A Phase II Study of the Gamma Secretase Inhibitor RO4929097 in Previously Treated Metastatic Pancreas Cancer
I. To determine the 6-month survival of patients with previously treated metastatic pancreas
cancer treated with gamma secretase RO4929097.
II. To determine the adverse events of RO4929097 in this patient population. III. To
correlate changes in tumor markers with RO4929097 exposure.
I. To evaluate the response rate and overall survival of this population treated with
II. To correlate clinical outcome with tumor markers (including stem cell markers) obtained
from pre- and post- treatment biopsies. (exploratory) III. To assess variants in genes
involved in RO4929097 disposition and their relation to RO4929097 exposure.
OUTLINE: This is a multicenter study.
Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 orally (PO)
once daily on days 1-3, 8-10, and 15-17. Courses repeat every 21 days for up to 24 months in
the absence of disease progression or unacceptable toxicity.
Patients may undergo tumor biopsy at baseline and on days 16 or 17 of course one for
biomarker and other correlative studies. Blood samples may also collected at baseline and
periodically during study for pharmacokinetic and angiogenesis marker studies.
After completion of study therapy, patients are followed up periodically for 2 years.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. If patients are lost to follow-up prior to the time required for the applicable primary endpoint, the final point estimate of the primary endpoint will be reported via the method of Kaplan and Meier (1958) which accounts for the censoring of such data at the date of last contact (last known alive). Otherwise, ninety percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner (Duffy and Santer, 1987).
University of Colorado Cancer Center - Anschutz Cancer Pavilion
United States: Food and Drug Administration
|Johns Hopkins University||Baltimore, Maryland 21205|
|University of Colorado||Denver, Colorado 80217|
|University of Colorado Cancer Center - Anschutz Cancer Pavilion||Aurora, Colorado 80045|