A Phase III Randomized Trial for Patients with de novo AML using Bortezomib and Sorafenib for patients with High Allelic Ratio FLT3/ITD


Cancer | Myeloid and Monocytic Leukemia

Trial Phase

Phase II-III

Trial Purpose and Description

Trial Purpose

To compare event free survival (EFS) and overall survival (OS) in patients with de novo acute myeloid leukemia (AML) without high allelic ratio FLT3/ITD+ mutations who are randomized to standard therapy versus bortezomib/standard combination therapy.

Participation Guidelines

0 Years - 30 Years

Eligibility Criteria

Inclusion Criteria:

  • Patients must be less than 30 years of age at the time of study enrollment.
  • Patients must be newly diagnosed with de novo actute myelogenous leukemia.
  • Patients with previously untreated primary AML who meet the customary criteria for AML with > 20% bone marrow blasts as set out in the 2008 WHO Myeloid Neoplasm Classification.
  • Patients with cytopenias and bone marrow blasts who do not meet the customary criteria for the diagnosis of AML (patients with < 20% blasts) are eligible if they have a karyotypic abnormality characteristic of de novo AML (t(821)(q22q22), inv(16)(p13q22) or t(1616)(p13q22) or 11q23 abnormalities), or if they have the unequivocal presence of megakaryoblasts, as set out in the 2008 WHO Myeloid Neoplasm Classification.
  • Patients with biopsy proved isolated myeloid sarcoma (myeloblastoma chloroma, including leukemia cutis) are eligible regardless as set out in the 2008 WHO Myeloid Neoplasm Classification.
  • Patients with any performance status are eligible for enrollment.
  • Prior therapy with hydroxyurea, all-trans retinoic acid (ATRA), corticosteroids (any route), and IT cytarabine given at diagnosis is allowed. Hydroxyurea and ATRA must be discontinued prior to initiation of protocol therapy. Patients who have previously received any other chemotherapy, radiation therapy or any other antileukemic therapy are not eligible for this protocol

Exclusion Criteria:

  • Patients with constitutional trisomy 21 are not eligible.
  • Patients with any of the following are not eligible: Fanconi anemia, Shwachman syndrome, or any other known bone marrow failure syndrome or another concurrent malignancy.
  • Patients with juvenile myelomonocytic leukemia (JMML) are not eligible.
  • Patients with Philadelphia chromosome positive AML, biphenotypic or bilineal acute leukemia, or acute promyelocytic leukemia are not eligible.
  • Pregnancy and Breast Feeding.
  • Female patients who are pregnant are ineligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs.
  • Lactating females are not eligible unless they have agreed not to breastfeed their infants.
  • Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained.
Children's Oncology Group (The)
Last Updated:
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