Description:
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.
Title
- Brief Title: VISmodegib for ORbital and Periocular Basal Cell Carcinoma
- Official Title: VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)
Clinical Trial IDs
- ORG STUDY ID:
UMCC 2014.022
- SECONDARY ID:
HUM00082579
- NCT ID:
NCT02436408
Conditions
Interventions
| Drug | Synonyms | Arms |
|---|
| Vismodegib | | Vismodegib |
Purpose
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.
Trial Arms
| Name | Type | Description | Interventions |
|---|
| Vismodegib | Experimental | 150mg taken orally once daily | |
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.
| Maximum Eligible Age: | N/A |
| Minimum Eligible Age: | 18 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
Primary Outcome Measures
| Measure: | The number of patients with a score of 21 or greater by the Visual Assessment Weighted Score (VAWS). |
| Time Frame: | Up to 15 months after start of study treatment |
| Safety Issue: | |
| Description: | The VAWS was developed for the purpose of this study. It is made up of standard ophthalmic exam points, as well as subjective assessment of tearing and overall patient satisfaction. The maximum score is 50 and a score of 21 or greater will be considered a good outcome. Patients will be assessed at 1 year after start of study treatment or 2 (+/- 1) months after tumor excision/debulking surgery, whichever is longer. |
Secondary Outcome Measures
| Measure: | Number of patients with progressive disease (PD) |
| Time Frame: | Up to 15 months after start of study treatment |
| Safety Issue: | |
| Description: | Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician). |
| Measure: | Number of patients with a complete response (CR), partial response (PR) or stable disease (SD) AND good tolerance of vismodegib |
| Time Frame: | Up to 15 months after start of study treatment |
| Safety Issue: | |
| Description: | Treatment response will be determined per RECIST, using clinical measurements and/or tumor imaging measurements (determined by treating physician). Tolerance will be self-reported by patient. |
| Measure: | Number of patients with a complete response (CR), partial response (PR) or stable disease (SD) AND poor tolerance of vismodegib |
| Time Frame: | Up to 15 months after start of study treatment |
| Safety Issue: | |
| Description: | Treatment response will be determined per RECIST, using clinical measurements and/or tumor imaging measurements (determined by treating physician). Tolerance will be self-reported by patient. |
Details
| Phase: | Phase 4 |
| Primary Purpose: | Interventional |
| Overall Status: | Active, not recruiting |
| Lead Sponsor: | University of Michigan Rogel Cancer Center |
Last Updated
April 30, 2020
