PBTC-032: A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children with Recurrent or Refractory Medulloblastoma.
What is the purpose of this study?
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
Who can participate?
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.
- Cancer - Brain and Spinal Tumors
What is involved?
- 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
- Plasma and cerebrospinal
fluid samples are collected periodically for pharmacokinetic and other
- After completion of study
treatment, patients are followed up for up to 12 months.
Who should I contact for more information?
For more information
Hospital Medical Center
Burnet Ave., Cincinnati, OH 45229-3039
Where can I find additional information?