Clinical trial NCT02436408: VISmodegib for ORbital and Periocular Basal Cell Carcinoma
Official title: VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Carcinoma, Basal Cell
Lead sponsor: University of Michigan Rogel Cancer Center
Sponsor class: OTHER
First posted: 2015-05-06
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
timeFrame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Secondary outcomes:
measure: Number of Patients With Progressive Disease (PD)
timeFrame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
measure: Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
timeFrame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
measure: Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
timeFrame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: University of Michigan Hospital
state: Michigan
Study officials:
affiliation: University of Michigan
name: Cagri Besirli, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.