Vismodegib in Treating Patients With Basal Cell Carcinoma

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Stanford University
ClinicalTrials.gov Identifier:
NCT01631331
First received: June 25, 2012
Last updated: August 28, 2013
Last verified: August 2013
  Purpose

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


Condition Intervention
Basal Cell Carcinoma of the Skin
Recurrent Skin Cancer
Drug: vismodegib
Procedure: Mohs surgery

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCC)

Resource links provided by NLM:


Further study details as provided by Stanford University:

Primary Outcome Measures:
  • Anti-tumor effect as measured by the percent change in surgical defect area against the null hypothesis of no change after the treatment period using calipers and photographs [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]
    This will be tested against the null hypothesis of no change as a one-sample t-test. A p-value of less than 0.05 will be considered statistically significant. We will first perform non-parametric paired t-test taking this account, and also use more sophisticated methods such as the MIXED procedure (PROC MIXED) treating each subject as a random effect and time as fixed effect, with appropriate correlation structure.


Secondary Outcome Measures:
  • Proportion of tumors with skip lesions present determined using bread-loafing of tumor block and histology [ Time Frame: Assessed up to 6 months ] [ Designated as safety issue: No ]
  • Average area of the subclinical tumor invasion calculated from the pre-surgical skin markings and by comparing the initial clinical margins to the final Mohs defect [ Time Frame: Assessed up to 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: June 2012
Estimated Study Completion Date: November 2015
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment (vismodegib and Mohs surgery)
Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.
Drug: vismodegib
Given PO
Other Names:
  • Erivedge
  • GDC-0449
  • Hedgehog antagonist GDC-0449
Procedure: Mohs surgery
Undergo Mohs surgery

Detailed Description:

PRIMARY OBJECTIVES:

I. The percent reduction in surgical defect area/size surrounding BCC tumor pre and post-vismodegib.

SECONDARY OBJECTIVES:

I. Bread-loafing histology will be used to determine if the tumor after vismodegib shrinks as one discrete lesion (contiguous) vs. with skip areas (non-contiguous) II. The percent reduction in tumor size pre and post-vismodegib. III. Safety, tolerability and percent drop-out after 3 vs. 6 months of vismodegib in otherwise healthy patients.

OUTLINE:

Patients receive vismodegib orally (PO) once daily (QD) for up to 3 months if the initial BCC size is < 2 cm and superficial or for up to 6 months if the initial BCC size is >= 2 cm or non-superficial. After completion of vismodegib treatment, patients undergo Mohs surgery.

After completion of study treatment, patients are followed up for 6 months.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01631331

Locations
United States, California
Stanford University
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Jean Tang Stanford University
  More Information

No publications provided

Responsible Party: Stanford University
ClinicalTrials.gov Identifier: NCT01631331     History of Changes
Other Study ID Numbers: SKIN0012, NCI-2012-01055, 24313
Study First Received: June 25, 2012
Last Updated: August 28, 2013
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Carcinoma
Skin Neoplasms
Carcinoma, Basal Cell
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms by Site
Skin Diseases
Neoplasms, Basal Cell

ClinicalTrials.gov processed this record on August 25, 2014