VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)
Primary Purpose
Carcinoma, Basal Cell
Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Vismodegib
Sponsored by
About this trial
This is an interventional treatment trial for Carcinoma, Basal Cell
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.
Sites / Locations
- University of Michigan Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Vismodegib
Arm Description
150mg taken orally once daily
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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).
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).
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).
Full Information
NCT ID
NCT02436408
First Posted
May 1, 2015
Last Updated
September 29, 2021
Sponsor
University of Michigan Rogel Cancer Center
1. Study Identification
Unique Protocol Identification Number
NCT02436408
Brief Title
VISmodegib for ORbital and Periocular Basal Cell Carcinoma
Acronym
VISORB
Official Title
VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2021
Overall Recruitment Status
Completed
Study Start Date
July 15, 2015 (Actual)
Primary Completion Date
September 2020 (Actual)
Study Completion Date
September 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Michigan Rogel Cancer Center
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Carcinoma, Basal Cell
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
35 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Vismodegib
Arm Type
Experimental
Arm Description
150mg taken orally once daily
Intervention Type
Drug
Intervention Name(s)
Vismodegib
Primary Outcome Measure Information:
Title
The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
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.
Time Frame
Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Secondary Outcome Measure Information:
Title
Number of Patients With Progressive Disease (PD)
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).
Time Frame
Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Title
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
Description
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.
Title
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
Description
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.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Cagri Besirli, M.D., Ph.D.
Organizational Affiliation
University of Michigan
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Michigan Hospital
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
33988881
Citation
Kahana A, Unsworth SP, Andrews CA, Chan MP, Bresler SC, Bichakjian CK, Durham AB, Demirci H, Elner VM, Nelson CC, Kim DS, Joseph SS, Swiecicki PL, Worden FP. Vismodegib for Preservation of Visual Function in Patients with Advanced Periocular Basal Cell Carcinoma: The VISORB Trial. Oncologist. 2021 Jul;26(7):e1240-e1249. doi: 10.1002/onco.13820. Epub 2021 May 31.
Results Reference
derived
PubMed Identifier
27054919
Citation
Rao RC, Chan MP, Andrews CA, Kahana A. EZH2, Proliferation Rate, and Aggressive Tumor Subtypes in Cutaneous Basal Cell Carcinoma. JAMA Oncol. 2016 Jul 1;2(7):962-3. doi: 10.1001/jamaoncol.2016.0021. No abstract available. Erratum In: JAMA Oncol. 2016 Jul 1;2(7):966.
Results Reference
derived
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VISmodegib for ORbital and Periocular Basal Cell Carcinoma
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