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A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCC)

18 Years
Open (Enrolling)
Basal Cell Carcinoma of the Skin, Recurrent Skin Cancer

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Trial Information

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.

Inclusion Criteria:

- Study patients must have at least one BCC, > 5 mm, eligible for Mohs surgical
removal; patients with BCCs that have been treated before (recurrent BCCs, BCCs that
failed other chemotherapy) are eligible for this trial, if they meet size criteria

- No Eastern Cooperative Oncology Group (ECOG) or Karnofsky performance status will be

- Normal hepatic function: aspartate aminotransferase (AST) and alanine
aminotransferase (ALT) =< 2 x the upper limit of normal (ULN)

- Normal renal function : normal serum creatinine defined as <= 2.5 mg/dL

- Clinically acceptable complete blood count (CBC)

- Ability to understand and the willingness to sign a written informed consent document

- The patient is willing to forego surgical treatment of BCCs by up to 6 months, except
when the principal investigator (PI) believes that delay in treatment potentially
might compromise the health of the subject

- Documented negative serum pregnancy test for women of childbearing potential, with
agreement to the use of two acceptable methods of contraception during the study and
for 7 months after discontinuation of vismodegib

- For men with female partners of childbearing potential, agreement to use a latex,
non-latex, or any other male condom and to advise their female partners to use an
additional acceptable method of birth control during the study and for 2 months after
discontinuation of study drug

- Be willing to not donate blood or semen for three months following discontinuation of
study medications

Exclusion Criteria:

- The patient has a history of invasive cancer within the past five years excluding
non-melanoma skin cancer, stage I cervical cancer, ductal carcinoma in situ of the
breast, or chronic lymphocytic leukemia (CLL) stage 0

- The subject has uncontrolled systemic disease, including known human immunodeficiency
virus (HIV) positive patients:

- The patient has history of congestive heart failure

- The patient has clinically important history of liver disease, including viral
or hepatitis, current alcohol abuse, or cirrhosis

- The patient has any condition or situation which in the investigator's opinion
may put the patient at significant risk, could confound the study results, or
could interfere significantly with the subject's participation in the study;
this includes history of other skin conditions or disease, metabolic
dysfunction, physical examination finding, or clinical laboratory finding giving
reasonable suspicion of a disease or condition that contraindicates use of an
investigational drug or that might affect interpretation of the results of the
study or render the patient at high risk from treatment complications

- The patient has a history of hypersensitivity to any of the ingredients in the study
medication formulations

- The patient is willing to abstain from application of non-study topical medications
to the skin for the duration of the study, including prescription and over the
counter preparations; for example, topical preparations containing corticosteroids or
vitamin A derivatives are not allowed

- Pregnant or nursing patients will be excluded from the study

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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

Outcome Description:

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.

Outcome Time Frame:

Baseline to 6 months

Safety Issue:


Principal Investigator

Jean Tang

Investigator Role:

Principal Investigator

Investigator Affiliation:

Stanford University


United States: Institutional Review Board

Study ID:




Start Date:

June 2012

Completion Date:

Related Keywords:

  • Basal Cell Carcinoma of the Skin
  • Recurrent Skin Cancer
  • Carcinoma
  • Skin Neoplasms
  • Carcinoma, Basal Cell
  • Carcinoma, Basosquamous
  • Carcinoma, Squamous Cell



Stanford University Stanford, California  94305