Carcinoma, Basal Cell
Conditions
Brief summary
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.
Interventions
DRUGVismodegib
Sponsors
University of Michigan Rogel Cancer Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS). | Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days. | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With Progressive Disease (PD) | Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days. | 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). |
| Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib | Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days. | Tolerance will be self-reported by patient. 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). |
| Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib | Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days. | Tolerance will be self-reported by patient. 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). |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Vismodegib 150mg taken orally once daily | 35 |
| Total | 35 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
Baseline characteristics
| Characteristic | Vismodegib |
|---|---|
| Age, Continuous | 69 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 34 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Region of Enrollment United States | 35 participants |
| Sex: Female, Male Female | 17 Participants |
| Sex: Female, Male Male | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 2 / 35 |
| other Total, other adverse events | 34 / 35 |
| serious Total, serious adverse events | 9 / 35 |
Outcome results
Primary
The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
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.
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Vismodegib | The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS). | 34 Participants |
Secondary
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
Tolerance will be self-reported by patient. 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).
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Vismodegib | Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib | 7 Participants |
Secondary
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
Tolerance will be self-reported by patient. 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).
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Vismodegib | Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib | 27 Participants |
Secondary
Number of Patients With Progressive Disease (PD)
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).
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Vismodegib | Number of Patients With Progressive Disease (PD) | 0 Participants |