A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
Condition: Basal Cell Carcinoma
MeSH terms: Carcinoma; Carcinoma, Basal Cell
Intervention: Vismodegib 150 mg
Intervention description: Vismodegib 150 mg was provided in hard gelatin capsules.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 104.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: BCC; Hedgehog Pathway Inhibitor; Hedgehog; Basal Cell Cancer

Summary:

      This was a Phase II, single-arm, two-cohort multicenter clinical trial evaluating the
      efficacy and safety of vismodegib (GDC-0449) in patients with advanced basal cell carcinoma.
      All patients received vismodegib until evidence of progression, intolerable toxicities most
      probably attributable to vismodegib, or withdrawal from the study.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Men and women ≥ 18 years of age.

          -  For patients with metastatic basal cell carcinoma (BCC), histological confirmation of
             distant BCC metastasis (eg, lung, liver, lymph nodes, or bone), with metastatic
             disease that is Response Evaluation Criteria in Solid Tumors (RECIST) measurable using
             computed tomography (CT) or magnetic resonance imaging (MRI).

          -  For patients with locally advanced BCC, histologically confirmed disease that is
             considered to be inoperable.

          -  For patients with locally advanced BCC, radiotherapy must have been previously
             administered for their locally advanced BCC, unless radiotherapy is contraindicated or
             inappropriate. For patients whose locally advanced BCC has been irradiated, disease
             must have progressed after radiation.

          -  For women of childbearing potential, agreement to the use of two acceptable methods of
             contraception, including one barrier method, during the study and for 12 months after
             discontinuation of vismodegib (GDC-0449).

          -  For men with female partners of childbearing potential, agreement to use a latex
             condom, and to advise their female partner to use an additional method of
             contraception during the study and for 3 months after discontinuation of vismodegib.

        Exclusion Criteria:

          -  Prior treatment with vismodegib or other Hedgehog pathway inhibitors.

          -  Pregnancy or lactation.

          -  Life expectancy of < 12 weeks.

          -  Patients with superficial multifocal BCC who may be considered unresectable due to
             breadth of involvement.

          -  Concurrent non-protocol-specified anti-tumor therapy (eg, chemotherapy, other targeted
             therapy, radiation therapy, or photodynamic therapy).

          -  Recent, current, or planned participation in an experimental drug study.

          -  History of other malignancies within 3 years of the first day of treatment with
             vismodegib in this study (Day 1), except for tumors with a negligible risk for
             metastasis or death, such as adequately treated squamous-cell carcinoma of the skin,
             ductal carcinoma in situ of the breast, or carcinoma in situ of the cervix.

          -  Uncontrolled medical illnesses such as infection requiring treatment with intravenous
             antibiotics.