Basal cell carcinoma (BCCA) is the most common human cancer, and frequently affects facial
structures. While rarely fatal, facial BCCA can be disfiguring and expensive to treat.
Vismodegib is a small molecule inhibitor of SMO developed for the treatment of tumors in
which the Hh signaling pathway appears to contribute to the development and maintenance of
tumorigenesis. Vismodegib was recently approved by the Food and Drug Administration (FDA)
for treatment of metastatic and locally advanced BCCA. Recent reports have suggested that
vismodegib treatment for orbital BCCA may facilitate eye preservation even if surgery is
In order to assess the potential of vismodegib to improve the ophthalmic outcomes following
treatment for orbital and/or periocular BCCA, this study will follow patients with
globe-threatening orbital and lacrimal-threatening periocular BCCA who are being treated
with vismodegib as standard of care.
- Adult patients over 18 years of age with locally advanced or recurrent orbital or
periorbital basal cell carcinoma (BCCA), or a medial canthal BCCA that threatens the
lacrimal drainage system.
- Clinical assessment score obtained at baseline.
- Medical Oncology screening performed at baseline.
- Adequate BCCA size and location.
- Adequate hematopoietic capacity, hepatic and renal function.
- Male patients must agree to use condoms during treatment and for 2 months after last
- Male patients must agree to not donate sperm during treatment and for 2 months after
- Participant must agree not to donate blood during the study and for 7 months after
- Informed consent signed.
- Patient must consent to blood biomarker analysis.
- Patient must be able to be present for monthly visits for the duration of the study.
- Inability or unwillingness to swallow capsules.
- Inability or unwillingness to comply with study procedures.
- Pregnant, lactating, or breast feeding women.
- Women of childbearing potential.
- Uncontrolled medical illness.
- Age under 18 years.