VISmodegib for ORbital and Periocular Basal Cell Carcinoma

NCT02436408 | Completed Phase 4 Results DMC

• 2 other identifiers: UMCC 2014.022HUM00082579
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

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.

Conditions

Carcinoma, Basal Cell

Outcome Measures

Primary Outcomes (1)

• 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.

  Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Secondary Outcomes (3)

• 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.

• 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.

• 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.

Study Arms (1)

Vismodegib

EXPERIMENTAL

150mg taken orally once daily

Drug: Vismodegib

Interventions

Vismodegib DRUG

Vismodegib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Michigan Rogel Cancer Center: lead

Study Sites (1)

University of Michigan Hospital

Ann Arbor, Michigan, 48109, United States

Location

Related Publications (3)

• Unsworth SP, Tingle CF, Heisel CJ, Eton EA, Andrews CA, Chan MP, Bresler SC, Kahana A. Analysis of residual disease in periocular basal cell carcinoma following hedgehog pathway inhibition: Follow up to the VISORB trial. PLoS One. 2022 Dec 1;17(12):e0265212. doi: 10.1371/journal.pone.0265212. eCollection 2022.

  PMID: 36455049 DERIVED

• 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.

  PMID: 33988881 DERIVED

• 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.

  PMID: 27054919 DERIVED

MeSH Terms

Conditions

Carcinoma, Basal Cell

Interventions

HhAntag691

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Basal Cell

Limitations and Caveats

The initial study design aimed to enroll 50 patients; however, the study was terminated early as a consequence of the therapeutic and administrative challenges posed by the novel coronavirus pandemic and because the interim analysis revealed that the study at the point of termination was sufficient to achieve statistical significance.

Results Point of Contact

• Title: Cagri Besirli, MD, PhD
• Organization: University of Michigan Rogel Cancer Center

cbesirli@med.umich.edu

734-232-8404

Study Officials

• Cagri Besirli, M.D., Ph.D.

  University of Michigan

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 1, 2015
• First Posted: May 6, 2015
• Study Start: July 15, 2015
• Primary Completion: September 1, 2020
• Study Completion: September 1, 2020
• Last Updated: October 28, 2021
• Results First Posted: October 28, 2021

Record last verified: 2021-09

Locations

US (1)