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
Intervention: sorafenib tosylate; pharmacological study; laboratory biomarker analysis
Intervention description: 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
Intervention MeSH terms: Sorafenib
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 2 Years
Maximum age: 30 Years
Healthy volunteers: No

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients must have had histologic verification of one of the malignancies listed below
             at original diagnosis or at relapse:

               -  Rhabdomyosarcoma (RMS)

               -  Wilms tumor

               -  Hepatocellular carcinoma (HCC)

               -  Papillary thyroid carcinoma (PTC)

          -  Patients must have relapsed or refractory disease (RMS, Wilms tumor, HCC, PTC)

               -  Patients must have radiographically measurable disease; measurable disease is
                  defined as the presence of at least one lesion on magnetic resonance imaging
                  (MRI) or computed tomography (CT) scan that can be accurately measured with the
                  longest diameter a minimum of 10 mm in at least one dimension (CT scan slice
                  thickness no greater than 5 mm)

                    -  The following do not qualify as measurable disease:

                         -  Malignant fluid collections (e.g., ascites, pleural effusions)

                         -  Bone marrow infiltration

                         -  Lesions only detected by nuclear medicine studies (e.g., bone, gallium,
                            or positron emission tomography [PET] scans)

                         -  Elevated tumor markers in plasma or cerebrospinal fluid(CSF)

                         -  Previously radiated lesions that have not demonstrated clear
                            progression post radiation

                         -  Leptomeningeal lesions that do not meet the requirements noted above

          -  Patients with HCC must be relapsed or refractory to conventional chemotherapy

          -  Patients with PTC must be refractory to radioactive iodine (RAI)

          -  Patient's current disease state must be one for which there is no known curative
             therapy or therapy proven to prolong survival with an acceptable quality of life

          -  Patients with known metastasis to the brain will be excluded from trial participation
             unless treated surgically or with radiotherapy and stable with no recurrent lesions
             for at least 3 months

          -  Rhabdomyosarcoma and Wilms strata: patients must be ≥ 24 months and ≤ 30 years of age
             at study enrollment

          -  Hepatocellular carcinoma (HCC): patients must be ≥ 24 months and < 18 years of age at
             study enrollment

          -  Papillary thyroid carcinoma (PTC): patients must be ≥ 24 months and ≤ 21 years of age
             at study enrollment

          -  Patients must have a Lansky or Karnofsky performance status score of ≥ 50%,
             corresponding to ECOG categories 0, 1, or 2

               -  Use Karnofsky for patients > 16 years of age and Lansky for patients ≤ 16 years
                  of age

               -  Patients who are unable to walk because of paralysis, but who are up in a
                  wheelchair, will be considered ambulatory for the purpose of assessing the
                  performance score

          -  Peripheral absolute neutrophil count (ANC) ≥ 1,000/μL

          -  Platelet count ≥ 75,000/μL (transfusion independent, defined as not receiving platelet
             transfusions within a 7-day period prior to enrollment)

          -  Hemoglobin 8.0 g/dL (may receive red blood cell[RBC] transfusions)

          -  Creatinine clearance or radioisotope glomerular filtration rate(GFR) 70 mL/min OR a
             serum creatinine based on age/gender as follows:

               -  0.8 mg/dL (2 to < 6 years of age)

               -  1.0 mg/dL (6 to < 10 years of age)

               -  1.2 mg/dL (10 to < 13 years of age)

               -  1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)

               -  1.7 mg/dL (male) or 1.4 mg/dL (female) (≥ 16 years of age)

          -  Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age

          -  SGPT (ALT) ≤ 135 U/L (for the purpose of this study, the ULN for SGPT is 45 U/L)

          -  PT, PTT, and INR < 1.5 times ULN

          -  Normal serum lipase and amylase (per institutional normal values)

          -  No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry > 94% if
             there is clinical indication for determination

          -  A blood pressure (BP) ≤ the 95^th percentile for age, height, and gender; and not
             receiving medication for treatment of hypertension

          -  Patients who are pregnant or breast-feeding are not eligible

          -  Negative pregnancy tests must be obtained in girls who are post-menarchal

          -  Males or females of reproductive potential may not participate unless they have agreed
             to use an effective contraceptive method beginning at the signing of the informed
             consent until at least 30 days after the last dose of the study drug

          -  Patients with clinical symptoms of hepatic encephalopathy or ascites are not eligible

          -  Patients who have an uncontrolled infection are not eligible

          -  Patients with evidence of bleeding diathesis are not eligible

          -  Patients with known Gilbert syndrome are not eligible

          -  Patients who, in the opinion of the investigator, may not be able to comply with the
             safety-monitoring requirements of the study are not eligible

          -  No concurrent chemotherapy, radiation therapy, immunomodulating agents, or other
             investigational agents

          -  Patients must have fully recovered from the acute toxic effects of all prior
             chemotherapy, immunotherapy, or radiotherapy prior to entering this study

          -  Patients with solid tumors must not have received myelosuppressive chemotherapy within
             3 weeks of enrollment onto this study (6 weeks if prior nitrosourea)

          -  At least 7 days must have elapsed since the completion of therapy with a growth factor
             (at least 14 days must have elapsed after receiving pegfilgrastim)

          -  At least 7 days must have elapsed since completion of therapy with a biologic agent;

               -  For agents that have known adverse events occurring beyond 7 days after
                  administration, this period prior to enrollment must be extended beyond the time
                  during which adverse events are known to occur

          -  At least 3 half-lives must have elapsed since prior therapy that included a monoclonal
             antibody

          -  At least 2 weeks must have elapsed since local palliative radiotherapy (XRT) (small
             port); ≥ 3 months must have elapsed if prior craniospinal XRT was received, if ≥ 50%
             of the pelvis was irradiated, or if TBI was received; ≥ 6 weeks must have elapsed if
             other substantial bone marrow irradiation was given

          -  No evidence of active graft-vs-host disease and ≥ 2 months must have elapsed since
             transplant (stem cell transplant or rescue without total-body irradiation)

          -  For patients with papillary thyroid carcinoma (PTC) only: ≥ 3 weeks from prior
             radioiodine (RAI) treatment

          -  Patients requiring corticosteroids that have not been on a stable or decreasing dose
             of corticosteroid for 7 days prior to enrollment are not eligible

          -  Patients who are currently receiving another investigational drug are not eligible

          -  Patients who are currently receiving other anti-cancer agents are not eligible

          -  Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either
             graft-versus-host disease post bone marrow transplant or organ rejection post
             transplant are not eligible for this trial

          -  Patients who take cytochrome P450 enzyme-inducing anti-epileptic drugs (phenytoin,
             carbamazepine, or phenobarbital), rifampin, grapefruit juice, or St. Johns wort will
             not be eligible for the trial

          -  Patients who have received prior treatment with sorafenib are not eligible

          -  Patients must not be on therapeutic anti-coagulation;

               -  Prophylactic anticoagulation (i.e., low-dose warfarin) of venous or arterial
                  devices is allowed provided that the requirements for prothrombin time(PT),
                  partial thromboplastin time(PTT), and international normalized ratio(INR) are met