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A Phase II, Multicenter, Single-Arm, Two-Cohort Trial Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Patients With Advanced Basal Cell Carcinoma

Phase 2
18 Years
Open (Enrolling)
Basal Cell Carcinoma

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Trial Information

A Phase II, Multicenter, Single-Arm, Two-Cohort Trial Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Patients With Advanced Basal Cell Carcinoma

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

Type of Study:


Study Design:

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

Outcome Measure:

Objective Response (OR) Determined by the Independent Review Facility

Outcome Description:

OR=complete (CR) or partial response (PR). Metastatic-CR:Disappearance of all targets. PR:≥30% decreased sum of longest diameter (SLD) of targets compared to baseline (B). Locally advanced-Response=No progressive disease (PD) and ≥30% decreased SLD from baseline (radiography [R]) or ≥30% decreased SLD from B (externally visible dimension [EVD]) or completely resolved ulceration. CR:Response with no residual BCC on tumor biopsy (otherwise response was PR). PD:Any of ≥20% increased SLD from nadir (R or EVD), new ulceration, new lesions (R or physical exam) or non-target lesion progression by R.

Outcome Time Frame:

From study initiation (enrollment of first patient) through 9 months following the first treatment of the last enrolled patient (clinical cutoff date of 26 November 2010), up to 90 weeks

Safety Issue:


Principal Investigator

Jeannie Hou, M.D.

Investigator Role:

Study Director

Investigator Affiliation:



United States: Food and Drug Administration

Study ID:




Start Date:

February 2009

Completion Date:

February 2014

Related Keywords:

  • Basal Cell Carcinoma
  • BCC
  • Hedgehog Pathway Inhibitor
  • Hedgehog
  • Basal Cell Cancer
  • Carcinoma
  • Carcinoma, Basal Cell



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