Vismodegib in Treating Patients With Basal Cell Carcinoma (BCC)

NCT01631331 | Completed Early Phase 1 Results DMC FDA Drug

• 3 other identifiers: IRB-24313NCI-2012-01055SKIN0012
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to learn about the effect of vismodegib on sporadic basal cell carcinoma (BCCs) prior to surgical removal.

Conditions

Basal Cell Carcinoma of the Skin; Recurrent Skin Cancer

Outcome Measures

Primary Outcomes (1)

• Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated

  At baseline, we selected 1 to 2 tumors per patient for surgery (13 target tumors selected). At baseline,1 Mohs surgeon measured the estimated surgical defect area around the target tumor. For tumors to be excised by Mohs we defined estimated surgical defect as the tumor size plus a 2-mm circumferential margin, presuming tumor clearance after a Mohs stage-1 excision. For the tumor undergoing standard (non-Mohs) excision, we used tumor size plus a standard 4-mm margin11 for the estimated surgical defect. On the day of the surgery, we measured the surgical defect area as the final tumor-free defect after the Mohs procedure or non-Mohs excision immediately before closure. We used the Image J software program (National Institutes of Health, Bethesda, MD) to calculate tumor area (cm2). Only target tumors are included in this analysis.

  average of 4 months

Secondary Outcomes (3)

• Number of Tumors Demonstrating Histologic Cure

  Average of 4 months

• Tumor Recurrence Rate of Treated BCCs

  average of 22 months

• Tumor Size Measurements Before and After Short Term Vismodegib Treatment

  4 months (average)

Study Arms (1)

Treatment (vismodegib and Mohs surgery)

EXPERIMENTAL

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

Drug: vismodegib; Procedure: Mohs surgery

Interventions

vismodegib DRUG

Given PO

Also known as: Erivedge, GDC-0449, Hedgehog antagonist GDC-0449

Treatment (vismodegib and Mohs surgery)

Mohs surgery PROCEDURE

Undergo Mohs surgery

Treatment (vismodegib and Mohs surgery)

Eligibility Criteria

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

You may qualify if:

• Study patients must have at least one BCC, \> 5 mm, eligible for Mohs surgical removal; patients with BCCs that have been treated before (recurrent BCCs, BCCs that failed other chemotherapy) are eligible for this trial, if they meet size criteria
• No Eastern Cooperative Oncology Group (ECOG) or Karnofsky performance status will be employed
• Normal hepatic function: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 2 x the upper limit of normal (ULN)
• Normal renal function : normal serum creatinine defined as \<= 2.5 mg/dL
• Clinically acceptable complete blood count (CBC)
• Ability to understand and the willingness to sign a written informed consent document
• The patient is willing to forego surgical treatment of BCCs by up to 6 months, except when the principal investigator (PI) believes that delay in treatment potentially might compromise the health of the subject
• Documented negative serum pregnancy test for women of childbearing potential, with agreement to the use of two acceptable methods of contraception during the study and for 7 months after discontinuation of vismodegib
• For men with female partners of childbearing potential, agreement to use a latex, non-latex, or any other male condom and to advise their female partners to use an additional acceptable method of birth control during the study and for 2 months after discontinuation of study drug
• Be willing to not donate blood or semen for three months following discontinuation of study medications

You may not qualify if:

• The patient has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) stage 0
• The subject has uncontrolled systemic disease, including known human immunodeficiency virus (HIV) positive patients:
• The patient has history of congestive heart failure
• The patient has clinically important history of liver disease, including viral or hepatitis, current alcohol abuse, or cirrhosis
• The patient has any condition or situation which in the investigator's opinion may put the patient at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study; this includes history of other skin conditions or disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or render the patient at high risk from treatment complications
• The patient has a history of hypersensitivity to any of the ingredients in the study medication formulations
• The patient is willing to abstain from application of non-study topical medications to the skin for the duration of the study, including prescription and over the counter preparations; for example, topical preparations containing corticosteroids or vitamin A derivatives are not allowed
• Pregnant or nursing patients will be excluded from the study

Sponsors & Collaborators

• Stanford University: lead
• National Cancer Institute (NCI): collaborator
• University Hospitals Cleveland Medical Center: collaborator

Study Sites (1)

Stanford University

Stanford, California, 94305, United States

Location

Related Publications (1)

• Ally MS, Aasi S, Wysong A, Teng C, Anderson E, Bailey-Healy I, Oro A, Kim J, Chang AL, Tang JY. An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. J Am Acad Dermatol. 2014 Nov;71(5):904-911.e1. doi: 10.1016/j.jaad.2014.05.020. Epub 2014 Jun 11.

  PMID: 24929884 RESULT

MeSH Terms

Conditions

Carcinoma, Basal Cell; Skin Neoplasms

Interventions

HhAntag691; Mohs Surgery

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Basal Cell; Neoplasms by Site; Skin Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Microsurgery; Surgical Procedures, Operative; Dermatologic Surgical Procedures; Plastic Surgery Procedures

Results Point of Contact

• Title: Dr. Gina Kwon
• Organization: Stanford University

gkwon@stanford.edu

650-724-3859

Study Officials

• Jean Tang

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Dermatology

Study Record Dates

• First Submitted: June 25, 2012
• First Posted: June 29, 2012
• Study Start: June 1, 2012
• Primary Completion: September 17, 2015
• Study Completion: May 31, 2016
• Last Updated: December 8, 2017
• Results First Posted: October 2, 2017

Record last verified: 2017-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)