VISmodegib for ORbital and Periocular Basal Cell Carcinoma

NCT ID: NCT02436408

Last Updated: 2021-10-28

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-15

Study Completion Date

2020-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Carcinoma, Basal Cell

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Vismodegib

150mg taken orally once daily

Group Type EXPERIMENTAL

Vismodegib

Intervention Type DRUG

Interventions

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Vismodegib

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Michigan Rogel Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cagri Besirli, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

University of Michigan

Locations

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University of Michigan Hospital

Ann Arbor, Michigan, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 36455049 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33988881 (View on PubMed)

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.

Reference Type DERIVED
PMID: 27054919 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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HUM00082579

Identifier Type: OTHER

Identifier Source: secondary_id

UMCC 2014.022

Identifier Type: -

Identifier Source: org_study_id