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Investigator(s):
Brigitte Widemann, M.D. Principal Investigator Phone: 301-496-7387 widemanb@pbmac.nci.nih.gov
Referral Contact(s):
Pediatric Oncology  Phone: 1-877-624-4878 (Toll free)

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Primary Eligibility:

• Diagnosis of one of the following:
• Histologically confirmed malignant solid tumor at either original diagnosis or relapse:
• Measurable or evaluable disease
• Fully recovered from toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy
• Adequate bone marrow function, as defined by the following:
• Absolute neutrophil count ≥ 1,000/mm³
• Platelet count ≥ 75,000/mm³ (transfusion independent)
• Hemoglobin ≥ 8.0 g/dL (red blood cell [RBC] transfusions allowed)
• Histologically confirmed: Acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), juvenile myelomonocytic leukemia (JMML), or chronic myelogenous leukemia (CML) in blast crisis:
• Patients with JMML have to meet the diagnostic criteria for JMML
• > 25% blasts in the bone marrow (this requirement does not apply to patients with JMML)
• Abnormal blood counts allowed but must meet the following criteria:
• Platelet count ≥ 20,000/mm³ (platelet transfusions allowed)
• Hemoglobin ≥ 8.0 g/L (RBC transfusions allowed)
• Recovered from the non-hematologic toxic effects of all prior therapy
• 2–21 years of age
• Relapsed or refractory disease
• Karnofsky ≥ 50% for patients > 10 years of age or Lansky ≥ 50% for patients ≤ 10 years of age
• Bone marrow or stem cell transplants must have been performed at least 3 months before study entry
• Adequate renal function, liver function, and pulmonary function
• Able to swallow tablets
• Patients with brain tumors or known metastasis to the brain are excluded
• Patients on therapeutic anticoagulation are excluded (prophylactic anticoagulation is allowed provided that the requirements for PT, INR, or PTT are met)
• Patients must NOT have baseline hypertension greater than grade 1
• Males or females of reproductive age must agree to use effective contraceptive methods
• No other medical condition or situation that would preclude study compliance

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Treatment Plan:

This is a dose-escalation, multicenter study. Patients are stratified according to diagnosis (malignant solid tumor vs leukemia).

• Sorafenib will be given orally twice daily on a continuous dosing schedule (28 days = 1 treatment cycle)
• Up to 24 cycles in the absence of disease progression or unacceptable toxicity
• Dose escalations will be performed to define the MTD
• Initially, the spectrum of toxicity and MTD will be defined in patients with solid tumors
• Once the dose-finding component of the trial is completed and the MTD is established in patients with solid tumors, six patients with leukemia will be enrolled at the MTD

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Additional Information:

• This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
• There is no charge for medical care received at NIH Clinical Center.
• PDQ (Physicians Data Query) - provides additional details about this study for health care providers.

Reviewed: 11/17/08
Updated: 2/11/08