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A Multi-Center Seamless Phase II-III Randomized Trial of High-dose Cytarabine in Initial Induction with Evaluation of Flow-cytometry-based Minimal Residual Disease for Children with de Novo Acute Myeloid Leukemia (AML-12)

Phase 2
Conditions
Acute Myeloid Leukemia
Registration Number
JPRN-UMIN000013288
Lead Sponsor
Japan Children's Cancer Group (JCCG)
Brief Summary

Primary endpoints of this trial were early mortality rate in the Phase II part and 3-year EFS and rate of patients with positive flow-cytometric MRD at end-of-Induction-1 in the Phase III part. The main analyses of the Phase III part were performed for the 324 patients (ECM arm, N=168; HD-ECM arm, N=156) defined as FAS. 3-year EFS: ECM 64.3% (95%CI, 56.5-71.0%), HD-ECM 61.2% (95%CI, 53.1-68.4%), p=0.589 Positive FCM-MRD rate at TP1: ECM 21.5% (95%CI, 14.9-29.4%), HD-ECM 25.2% (95%CI, 17.9-33.7%), p=0.517

Detailed Description

Not available

Recruitment & Eligibility

Status
Complete: follow-up complete
Sex
All
Target Recruitment
387
Inclusion Criteria

Not provided

Exclusion Criteria

1) Patients with severe CNS hemorrhage (grade 3 or higher in CTCAE ver4.0) 2) Patients with uncontrollable infection (including those with active tuberculosis or positive HIV antibody) 3) Patients who are pregnant or breast-feeding mother 4) Patients with history of primary or acquired immunodeficiency 5) Patients with uncontrollable heart failure; presence of heart anomaly itself is not an exclusion criteria 6) Patients with any other inappropriate status judged by physician

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<Phase II study> Early death rate <Phase III study> 1) 3-year event-free survival (EFS) rate 2) Positive rate of flow-cytometry-based minimal residual disease (FCM-MRD) after initial induction course (TP-1)
Secondary Outcome Measures
NameTimeMethod
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