MedPath

Combination Chemotherapy With or Without Gemtuzumab in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia

Registration Number
NCT00372593
Lead Sponsor
Children's Oncology Group
Brief Summary

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Monoclonal antibodies, such as gemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Giving combination chemotherapy together with gemtuzumab may kill more cancer cells. It is not yet known whether combination chemotherapy is more effective with or without gemtuzumab in treating patients with newly diagnosed acute myeloid leukemia.

PURPOSE: This randomized phase III trial is studying combination chemotherapy and gemtuzumab to see how well they work compared with combination chemotherapy alone in treating young patients with newly diagnosed acute myeloid leukemia.

Detailed Description

OBJECTIVES:

Primary

* Compare the event-free survival (EFS) and overall survival (OS) of young patients with newly diagnosed acute myeloid leukemia (AML) treated with conventional combination chemotherapy with vs without gemtuzumab ozogamicin (GMTZ).

Secondary

* Compare the remission induction rates after two courses of therapy in these patients.

* Compare disease-free survival and OS in patients who are eligible for an human leukocyte antigen (HLA)-matched family donor (MFD) stem cell transplant (SCT) by virtue of their risk classification, with patients assigned to MFD SCT if a MFD is available, or to chemotherapy if a MFD is not available.

* Determine the outcome of patients with Down syndrome who are 4 years of age or older at diagnosis and treated with conventional combination chemotherapy without GMTZ.

* Compare the EFS and OS of patients with de novo AML treated with conventional combination chemotherapy with vs without GMTZ censoring MFD SCT recipients.

* Determine the prevalence and prognostic significance of molecular abnormalities of KIT, CCAAT-enhancer binding protein alpha (CEBPα) and MLL-Partial tandem duplications (PTD) genes in these patients.

* Determine the leukemic involvement of hematopoietic early progenitor and its role in defining response to therapy.

* Assess the ability of a second-generation flow cytometric assay to predict patients at high risk for relapse during periods of clinical remission.

* Examine whether GMTZ significantly improves EFS and OS in patients with higher CD33 concentrations/intensity.

* Examine whether GMTZ significantly improves complete remission, EFS, and OS in each of the cytogenetic risk groups (high-, intermediate-, and low-risk) identified in prior Medical Research Council trials.

* Utilize fluorescence in situ hybridization (FISH) analysis to identify variant patterns among subgroups of patients who demonstrate the same G-banded chromosomal abnormality (e.g., inv\[16\]/t\[16;16\], t\[8;21\], 11q23 abnormality) and determine whether these variant patterns account for the heterogeneity of responses to therapy.

* Examine the impact of complex karyotypes (≥ 3, ≥ 4, and ≥ 5 abnormalities) on OS and EFS in intermediate-risk patients for whom no high-risk or low-risk cytogenetic abnormalities exist.

OUTLINE: This is a randomized, multicenter study. Patients are stratified according to relapse risk (high vs intermediate vs low). Patients are randomized to 1 of 2 treatment arms. Patients with Down syndrome are nonrandomly assigned to arm I (but do not undergo allogeneic stem cell transplant \[SCT\]).

* Arm I (standard therapy):

* Induction 1: Patients receive cytarabine IT at the time of diagnosis or on day 1\*. Patients also receive cytarabine IV on days 1-10, daunorubicin hydrochloride IV over 6 hours on days 1, 3, and 5, and etoposide IV over 4 hours on days 1-5. After 3 weeks of rest, all patients (regardless of remission status) proceed to induction 2.

NOTE: \*Patients with Central Nervous System (CNS) disease receive cytarabine IT twice weekly until the cerebrospinal fluid is clear, followed by two additional IT treatments. Patients with refractory CNS leukemia after 6 doses of IT treatment are removed from the study.

* Induction 2: Patients receive cytarabine IT on day 1, cytarabine IV on days 1-8, daunorubicin hydrochloride IV over 6 hours on days 1, 3, and 5, and etoposide IV over 4 hours on days 1-5. After 3 weeks of rest, patients in complete remission (CR) proceed to intensification 1. Patients with refractory disease are removed from protocol therapy.

* Intensification 1: Patients receive cytarabine IT on day 1, high-dose cytarabine IV over 1 hour on days 1-5, and etoposide IV over 1 hour on days 1-5. After 3 weeks of rest, patients in remission proceed to intensification 2, followed by intensification 3. Patients in remission proceed to allogeneic SCT 2-8 weeks after blood counts recover. Patients with high-risk disease with an alternative donor proceed to intensification 2 and 3, followed by allogeneic SCT. Patients not in remission are removed from protocol therapy.

* Intensification 2: Patients receive cytarabine IT on day 1, high-dose cytarabine IV over 2 hours on days 1-4, and mitoxantrone hydrochloride IV over 1 hour on days 3-6. After 3 weeks of rest, patients proceed to intensification 3.

* Intensification 3: Patients receive high-dose cytarabine IV over 3 hours on days 1, 2, 8, and 9 and asparaginase intramuscularly on days 2 and 9.

* Arm II:

* Induction 1: Patients receive treatment as in induction 1 of arm I. Patients also receive gemtuzumab ozogamicin (GMTZ) IV over 2 hours on day 6.

* Induction 2: Patients receive treatment as in induction 2 of arm I.

* Intensification 1: Patients receive treatment as in intensification 1 of arm I.

* Intensification 2: Patients receive treatment as in intensification 2 of arm I. Patients also receive GMTZ IV over 2 hours on day 7.

* Intensification 3: Patients receive treatment as in intensification 3 of arm I.

* Allogeneic SCT (for patients with intermediate- or high-risk disease):

* MFD: Patients receive a conditioning regimen comprising busulfan IV over 2 hours every 6 hours on days -9 to -6 and cyclophosphamide IV over 1 hour on days -5 to -2. Patients undergo allogeneic SCT on day 0. Patients receive cyclosporine IV or orally twice daily on days -1 to 180 and methotrexate IV on days 1, 3, 6, and 11. Patients receive graft-vs-host disease (GVHD) prophylaxis comprising cyclosporine IV over 1-4 hours or orally twice daily on days -1 to 180 and methotrexate IV on days 1, 3, 6, and 11.

* Matched alternative donor: Patients receive a conditioning regimen comprising busulfan and cyclophosphamide as above. Patients also receive antithymocyte globulin IV over 6-8 hours on days -3 to -1. Patients then undergo allogeneic SCT and receive GVHD prophylaxis as above.

After completion of study treatment, patients are followed periodically for 3 years and then annually thereafter.

PROJECTED ACCRUAL: A total of 1,012 patients will be accrued for this study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
1070
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm B: Experimental - with GMTZ, AML Pts w/out Down Syndromedaunorubicin hydrochloridePts receive IT ARA-C at diagnosis or on day 1 of treatment or twice a week for up to six doses. They also receive an infusion of ARA-C on days 1-10; a 6-hr infusion of daunorubicin on days 1, 3, \& 5; a 4-hr infusion of etoposide on days 1-5; and a 2-hr infusion of GMTZ - gemtuzumab ozogamicin (Mylotarg) on day 6. After 3 wks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 wks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hr infusion of mitoxantrone hydrochloride on days 3-6. They also receive a 2-hr infusion of gemtuzumab on day 7. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Pts w/out Down Syndromedaunorubicin hydrochloridePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm B: Experimental - with GMTZ, AML Pts w/out Down Syndromemitoxantrone hydrochloridePts receive IT ARA-C at diagnosis or on day 1 of treatment or twice a week for up to six doses. They also receive an infusion of ARA-C on days 1-10; a 6-hr infusion of daunorubicin on days 1, 3, \& 5; a 4-hr infusion of etoposide on days 1-5; and a 2-hr infusion of GMTZ - gemtuzumab ozogamicin (Mylotarg) on day 6. After 3 wks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 wks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hr infusion of mitoxantrone hydrochloride on days 3-6. They also receive a 2-hr infusion of gemtuzumab on day 7. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Pts w/out Down Syndromemitoxantrone hydrochloridePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Patients with Down Syndromedaunorubicin hydrochloridePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Patients with Down Syndromemitoxantrone hydrochloridePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Pts w/out Down SyndromeasparaginasePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Pts w/out Down SyndromecytarabinePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm B: Experimental - with GMTZ, AML Pts w/out Down SyndromecytarabinePts receive IT ARA-C at diagnosis or on day 1 of treatment or twice a week for up to six doses. They also receive an infusion of ARA-C on days 1-10; a 6-hr infusion of daunorubicin on days 1, 3, \& 5; a 4-hr infusion of etoposide on days 1-5; and a 2-hr infusion of GMTZ - gemtuzumab ozogamicin (Mylotarg) on day 6. After 3 wks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 wks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hr infusion of mitoxantrone hydrochloride on days 3-6. They also receive a 2-hr infusion of gemtuzumab on day 7. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm B: Experimental - with GMTZ, AML Pts w/out Down SyndromeasparaginasePts receive IT ARA-C at diagnosis or on day 1 of treatment or twice a week for up to six doses. They also receive an infusion of ARA-C on days 1-10; a 6-hr infusion of daunorubicin on days 1, 3, \& 5; a 4-hr infusion of etoposide on days 1-5; and a 2-hr infusion of GMTZ - gemtuzumab ozogamicin (Mylotarg) on day 6. After 3 wks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 wks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hr infusion of mitoxantrone hydrochloride on days 3-6. They also receive a 2-hr infusion of gemtuzumab on day 7. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Pts w/out Down SyndromeetoposidePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm B: Experimental - with GMTZ, AML Pts w/out Down SyndromeetoposidePts receive IT ARA-C at diagnosis or on day 1 of treatment or twice a week for up to six doses. They also receive an infusion of ARA-C on days 1-10; a 6-hr infusion of daunorubicin on days 1, 3, \& 5; a 4-hr infusion of etoposide on days 1-5; and a 2-hr infusion of GMTZ - gemtuzumab ozogamicin (Mylotarg) on day 6. After 3 wks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 wks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hr infusion of mitoxantrone hydrochloride on days 3-6. They also receive a 2-hr infusion of gemtuzumab on day 7. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm B: Experimental - with GMTZ, AML Pts w/out Down Syndromegemtuzumab ozogamicinPts receive IT ARA-C at diagnosis or on day 1 of treatment or twice a week for up to six doses. They also receive an infusion of ARA-C on days 1-10; a 6-hr infusion of daunorubicin on days 1, 3, \& 5; a 4-hr infusion of etoposide on days 1-5; and a 2-hr infusion of GMTZ - gemtuzumab ozogamicin (Mylotarg) on day 6. After 3 wks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 wks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hr infusion of mitoxantrone hydrochloride on days 3-6. They also receive a 2-hr infusion of gemtuzumab on day 7. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Patients with Down SyndromecytarabinePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Patients with Down SyndromeasparaginasePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Arm A: Standard Arm - No GMTZ, AML Patients with Down SyndromeetoposidePatients receive intrathecal (IT) cytarabine (ARA-C) at diagnosis or on day 1 of treatment or twice a week for up to 6 doses. They also receive an infusion of ARA-C on days 1-10; a 6-hour infusion of daunorubicin hydrochloride on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, patients receive IT ARA-C on day 1. They also receive an infusion of ARA-C on days 1-8; a 6-hr infusion of daunorubicin on days 1, 3, and 5; and a 4-hr infusion of etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1 and 1-hr infusions of ARA-C and etoposide on days 1-5. After 3 weeks of rest, some patients receive IT ARA-C on day 1; a 2-hr infusion of ARA-C on days 1-4; and a 1-hour infusion of mitoxantrone on days 3-6. After 3 weeks of rest, they receive a 3-hr infusion of ARA-C on days 1, 2, 8, and 9 and an injection of asparaginase on days 2 and 9.
Primary Outcome Measures
NameTimeMethod
Event-free Survival at 3 YearsTime from study entry to time of induction failure, relapse, or death, assessed at 3 years

The Kaplan-Meier method will be used to calculate estimates of Event Free Survival (EFS). The log-rank test will be used to compare survival between treatment groups. Analysis of EFS of Down syndrome patients will be performed separately. Monitoring for efficacy of GMTZ with respect to Overall Survival (OS) and EFS will utilize monitoring based on the Lan-DeMets criterion with α-spending function αt\^2 (truncated at 3 standard deviations) and 2.5% type I error.

Overall Survival at 3 YearsTime from study entry, assessed at 3 years

The Kaplan-Meier method will be used to calculate estimates of OS. Analysis of OS of Down syndrome patients will be performed separately. Monitoring for efficacy of GMTZ with respect to OS and EFS will utilize monitoring based on the Lan-DeMets criterion with α-spending function αt\^2 (truncated at 3 standard deviations) and 2.5% type I error.

Secondary Outcome Measures
NameTimeMethod
Time to Marrow RecoveryAt 25 days after treatment with Induction I, Induction II, and Intensification I

Mean time to ANC recovery - defined as ANC greater than 500/MicroLiter for 3 consecutive days.

Disease-free Survival (DFS)At 3 years from end of Intensification I

Time from end of Intensification I to relapse, death or last contact

Toxicities, Including Infectious ComplicationsFrom the time therapy is initiated, assessed up to 10 years

Number of participants with at least one grade 3 or higher adverse event during therapy.

Remission Induction Rate After 2 Courses of Induction TherapyAfter 2 courses of induction (I and II) therapy, assessed for up to 10 years

Patients without an evaluable bone marrow at the end of Induction I will be excluded from the calculation of remission rate after 2 courses of therapy because their responses are not evaluable. The following patients will be considered to not be in complete remission (CR) after 2 courses of therapy: (1) patients who die during Induction I and II; (2) patients with ≥ 5% blasts or extramedullary disease at the end of Induction II.

MortalityDuring the first three courses of therapy

Number of participants who died during the first three courses of therapy.

Trial Locations

Locations (198)

University of Illinois Cancer Center

🇺🇸

Chicago, Illinois, United States

Childrens Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Arkansas Cancer Research Center at University of Arkansas for Medical Sciences

🇺🇸

Little Rock, Arkansas, United States

Kaiser Permanente Medical Center - Oakland

🇺🇸

Oakland, California, United States

Alfred I. duPont Hospital for Children

🇺🇸

Wilmington, Delaware, United States

Southern California Permanente Medical Group

🇺🇸

Downey, California, United States

Memorial Cancer Institute at Memorial Regional Hospital

🇺🇸

Hollywood, Florida, United States

Loma Linda University Cancer Institute at Loma Linda University Medical Center

🇺🇸

Loma Linda, California, United States

City of Hope Comprehensive Cancer Center

🇺🇸

Duarte, California, United States

University of South Alabama Mitchell Cancer Institute

🇺🇸

Mobile, Alabama, United States

Banner Desert Medical Center

🇺🇸

Mesa, Arizona, United States

Samuel Oschin Comprehensive Cancer Institute at Cedars-Sinai Medical Center

🇺🇸

Los Angeles, California, United States

Arizona Cancer Center at University of Arizona Health Sciences Center

🇺🇸

Tucson, Arizona, United States

Children's Hospital of The King's Daughters

🇺🇸

Norfolk, Virginia, United States

CCOP - Scott and White Hospital

🇺🇸

Temple, Texas, United States

Medical City Dallas Hospital

🇺🇸

Dallas, Texas, United States

Sanford Cancer Center at Sanford USD Medical Center

🇺🇸

Sioux Falls, South Dakota, United States

University of Virginia Cancer Center

🇺🇸

Charlottesville, Virginia, United States

Westmead Institute for Cancer Research at Westmead Hospital

🇦🇺

Westmead, New South Wales, Australia

Covenant Children's Hospital

🇺🇸

Lubbock, Texas, United States

Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas

🇺🇸

Dallas, Texas, United States

Driscoll Children's Hospital

🇺🇸

Corpus Christi, Texas, United States

Children's Hospital of Western Ontario

🇨🇦

London, Ontario, Canada

Centre Hospitalier Universitaire de Quebec

🇨🇦

Quebec, Canada

Women's and Children's Hospital

🇦🇺

North Adelaide, South Australia, Australia

Christchurch Hospital

🇳🇿

Christchurch, New Zealand

Hospital for Sick Children

🇨🇦

Toronto, Ontario, Canada

Starship Children's Health

🇳🇿

Grafton, New Zealand

Baylor University Medical Center - Houston

🇺🇸

Houston, Texas, United States

University of Miami Sylvester Comprehensive Cancer Center - Miami

🇺🇸

Miami, Florida, United States

Indiana University Melvin and Bren Simon Cancer Center

🇺🇸

Indianapolis, Indiana, United States

St. Vincent Indianapolis Hospital

🇺🇸

Indianapolis, Indiana, United States

Rady Children's Hospital - San Diego

🇺🇸

San Diego, California, United States

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

UCSF Helen Diller Family Comprehensive Cancer Center

🇺🇸

San Francisco, California, United States

Children's Hospitals and Clinics of Minnesota - Minneapolis

🇺🇸

Minneapolis, Minnesota, United States

Vanderbilt-Ingram Cancer Center

🇺🇸

Nashville, Tennessee, United States

University of Texas Health Science Center at San Antonio

🇺🇸

San Antonio, Texas, United States

Methodist Children's Hospital of South Texas

🇺🇸

San Antonio, Texas, United States

Primary Children's Medical Center

🇺🇸

Salt Lake City, Utah, United States

Jonsson Comprehensive Cancer Center at UCLA

🇺🇸

Los Angeles, California, United States

Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

CCOP - MeritCare Hospital

🇺🇸

Fargo, North Dakota, United States

Children's Hospital of Orange County

🇺🇸

Orange, California, United States

Children's Hospital Central California

🇺🇸

Madera, California, United States

Children's Hospital and Research Center Oakland

🇺🇸

Oakland, California, United States

Santa Barbara Cottage Children's Hospital

🇺🇸

Santa Barbara, California, United States

Jonathan Jaques Children's Cancer Center at Miller Children's Hospital

🇺🇸

Torrance, California, United States

Broward General Medical Center Cancer Center

🇺🇸

Fort Lauderdale, Florida, United States

Miami Children's Hospital

🇺🇸

Miami, Florida, United States

Baptist-South Miami Regional Cancer Program

🇺🇸

Miami, Florida, United States

Sacred Heart Cancer Center at Sacred Heart Hospital

🇺🇸

Pensacola, Florida, United States

All Children's Hospital

🇺🇸

Saint Petersburg, Florida, United States

Kaplan Cancer Center at St. Mary's Medical Center

🇺🇸

West Palm Beach, Florida, United States

AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus

🇺🇸

Atlanta, Georgia, United States

Winship Cancer Institute of Emory University

🇺🇸

Atlanta, Georgia, United States

MBCCOP - Medical College of Georgia Cancer Center

🇺🇸

Augusta, Georgia, United States

Curtis and Elizabeth Anderson Cancer Institute at Memorial Health University Medical Center

🇺🇸

Savannah, Georgia, United States

Tripler Army Medical Center

🇺🇸

Tripler AMC, Hawaii, United States

Mountain States Tumor Institute at St. Luke's Regional Medical Center

🇺🇸

Boise, Idaho, United States

Children's Memorial Hospital - Chicago

🇺🇸

Chicago, Illinois, United States

Cardinal Bernardin Cancer Center at Loyola University Medical Center

🇺🇸

Maywood, Illinois, United States

University of Chicago Cancer Research Center

🇺🇸

Chicago, Illinois, United States

Advocate Christ Medical Center

🇺🇸

Oak Lawn, Illinois, United States

Keyser Family Cancer Center at Advocate Hope Children's Hospital

🇺🇸

Oak Lawn, Illinois, United States

Advocate Lutheran General Cancer Care Center

🇺🇸

Park Ridge, Illinois, United States

Simmons Cooper Cancer Institute

🇺🇸

Springfield, Illinois, United States

Holden Comprehensive Cancer Center at University of Iowa

🇺🇸

Iowa City, Iowa, United States

Alvin and Lois Lapidus Cancer Institute at Sinai Hospital

🇺🇸

Baltimore, Maryland, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Greenebaum Cancer Center at University of Maryland Medical Center

🇺🇸

Baltimore, Maryland, United States

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

🇺🇸

Baltimore, Maryland, United States

Floating Hospital for Children at Tufts - New England Medical Center

🇺🇸

Boston, Massachusetts, United States

Baystate Regional Cancer Program at D'Amour Center for Cancer Care

🇺🇸

Springfield, Massachusetts, United States

Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

UMASS Memorial Cancer Center - University Campus

🇺🇸

Worcester, Massachusetts, United States

Hurley Medical Center

🇺🇸

Flint, Michigan, United States

Butterworth Hospital at Spectrum Health

🇺🇸

Grand Rapids, Michigan, United States

Van Elslander Cancer Center at St. John Hospital and Medical Center

🇺🇸

Grosse Pointe Woods, Michigan, United States

CCOP - Kalamazoo

🇺🇸

Kalamazoo, Michigan, United States

Breslin Cancer Center at Ingham Regional Medical Center

🇺🇸

Lansing, Michigan, United States

Ellis Fischel Cancer Center at University of Missouri - Columbia

🇺🇸

Columbia, Missouri, United States

Masonic Cancer Center at University of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis

🇺🇸

Saint Louis, Missouri, United States

Newark Beth Israel Medical Center

🇺🇸

Newark, New Jersey, United States

University of New Mexico Cancer Center

🇺🇸

Albuquerque, New Mexico, United States

Albany Medical Center Hospital

🇺🇸

Albany, New York, United States

Brooklyn Hospital Center

🇺🇸

Brooklyn, New York, United States

Barbara Ann Karmanos Cancer Institute

🇺🇸

Detroit, Michigan, United States

Winthrop University Hospital

🇺🇸

Mineola, New York, United States

Schneider Children's Hospital

🇺🇸

New Hyde Park, New York, United States

New York Weill Cornell Cancer Center at Cornell University

🇺🇸

New York, New York, United States

NYU Cancer Institute at New York University Medical Center

🇺🇸

New York, New York, United States

Mount Sinai Medical Center

🇺🇸

New York, New York, United States

Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center

🇺🇸

New York, New York, United States

James P. Wilmot Cancer Center at University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

SUNY Upstate Medical University Hospital

🇺🇸

Syracuse, New York, United States

Stony Brook University Cancer Center

🇺🇸

Stony Brook, New York, United States

Mission Hospitals - Memorial Campus

🇺🇸

Asheville, North Carolina, United States

Presbyterian Cancer Center at Presbyterian Hospital

🇺🇸

Charlotte, North Carolina, United States

Blumenthal Cancer Center at Carolinas Medical Center

🇺🇸

Charlotte, North Carolina, United States

Duke Comprehensive Cancer Center

🇺🇸

Durham, North Carolina, United States

Leo W. Jenkins Cancer Center at ECU Medical School

🇺🇸

Greenville, North Carolina, United States

Akron Children's Hospital

🇺🇸

Akron, Ohio, United States

Rainbow Babies and Children's Hospital

🇺🇸

Cleveland, Ohio, United States

Cleveland Clinic Taussig Cancer Center

🇺🇸

Cleveland, Ohio, United States

Nationwide Children's Hospital

🇺🇸

Columbus, Ohio, United States

Children's Medical Center - Dayton

🇺🇸

Dayton, Ohio, United States

Toledo Hospital

🇺🇸

Toledo, Ohio, United States

Medical University of Ohio Cancer Center

🇺🇸

Toledo, Ohio, United States

Oklahoma University Cancer Institute

🇺🇸

Oklahoma City, Oklahoma, United States

Lehigh Valley Hospital - Muhlenberg

🇺🇸

Bethlehem, Pennsylvania, United States

Geisinger Cancer Institute at Geisinger Health

🇺🇸

Danville, Pennsylvania, United States

Penn State Cancer Institute at Milton S. Hershey Medical Center

🇺🇸

Hershey, Pennsylvania, United States

Penn State Children's Hospital

🇺🇸

Hershey, Pennsylvania, United States

St. Christopher's Hospital for Children

🇺🇸

Philadelphia, Pennsylvania, United States

Children's Hospital of Philadelphia

🇺🇸

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

Palmetto Health South Carolina Cancer Center

🇺🇸

Columbia, South Carolina, United States

Texas Tech University Health Sciences Center School of Medicine - Amarillo

🇺🇸

Amarillo, Texas, United States

Naval Medical Center - Portsmouth

🇺🇸

Portsmouth, Virginia, United States

Mary Bridge Children's Hospital and Health Center - Tacoma

🇺🇸

Tacoma, Washington, United States

Marshfield Clinic - Marshfield Center

🇺🇸

Marshfield, Wisconsin, United States

St. Vincent Hospital Regional Cancer Center

🇺🇸

Green Bay, Wisconsin, United States

Royal Children's Hospital

🇦🇺

Herston, Queensland, Australia

Princess Margaret Hospital for Children

🇦🇺

Perth, Western Australia, Australia

CancerCare Manitoba

🇨🇦

Winnipeg, Manitoba, Canada

UAB Comprehensive Cancer Center

🇺🇸

Birmingham, Alabama, United States

Legacy Emanuel Hospital and Health Center and Children's Hospital

🇺🇸

Portland, Oregon, United States

Knight Cancer Institute at Oregon Health and Science University

🇺🇸

Portland, Oregon, United States

Phoenix Children's Hospital

🇺🇸

Phoenix, Arizona, United States

Tulane Cancer Center Office of Clinical Research

🇺🇸

Alexandria, Louisiana, United States

Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School

🇺🇸

New Brunswick, New Jersey, United States

Nemours Children's Clinic

🇺🇸

Jacksonville, Florida, United States

Norris Cotton Cancer Center at Dartmouth-Hitchcock Medical Center

🇺🇸

Lebanon, New Hampshire, United States

Children's National Medical Center

🇺🇸

Washington, District of Columbia, United States

St. Joseph's Hospital and Medical Center

🇺🇸

Paterson, New Jersey, United States

Maine Children's Cancer Program at Barbara Bush Children's Hospital

🇺🇸

Portland, Maine, United States

Swiss Pediatric Oncology Group Geneva

🇨🇭

Geneva, Switzerland

Janeway Children's Health and Rehabilitation Centre

🇨🇦

Saint John's, Newfoundland and Labrador, Canada

IWK Health Centre

🇨🇦

Halifax, Nova Scotia, Canada

East Tennessee Children's Hospital

🇺🇸

Knoxville, Tennessee, United States

Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center

🇺🇸

Farmington, Connecticut, United States

CancerCare of Maine at Eastern Maine Medical Center

🇺🇸

Bangor, Maine, United States

West Virginia University Health Sciences Center - Charleston

🇺🇸

Charleston, West Virginia, United States

Walter Reed Army Medical Center

🇺🇸

Washington, District of Columbia, United States

University of Mississippi Cancer Clinic

🇺🇸

Jackson, Mississippi, United States

T.C. Thompson Children's Hospital

🇺🇸

Chattanooga, Tennessee, United States

CCOP - Nevada Cancer Research Foundation

🇺🇸

Las Vegas, Nevada, United States

Fletcher Allen Health Care - University Health Center Campus

🇺🇸

Burlington, Vermont, United States

San Jorge Children's Hospital

🇵🇷

Santurce, Puerto Rico

Greenville Hospital Cancer Center

🇺🇸

Greenville, South Carolina, United States

Memorial Sloan-Kettering Cancer Center

🇺🇸

New York, New York, United States

Hopital Sainte Justine

🇨🇦

Montreal, Quebec, Canada

Swiss Pediatric Oncology Group Bern

🇨🇭

Bern, Switzerland

McMaster Children's Hospital at Hamilton Health Sciences

🇨🇦

Hamilton, Ontario, Canada

Cancer Centre of Southeastern Ontario at Kingston General Hospital

🇨🇦

Kingston, Ontario, Canada

Cardinal Glennon Children's Hospital

🇺🇸

Saint Louis, Missouri, United States

Children's Hospital Center for Cancer and Blood Disorders

🇺🇸

Aurora, Colorado, United States

St. Joseph's Cancer Institute at St. Joseph's Hospital

🇺🇸

Tampa, Florida, United States

Children's Hospital

🇺🇸

Omaha, Nebraska, United States

UNMC Eppley Cancer Center at the University of Nebraska Medical Center

🇺🇸

Omaha, Nebraska, United States

Midwest Children's Cancer Center at Children's Hospital of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Lee Cancer Care of Lee Memorial Health System

🇺🇸

Fort Myers, Florida, United States

Rhode Island Hospital Comprehensive Cancer Center

🇺🇸

Providence, Rhode Island, United States

Cook Children's Medical Center - Fort Worth

🇺🇸

Fort Worth, Texas, United States

Carilion Medical Center for Children at Roanoke Community Hospital

🇺🇸

Roanoke, Virginia, United States

Providence Cancer Center at Sacred Heart Medical Center

🇺🇸

Spokane, Washington, United States

Children's Hospital and Regional Medical Center - Seattle

🇺🇸

Seattle, Washington, United States

Mayo Clinic Cancer Center

🇺🇸

Rochester, Minnesota, United States

Sutter Cancer Center

🇺🇸

Sacramento, California, United States

University of California Davis Cancer Center

🇺🇸

Sacramento, California, United States

C.S. Mott Children's Hospital at University of Michigan Medical Center

🇺🇸

Ann Arbor, Michigan, United States

Wake Forest University Comprehensive Cancer Center

🇺🇸

Winston-Salem, North Carolina, United States

Madigan Army Medical Center - Tacoma

🇺🇸

Tacoma, Washington, United States

Children's Hospital of Eastern Ontario

🇨🇦

Ottawa, Ontario, Canada

Inova Fairfax Hospital

🇺🇸

Falls Church, Virginia, United States

Hackensack University Medical Center Cancer Center

🇺🇸

Hackensack, New Jersey, United States

Yale Cancer Center

🇺🇸

New Haven, Connecticut, United States

Florida Hospital Cancer Institute at Florida Hospital Orlando

🇺🇸

Orlando, Florida, United States

M.D. Anderson Cancer Center at Orlando

🇺🇸

Orlando, Florida, United States

Nemours Children's Clinic - Orlando

🇺🇸

Orlando, Florida, United States

Kosair Children's Hospital

🇺🇸

Louisville, Kentucky, United States

Children's Mercy Hospital

🇺🇸

Kansas City, Missouri, United States

Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Hollings Cancer Center at Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Cancer Research Center of Hawaii

🇺🇸

Honolulu, Hawaii, United States

University of Florida Shands Cancer Center

🇺🇸

Gainesville, Florida, United States

Lucille P. Markey Cancer Center at University of Kentucky

🇺🇸

Lexington, Kentucky, United States

Children's Hospital of New Orleans

🇺🇸

New Orleans, Louisiana, United States

Blank Children's Hospital

🇺🇸

Des Moines, Iowa, United States

Dell Children's Medical Center of Central Texas

🇺🇸

Austin, Texas, United States

Albert Einstein Cancer Center at Albert Einstein College of Medicine

🇺🇸

Bronx, New York, United States

Virginia Commonwealth University Massey Cancer Center

🇺🇸

Richmond, Virginia, United States

University of Wisconsin Paul P. Carbone Comprehensive Cancer Center

🇺🇸

Madison, Wisconsin, United States

Children's & Women's Hospital of British Columbia

🇨🇦

Vancouver, British Columbia, Canada

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

Overlook Hospital

🇺🇸

Summit, New Jersey, United States

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