Phase 2
N=104
A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
Basal Cell Carcinoma
Bottom Line
View on ClinicalTrials.gov: NCT00833417 ↗Enrolled (actual)
104
Serious AEs
34.6%
Results posted
Apr 2012
Primary outcome: Primary: Objective Response (OR) Determined by the Independent Review Facility — 30.3; 42.9 Percentage of participants — p=0.0011
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Vismodegib 150 mg (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Genentech, Inc.
- Primary completion
- Nov 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Objective Response (OR) Determined by the Independent Review Facility |
30.3; 42.9 | 0.0011 sig |
| SECONDARY Duration of Objective Response (OR) Determined by the Independent Review Facility |
7.6; 7.6 | — |
| SECONDARY Progression-Free Survival (PFS) Determined by the Independent Review Facility |
9.5; 9.5 | — |
| SECONDARY Overall Survival |
33.4; NA | — |
| SECONDARY Change From Baseline in Short Form 36 (SF-36) Health Survey Scores |
2.20; 2.29; -3.80; -1.25; -1.90; -2.86 | — |
| SECONDARY Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort |
54.0; 9.5; 42.9; 1.6 | — |
Summary
This is 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 receive 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.
Data sourced from ClinicalTrials.gov (NCT00833417). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.