A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Adults With Recurrent or Refractory Medulloblastoma
PRIMARY OBJECTIVES:
I. To estimate the efficacy of GDC-0449 treatment for adult patients with recurrent or
refractory medulloblastoma, as measured by the objective response rates for patients without
(Stratum A) and with (Stratum B) evidence of activation of Sonic Hedgehog (SHH) signaling
pathway tumors.
SECONDARY OBJECTIVES:
I. To assess the safety and tolerability of GDC-0449 administered on a once daily schedule.
II. To estimate the duration of objective response and progression-free survival (PFS).
III. To characterize the pharmacokinetics (plasma and cerebrospinal fluid) of GDC-0449 in
adults with refractory medulloblastoma.
IV. To document pathologic and genomic methods to identify CNS tumors with activation of the
PTCH/SHH pathway.
V. To describe the objective responses observed in patients whose pathologic assessment of
tumor result in unknown (Stratum C) evidence of activation of Sonic Hedgehog (SHH) signaling
pathway tumors.
OUTLINE: This is a multicenter study. Patients are stratified according to PTCH/Sonic
Hedgehog signaling pathway activation (inactivated vs activated 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.
After completion of study treatment, patients are followed up periodically for up to 12
months.
Interventional
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Objective response rates (PR and CR) graded using RECIST criteria
Ninety-five percent confidence interval estimates of the true, unknown objective response rate will be constructed for each of the three strata.
Up to 12 months
No
Amar Gajjar
Principal Investigator
Pediatric Brain Tumor Consortium
United States: Food and Drug Administration
NCI-2012-03020
NCT00939484
June 2009
Name | Location |
---|---|
Pediatric Brain Tumor Consortium | Memphis, Tennessee 38105 |