PBTC-032: A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children with Recurrent or Refractory Medulloblastoma.

Why are we doing this research?

This phase II trial is studying how well GDC-0449 works in treating younger patients with recurrent or refractory medulloblastoma. GDC-0449 may slow the growth of tumor cells

Who can participate?

Patients between the ages of 3 and 21 with a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive, or refractory to standard therapy and for which there is no known curative therapy.


  • From 3 to 21 years old


  • Cancer - Brain and Spinal Tumors


  • Female
  • Male

What will happen in the study?


  • This is a multicenter study. Patients are stratified according to evidence of activation of Hedgehog signaling pathway in their tumors (without vs. with vs. unknown).
  • Patients receive oral Hedgehog antagonist GDC-0449 once daily on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.
  • Plasma and cerebrospinal fluid samples are collected periodically for pharmacokinetic and other correlative studies.
  • After completion of study treatment, patients are followed up for up to 12 months.


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