ADVL0413: A Phase I/II Study of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib (BAY 43-9006, NSC# 724772, IND# 69896) in Children With Refractory Solid Tumors or Refractory Leukemias

What is the purpose of this study?

This phase I trial is studying the side effects and best dose of sorafenib in treating young patients with relapsed or refractory solid tumors or leukemia.

Who can participate?

  • Diagnosis of 1 of the following:
  • Histologically confirmed malignant solid tumor at original diagnosis or relapse
  • Measurable or evaluable disease by CT scan or MRI
  • Histologically confirmed leukemia, including 1 of the following:
  • Acute lymphoblastic leukemia (ALL)
  • Greater than 25% blasts in the bone marrow (M3 bone marrow)
  • Acute myeloid leukemia (AML)
  • Greater than 25% blasts in the bone marrow (M3 bone marrow)

AML and FLT3-ITD mutation

  • Patients must have ≥5% blasts in the bone marrow
  • Active extramedullary disease (except leptomeningeal disease) allowed
  • Juvenile myelomonocytic leukemia (JMML) meeting the following criteria:
  • Peripheral blood monocytosis > 1,000/mm^3
  • Blasts (including promonocytes) are < 20% of the WBCs in the blood and of the nucleated bone marrow cells
  • No Philadelphia chromosome (Ph) or BCR/ABL fusion gene
  • Has ≥2 of the following additional diagnostic criteria:
  • Hemoglobin F increased for age
  • Immature granulocytes in the peripheral blood
  • WBC > 10,000/mm^3
  • Clonal chromosomal abnormality (e.g., may be monosomy 7)
  • Sargramostim (GM-CSF) hypersensitivity of myeloid progenitors in vitro
  • Chronic myelogenous leukemia (CML) in blast crisis
  • Greater than 25% blasts in the bone marrow (M3 bone marrow)
  • Patients with Ph-positive CML must be refractory to imatinib mesylate
  • Relapsed or refractory disease
  • Patients with acute promyelocytic leukemia (APL) must be refractory to treatment with tretinoin and arsenic trioxide
  • Standard curative therapies or therapies proven to prolong survival with an acceptable quality of life do not exist
  • Active extramedullary disease, except active leptomeningeal leukemia, allowed
  • No brain tumors or known brain metastases


  • Cancer - Kidney Tumors
  • Cancer - Leukemia and Lymphoma
  • Cancer - Liver Tumors
  • Medications


Cincinnati Children’s Hospital Medical Center
Division of Hematology/Oncology
3333 Burnet Ave., Cincinnati, OH 45229-3039
Phone: 513-636-2799