A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00833417
• Date of registration: 30/01/2009
• Prospective Registration: Yes
• Primary sponsor: Genentech, Inc.
• Public title: A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
• Scientific title: A Pivotal Phase II, Multicenter, Single-arm, Two-cohort Trial Evaluating the Efficacy and Safety of GDC-0449 in Patients With Advanced Basal Cell Carcinoma
• Date of first enrolment: February 2009
• Target sample size: 104
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00833417
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, France, Germany, United Kingdom, United States

Contacts

• Name: Jeannie Hou, M.D.
• Affiliation: Genentech, Inc.

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Basal Cell Carcinoma

Intervention(s)

Drug: Vismodegib 150 mg

Primary Outcome(s)

Objective Response (OR) Determined by the Independent Review Facility [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]

Secondary Outcome(s)

Progression-free Survival (PFS) Determined by the Independent Review Facility [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]

Duration of Objective Response (OR) Determined by the Independent Review Facility [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]

Percentage of Patients With Absence of Residual Basal Cell Carcinoma (BCC) in Patients With Locally Advanced BCC [Time Frame: From baseline through end of the study, up to 90 weeks]

Change From Baseline in Short Form 36 (SF-36) Health Survey Scores [Time Frame: Baseline, Week 12, Week 24, and at the end of the study or early termination visit, up to 90 weeks]

Overall Survival [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]

Secondary ID(s)

SHH4476g

GO01541

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/04/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00833417