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

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

Official Title
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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

Conditions
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Basal Cell Carcinoma

Intervention / Treatment
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* Drug: Placebo
* Drug: Vismodegib


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

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

What is the study measuring?
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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) |