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
Conditions
- Cancer - Kidney Tumors
- Cancer - Leukemia and Lymphoma
- Cancer - Liver Tumors
- Medications
Who should I contact for more information?
Cincinnati Children’s Hospital Medical Center
Division of Hematology/Oncology
3333 Burnet Ave., Cincinnati, OH 45229-3039
Phone: 513-636-2799
cancer@cchmc.org
Where can I find additional information?
