A Phase 2 Study of GDC-0449 in Patients With Advanced Chondrosarcomas
I. Evaluate the antitumor activity of GDC-0449 (vismodegib) in terms of 6-month clinical
benefit rate (complete response, partial response, and stable disease, as per the revised
RECIST criteria 2009).
I. Determine the best overall response (as per the revised RECIST criteria 2009).
II. Determine the 1- and 2-year progression-free survival. III. Determine the 1- and 2-year
overall survival. IV. Assess GDC-0449 safety. V. Conduct a pharmacogenomic analysis of
predictive markers of treatment outcome.
OUTLINE: This is a multicenter study.
Patients receive vismodegib orally (PO) on days 1-28. Courses repeat every 28 days in the
absence of disease progression or unacceptable toxicities.
Tumor samples are analyzed for correlative, including biomarker, studies.
After completion of study therapy, patients are followed up every 3 months.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Clinical benefit (CR + PR + SD) rate
At six months, patients will be classified as success (alive at 6 months AND CR/PR/ SD) or failure (dead OR alive with progression).
At 6 months
Institut Bergonie Cancer Center
United States: Food and Drug Administration