A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCC)
I. The percent reduction in surgical defect area/size surrounding BCC tumor pre and
I. Bread-loafing histology will be used to determine if the tumor after vismodegib shrinks
as one discrete lesion (contiguous) vs. with skip areas (non-contiguous) II. The percent
reduction in tumor size pre and post-vismodegib. III. Safety, tolerability and percent
drop-out after 3 vs. 6 months of vismodegib in otherwise healthy patients.
Patients receive vismodegib orally (PO) once daily (QD) for up to 3 months if the initial
BCC size is < 2 cm and superficial or for up to 6 months if the initial BCC size is >= 2 cm
or non-superficial. After completion of vismodegib treatment, patients undergo Mohs surgery.
After completion of study treatment, patients are followed up for 6 months.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Anti-tumor effect as measured by the percent change in surgical defect area against the null hypothesis of no change after the treatment period using calipers and photographs
This will be tested against the null hypothesis of no change as a one-sample t-test. A p-value of less than 0.05 will be considered statistically significant. We will first perform non-parametric paired t-test taking this account, and also use more sophisticated methods such as the MIXED procedure (PROC MIXED) treating each subject as a random effect and time as fixed effect, with appropriate correlation structure.
Baseline to 6 months
United States: Institutional Review Board
|Stanford University||Stanford, California 94305|