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Clofarabine or Daunorubicin Hydrochloride and Cytarabine Followed By Decitabine or Observation in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia

Phase 3
Completed
Conditions
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Adult Acute Monoblastic Leukemia (M5a)
Adult Acute Megakaryoblastic Leukemia (M7)
Adult Acute Myeloblastic Leukemia Without Maturation (M1)
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Adult Acute Myeloid Leukemia With Del(5q)
Adult Pure Erythroid Leukemia (M6b)
Secondary Acute Myeloid Leukemia
Adult Acute Myeloblastic Leukemia With Maturation (M2)
Interventions
Other: Observation
Procedure: Allogeneic hematopoietic stem cell transplantation
Registration Number
NCT02085408
Lead Sponsor
ECOG-ACRIN Cancer Research Group
Brief Summary

This randomized phase III trial studies clofarabine to see how well it works compared with daunorubicin hydrochloride and cytarabine when followed by decitabine or observation in treating older patients with newly diagnosed acute myeloid leukemia. Drugs used in chemotherapy, such as clofarabine, daunorubicin hydrochloride, cytarabine, and decitabine, 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. It is not yet known which chemotherapy regimen is more effective in treating acute myeloid leukemia.

Detailed Description

PRIMARY OBJECTIVES:

I. To evaluate the effect of clofarabine induction and consolidation therapy on overall survival in comparison with standard therapy (daunorubicin \[daunorubicin hydrochloride\] \& cytarabine) in newly-diagnosed acute myeloid leukemia (AML) patients age \>= 60 years.

SECONDARY OBJECTIVES:

I. To evaluate complete remission (CR) rates, duration of remission, and toxicity/treatment-related mortality of clofarabine in comparison with standard therapy (daunorubicin \& cytarabine) in newly-diagnosed AML patients age \>= 60 years.

II. To evaluate the feasibility of consolidation with reduced-intensity conditioning and allogeneic hematopoietic stem cell transplantation from human leukocyte antigen (HLA)-identical donors in patients who achieve a response to induction therapy, including the incidence of successful engraftment, acute and chronic graft-versus-host disease, transplant-related mortality, and its impact on overall survival in comparison to patients receiving chemotherapy.

III. To evaluate the duration of remission and disease-free survival of patients in complete remission following completion of consolidation therapy who are subsequently randomized to receive scheduled low-dose decitabine maintenance in comparison with observation.

IV. To perform expression and methylation profiling on all patients receiving decitabine and to correlate their integrated epigenetic signatures with response to decitabine.

V. To examine the epigenetic profiles of remission marrow in patients randomized to observation vs. decitabine to determine whether epigenetic signature of apparently morphologically normal bone marrow is predictive of relapse or response to decitabine maintenance.

VI. To explore the possible association of response to clofarabine with ABC-transporter P-glycoprotein (Pgp).

VII. To assess the intensity of expression of CXC chemokine receptor type 4 (CXCR4) on diagnostic leukemia cells and to correlate this parameter with other established prognostic factors.

VIII. To assess the entire spectrum of somatic mutations and affected pathways at diagnosis of AML and elucidate the association between gene mutation and outcome.

IX. To examine the impact of smoking, obesity, regular acetaminophen use, regular aspirin use, benzene exposure, living in a rural/farm environment and some other underlying exposures and lifestyle factors associated with AML development on overall survival (OS).

X. To investigate potential correlative results between array comparative genomic hybridization (CGH) findings and acute myeloid leukemia patient characteristics.

TERTIARY OBJECTIVES:

I. To compare health-related quality of life (QOL) (physical, functional, leukemia-specific well-being) and fatigue in elderly AML patients receiving standard induction therapy with those receiving clofarabine.

II. To measure the change in health-related QOL that occurs over time (within treatment groups).

III. To comprehensively assess patient function at the time of study enrollment.

IV. To determine if components of a comprehensive geriatric assessment or QOL scales predict ability to complete AML treatment.

V. To describe the impact of transplant on QOL in AML patients above age 60.

OUTLINE:

INDUCTION THERAPY: Patients are randomized to 1 of 2 treatment arms.

ARM A (STANDARD THERAPY): Patients receive daunorubicin hydrochloride at 60 mg/m\^2 intravenously (IV) over 10-15 minutes on days 1-3 and cytarabine at 100 mg/m\^2 IV continuously on days 1-7. Patients with residual disease or those who do not achieve an aplastic bone marrow on day 12-14 (i.e., \< 5% blasts and \< 20% cellularity or markedly/moderately hypocellular) may receive a second course of induction therapy beginning no sooner than day 14.

ARM B: Patients receive clofarabine at 30 mg/m\^2 IV over 1 hour on days 1-5. Patients with residual disease or those who do not achieve an aplastic bone marrow on day 12-14 (i.e., \< 5% blasts and \< 20% cellularity or markedly/moderately hypocellular) may receive a second course of induction therapy beginning no sooner than day 21 and no later than day 56.

Patients who achieve a complete remission (CR) or CR with incomplete marrow recovery (CRi) after induction therapy proceed to consolidation therapy (Arms C and D). Patients who are 60-69 years of age who achieve a "morphologic leukemia-free state" after induction therapy and who have an HLA-identical donor proceed to allogeneic stem cell transplantation.

CONSOLIDATION THERAPY: Beginning within 60 days after documentation of CR or CRi, patients receive consolidation therapy in the same arm they were randomized to for induction therapy.

ARM C (STANDARD THERAPY): Patients receive cytarabine at 1500 mg/m\^2 IV over 1 hour once or twice daily on days 1-6. Treatment repeats every 4-6 weeks for 2 courses.

ARM D: Patients receive clofarabine at 20 mg/m\^2 IV over 1 hour on days 1-5. Treatment repeats every 4-6 weeks for 2 courses.

Patients who remain in CR after completion of consolidation therapy are randomized to one of the two maintenance therapy arms (Arms E and F).

MAINTENANCE THERAPY: Beginning within 60 days after completion of consolidation therapy, patients receive maintenance therapy and are randomized to 1 of 2 arms. Patients not eligible for randomization to decitabine maintenance after recovery from consolidation will be followed according to Arm E.

ARM E: Patients undergo observation monthly for 12 months.

ARM F: Patients receive decitabine at 20 mg/m\^2 IV over 1 hour on days 1-3. Treatment repeats every 4 weeks for 12 months the absence of unacceptable toxicity.

ALLOGENEIC STEM CELL TRANSPLANTATION WITH REDUCED-INTENSITY CONDITIONING REGIMEN (Arm G): Patients begin reduced-intensity conditioning 30-90 days after the initiation of induction therapy.

CONDITIONING REGIMEN: Patients receive fludarabine phosphate at 30 mg/m\^2 IV over 30 minutes on days -7 to -3, busulfan at 0.8 mg/kg IV over 2 hours every 6 hours on days -4 and -3 (for a total of 8 doses), and anti-thymocyte globulin at 2.5 mg/kg/day IV over 4-6 hours on days -4 to -2.

TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplantation on day 0.

After completion of study treatment, patients are followed up every 3 months for 4 years, every 6 months for 1 year, and then annually thereafter.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
727
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
A (Induction:daunorubicin/cytarabine; consolidation:cytarabine; maintenance:observation/transplant)ObservationSee Detailed Description
A (Induction:daunorubicin/cytarabine; consolidation:cytarabine; maintenance:observation/transplant)Allogeneic hematopoietic stem cell transplantationSee Detailed Description
B (Induction: clofarabine; Consolidation: clofarabine; Maintenance: decitabine or transplant)Allogeneic hematopoietic stem cell transplantationSee Detailed Description
A (Induction:daunorubicin/cytarabine; consolidation:cytarabine; maintenance:observation/transplant)DaunorubicinSee Detailed Description
A (Induction:daunorubicin/cytarabine; consolidation:cytarabine; maintenance:observation/transplant)CytarabineSee Detailed Description
B (Induction: clofarabine; Consolidation: clofarabine; Maintenance: decitabine or transplant)ClofarabineSee Detailed Description
B (Induction: clofarabine; Consolidation: clofarabine; Maintenance: decitabine or transplant)DecitabineSee Detailed Description
Primary Outcome Measures
NameTimeMethod
Overall SurvivalAssessed every 3 months for 4 years and then every 6 months for 1 year

Overall survival is defined as the time from randomization to death or date last known alive.

Secondary Outcome Measures
NameTimeMethod
Proportion of Patients With Complete RemissionAssessed every 3 months for 4 years and then every 6 months for 1 year

Patients are required to have all of the following to be considered as having a completion remission (CR).

* Peripheral Blood Counts

1. Neutrophil count \> 1.0 x 10\^9 /L

2. Platelet count ≥ 100 x 10\^9 /L

3. Reduced hemoglobin concentration or hematocrit has no bearing on remission status

4. Leukemic blasts must not be present in the peripheral blood

* Bone Marrow Aspirate and Biopsy

1. Cellularity of bone marrow biopsy must be \> 20% with maturation of all cell lines

2. \< 5% blasts by morphologic review

3. Auer rods must not be detectable

* Extramedullary leukemia, such as CNS or soft tissue involvement, must not be present

Overall Survival by Donor StatusAssessed every 3 months for 4 years and then every 6 months for 1 year

Overall survival is defined as time between achieving leukemia-free state after induction therapy to death from any cause or date last known alive. The association between overall survival and donor status was evaluated regardless of assigned treatment arms. Patients with transplant donor information (either had donor or did not have a donor) reported after achieving CR/Cri/leukemia-free state post induction therapy were included in this analysis.

Disease-free Survival for MaintenanceAssessed every 3 months for 4 years and then every 6 months for 1 year

DFS for maintenance comparison is defined as the time from maintenance randomization until relapse or death of any cause. The censored follow-up time for patients without relapse and death information is the date of last contact. Only patients who remained in CR or CRi after completion of consolidation therapy that were randomized to either observation or decitabine in the maintenance Step were included in this analysis.

Trial Locations

Locations (269)

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Mayo Clinic in Arizona

🇺🇸

Scottsdale, Arizona, United States

Arizona Cancer Center at University Medical Center North

🇺🇸

Tucson, Arizona, United States

University of Arizona Health Sciences Center

🇺🇸

Tucson, Arizona, United States

The Medical Center of Aurora

🇺🇸

Aurora, Colorado, United States

Boulder Community Hospital

🇺🇸

Boulder, Colorado, United States

Penrose-Saint Francis Healthcare

🇺🇸

Colorado Springs, Colorado, United States

Saint Anthony Central Hospital

🇺🇸

Denver, Colorado, United States

Porter Adventist Hospital

🇺🇸

Denver, Colorado, United States

Exempla Saint Joseph Hospital

🇺🇸

Denver, Colorado, United States

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University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States

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