLFS to time of disease recurrence or last follow up, assessed up to 4 years
Changes in gene expressionBaseline to day 4

Nonquantitative comparisons will be made between responders and non-responders with respect to changes in gene expression.

Changes in topoisomerase II levelsBaseline to day 4

These will be compared between responders (i.e. those achieving either CR or morphologic leukemia-free state \[MLFS\]) vs. non-responders (those not achieving CR/MLFS after 1-2 induction cycles), with 95% confidence intervals and 2-tailed t-tests of significance.

Trial Locations

Locations (2)

Juravinski Cancer Centre at Hamilton Health Sciences

🇨🇦

Hamilton, Ontario, Canada

University Health Network-Princess Margaret Hospital

🇨🇦

Toronto, Ontario, Canada

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