ADVL1121: A Phase II trial of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib(IND# 69896) in Children and Young Adults with Relapsed/Refractory Rhabdomyosarcomas, Wilms Tumors, Hepatocellular Carcinoma, and Papillary Thyroid Carcinoma

Why are we doing this research?

This Phase II trial will study how well sorafenib tosylate works in treating younger patients with relapsed or refractory Rhabdomyosarcoma, Wilms Tumors, Liver cancer or Thyroid cancer 

Who can participate?

  • 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/1.73m2 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


  • From 3 to 30 years old


  • Female
  • Male


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