A Study of Vismodegib With Surgery in Participants With Previously Untreated Basal Cell Carcinoma

A Randomized, Double-Blind, Placebo-Controlled, Phase II Study to Assess the Efficacy and Safety of Oral Vismodegib for the Treatment of Basal Cell Carcinoma Preceding Excision by Mohs Micrographic Surgery

This randomized, double-blind, placebo-controlled study will assess the efficacy and safety of vismodegib with surgery in participants with basal cell carcinoma.

Study Overview

• Status: Terminated
• Conditions: Basal Cell Carcinoma
• Intervention / Treatment: Drug: Placebo; Drug: Vismodegib

• Study Type: Interventional
• Enrollment (Actual): 18
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Mayo Clinic
  • California
    • Beverly Hills, California, United States, 90210
      • Moy-Fincher-Chipps Facial Plastics and Dermatology
    • La Jolla, California, United States, 92037
      • Scripps Clinic
    • Loma Linda, California, United States, 92354
      • Loma Linda University Medical Center
    • Palo Alto, California, United States, 94305
      • Stanford University
    • San Francisco, California, United States, 94107
      • California Pacific Medical Center
    • San Francisco, California, United States, 94115
      • Univ of Calif-San Francisco
  • Florida
    • St. Petersburg, Florida, United States, 33716
      • Spencer Derma & Skin Surg Ctr
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Indiana
    • Carmel, Indiana, United States, 46032
      • Laser & Skin Surgery Center of Indiana
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • New York
    • New York, New York, United States, 10011
      • Beth Israel Cancer Center; West Campus
    • Rochester, New York, United States, 14642
      • University of Rochester Medical Center; University Dermatology Associates
    • West Islip, New York, United States, 11795
      • Mariwalla Dermatology
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27106
      • The Skin Surgery Center
  • Oregon
    • Portland, Oregon, United States, 97239-4501
      • Oregon Health & Science University; Department of Dermatology
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey Medical Center
    • Philadelphia, Pennsylvania, United States, 19111
      • Fox Chase Cancer Center
  • Texas
    • Houston, Texas, United States, 77030-4095
      • MD Anderson Cancer Center
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • Huntsman Cancer Institute at The University of Utah
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of non-infected, not recurrent, previously untreated basal cell carcinoma
• Free of any significant physical abnormalities (e.g., tattoos) at the target basal cell carcinoma site
• Willing and able to participate in the study as an outpatient and agreement to make frequent visits to the clinic during the treatment and follow-up periods and to comply with study requirements

Exclusion Criteria:

• Prior treatment with vismodegib
• Known hypersensitivity to any of the study drug excipients
• Any metastatic basal cell carcinoma
• Any locally advanced basal cell carcinoma considered to be inoperable or to have a medical contraindication to surgery
• Evidence of clinically significant and unstable diseases or conditions (e.g., cardiovascular, immunosuppressive, hematologic)
• Any dermatological disease at the target basal cell carcinoma site that may cause difficulty with examination
• Recent, current, or planned participation in another experimental drug study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Participants will receive matching placebo to vismodegib capsule orally once daily for 12 weeks.	Drug: Placebo; Participants will receive matching placebo to vismodegib for 12 weeks.
Experimental: Vismodegib; Participants will receive vismodegib 150 milligrams (mg) capsule orally once daily for 12 weeks.	Drug: Vismodegib; Participants will receive vismodegib 150 mg oral capsule once a day for 12 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change in Target Basal Cell Carcinoma (BCC) Expected Surgical Defect Area at Mohs Micrographic Surgery (MMS) Visit	The percent change in target BCC expected surgical defect area was defined as ([baseline expected surgical defect area - expected surgical defect area at MMS visit]/ baseline expected surgical defect area) × 100 percent (%) where expected surgical defect area was manually outlined on a digital photograph and measured by a computer (computer aided planimetry). MMS visit was defined as the visit that occurred within 2 weeks of the last study treatment.	Baseline, MMS visit (Week 12-14)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Actual Change in Target BCC Expected Surgical Defect Area at MMS Visit	Actual change was defined as (baseline expected surgical defect area - expected surgical defect area at MMS visit). MMS visit was defined as the visit that occurred within 2 weeks of the last study treatment. Expected surgical defect area was manually outlined on a digital photograph and measured by a computer (computer aided planimetry).	Baseline, MSS Visit (Week 12-14)
Percentage Change in Target BCC Actual Tumor-Free Margin Excision Area at MMS Visit	Percent change in target BCC actual tumor-free margin excision area was defined as = (expected surgical defect area pre-treatment - actual tumor-free margin excision area at MMS visit) / expected surgical defect area pre-treatment) * 100%. The actual tumor-free margin excision area (includes 2 millimeters [mm] margin) was measured during MMS. The area was photographed and traced on the digital photograph then calculated by computer-aided planimetry. MMS visit was defined as the visit that occurred within 2 weeks of the last study treatment.	Baseline, MMS visit (Week 12-14)
Percentage of Participants With Clinical Response	Clinical response was defined as a complete response (CR) or partial response (PR) at the post-treatment MMS excision. CR was defined as no histological evidence of BCC. PR was defined as a reduction of at least 50 % in the expected surgical defect area with histologic evidence of residual BCC. MMS visit was defined the visit that occurred within 2 weeks of the last study treatment.	MMS visit (Week 12-14)
Percentage of Participants With Skip Area	Skip area was defined as the presence of non-contiguous residual tumor at the MMS visit, as determined by an independent dermatopathologist. MMS visit occurred within 2 weeks of the last study treatment.	MMS visit (Week 12-14)
Percentage of Participants With BCC Recurrence		Baseline, 12, 24, and 52 weeks post MMS Visit (MMS Visit = Week 12-14)

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start (Actual): January 23, 2014
• Primary Completion (Actual): January 26, 2016
• Study Completion (Actual): January 26, 2016

Study Registration Dates

• First Submitted: July 3, 2013
• First Submitted That Met QC Criteria: July 9, 2013
• First Posted (Estimate): July 12, 2013

Study Record Updates

• Last Update Posted (Actual): May 8, 2017
• Last Update Submitted That Met QC Criteria: March 27, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Basal Cell; Carcinoma; Carcinoma, Basal Cell
• Other Study ID Numbers: ML28726