Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer
Condition: Childhood Hepatocellular Carcinoma; Papillary Thyroid Cancer; Previously Treated Childhood Rhabdomyosarcoma; Recurrent Childhood Liver Cancer; Recurrent Childhood Rhabdomyosarcoma; Recurrent Thyroid Cancer; Recurrent Wilms Tumor and Other Childhood Kidney Tumors
MeSH terms: Carcinoma; Carcinoma, Hepatocellular; Liver Neoplasms; Thyroid Neoplasms; Rhabdomyosarcoma; Wilms Tumor; Rhabdomyosarcoma, Embryonal; Thyroid Cancer, Papillary; Thyroid Diseases; Recurrence
Study type: Interventional
Phase: Phase 2

Summary: 
      This phase II trial studies how well sorafenib tosylate works in treating younger patients
      with relapsed or refractory rhabdomyosarcoma, Wilms tumor, liver cancer, or thyroid cancer.
      Sorafenib tosylate may stop the growth of cancer cells by blocking some of the enzymes needed
      for cell growth.
    

Detailed description: 
      PRIMARY OBJECTIVES:

      I. To determine the objective response rate to sorafenib tosylate (sorafenib) in children
      with relapsed or refractory rhabdomyosarcoma, Wilms tumor, hepatocellular carcinoma (HCC), or
      papillary thyroid carcinoma (PTC).

      SECONDARY OBJECTIVES:

      I. To further define and describe the toxicities of sorafenib administered on an oral,
      twice-daily continuous schedule.

      II. To further characterize the pharmacokinetics of sorafenib in children with refractory
      cancer.

      III. To estimate the progression-free survival on sorafenib for rhabdomyosarcoma, Wilms
      tumor, and hepatocellular carcinoma and compare to a group of patients enrolled on selected
      closed Phase II studies of Children Oncology Group (COG).

      IV. To assess the biologic activity of sorafenib on vascular endothelial growth factor (VEGF)
      and soluble vascular endothelial growth factor receptor-2 (VEGFR-2) in peripheral blood
      samples. (Exploratory) V. To evaluate the presence of BRAF mutations and RET/PTC
      rearrangements in patients with PTC. (Exploratory)

      OUTLINE: This is a multicenter study. Patients are stratified according to diagnosis
      (rhabdomyosarcoma vs Wilms tumor vs hepatocellular carcinoma vs papillary thyroid carcinoma).

      Patients receive sorafenib tosylate orally (PO) twice daily (BID) on days 1-28. Treatment
      repeats every 28 days for up to 24 courses in the absence of disease progression or
      unacceptable toxicity.

      Patients undergo blood sample collection at baseline and periodically during study for
      pharmacokinetic studies, and VEGF and VEGFR-2 analysis by ELISA. Previously collected
      formalin-fixed paraffin-embedded tissue samples, from patients with papillary thyroid
      carcinoma, are also analyzed for BRAF mutation and RET/PTC rearrangements by PCR.

      After completion of study treatment, patients are followed up for up to 5 years.
    

Intervention name(s): sorafenib tosylate; pharmacological study; laboratory biomarker analysis
Intervention type(s): Drug; Other; Other
Intervention description(s): Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28; Optional correlative studies; Optional correlative studies
Other intervention name(s): BAY 43-9006; BAY 43-9006 Tosylate Salt; BAY 54-9085; Nexavar; SFN; pharmacological studies
Intervention MeSH terms: Sorafenib
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment