PBTC-025B: A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Adults with Recurrent or Refractory Medulloblastoma

Why are we doing this research?

This phase II trial is studying how well GDC-0449 works in treating adult patients with recurrent or refractory medulloblastoma.

Who can participate?


  • ECOG performance status 0-2
  • Life expectancy ≥ 12 weeks (as determined by treating physician)
  • ANC ≥ 1,000/mm^3
  • Platelet count ≥ 100,000/mm^3 (transfusion-independent)
  • Hemoglobin ≥ 8.0 g/dL (RBC transfusions allowed)
  • Creatine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine ≤ 1.5 mg/dL
  • Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age
  • ALT and AST ≤ 2.5 times ULN
  • Serum albumin ≥ 2.5 g/dL
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use two effective methods of contraception (including one barrier method) during and for 12 months after completion of study treatment
  • Able to return for follow-up visits and complete follow-up studies required to assess toxicity to study treatment
  • Able to swallow capsules
  • No uncontrolled hypocalcemia, hypomagnesemia, hyponatremia, or hypokalemia, defined as less than the lower limit of normal despite adequate electrolyte supplementation
  • No malabsorption syndrome or other condition that would interfere with enteral absorption
  • No history of congestive heart failure
  • No history of ventricular arrhythmia requiring medication
  • No congenital long QT syndrome
  • No clinically significant unrelated systemic illness (e.g., serious infection or significant cardiac, pulmonary, hepatic, or other organ dysfunction) that would compromise the patient's ability to tolerate study treatment or would likely interfere with study procedures or results
  • No blood donation for ≥ 12 months after completion of study treatment


  • Recovered from prior treatment-related toxicity
  • No prior hedgehog antagonist GDC-0449 or other antagonists of the hedgehog pathway
  • More than 4 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas) or immunotherapy
  • At least 3 months since prior craniospinal irradiation (≥ 23 Gy)
  • At least 8 weeks since prior local irradiation to primary tumor
  • At least 2 weeks since prior focal irradiation for symptomatic metastatic sites
  • At least 1 week since prior colony-stimulating factors (e.g., G-CSF, GM-CSF, or erythropoietin)
  • Concurrent decadron allowed provided the dose is stable or decreasing for ≥ 1 week before starting study treatment


  • years old and above


  • Cancer - Brain and Spinal Tumors


  • Female
  • Male


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