Vismodegib in Treating Patients With Basal Cell Carcinoma
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|