Vismodegib in Treating Patients With Basal Cell Carcinoma
The purpose of this study is to learn about the effect of vismodegib on sporadic basal cell carcinoma (BCCs) prior to surgical removal.
Basal Cell Carcinoma of the Skin
Recurrent Skin Cancer
Procedure: Mohs surgery
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCC)|
- 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 [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]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.
- Proportion of tumors with skip lesions present determined using bread-loafing of tumor block and histology [ Time Frame: Assessed up to 6 months ] [ Designated as safety issue: No ]
- Average area of the subclinical tumor invasion calculated from the pre-surgical skin markings and by comparing the initial clinical margins to the final Mohs defect [ Time Frame: Assessed up to 6 months ] [ Designated as safety issue: No ]
|Study Start Date:||June 2012|
|Estimated Study Completion Date:||November 2015|
|Estimated Primary Completion Date:||November 2015 (Final data collection date for primary outcome measure)|
Experimental: Treatment (vismodegib and Mohs surgery)
Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.
Other Names:Procedure: Mohs surgery
Undergo Mohs surgery
I. The percent reduction in surgical defect area/size surrounding BCC tumor pre and post-vismodegib.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01631331
|United States, California|
|Stanford, California, United States, 94305|
|Principal Investigator:||Jean Tang||Stanford University|