VISmodegib for ORbital and Periocular Basal Cell Carcinoma
Condition: Carcinoma, Basal Cell
MeSH terms: Carcinoma; Carcinoma, Basal Cell
Intervention: Vismodegib
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 35.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      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
      eventually required

      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.

      Patients with tumors that do not respond to treatment with Vismodegib, and those who have a
      good response but poor tolerance of Vismodegib, will be offered surgical excision of the
      tumor. Patients with a good response and good tolerance of Vismodegib may continue the
      treatment as long as clinically indicated.
    

Eligibility criteria:

        Inclusion Criteria:

          -  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 3 months after last
             dose.

          -  Male patients must agree to not donate sperm during treatment and for 3 months after
             last dose.

          -  Participant must agree not to donate blood during the study and for 7 months after
             last dose.

          -  Informed consent signed.

          -  If the patient consents to enroll, then blood will be drawn and stored for biomarker
             analysis.

        Exclusion Criteria:

          -  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.

          -  Dementia or significantly altered mental status that would prohibit the understanding
             or rendering of informed consent and compliance with the requirements of this
             protocol.

          -  Age under 18 years.