This randomized, double-blind, regimen-controlled, phase II, multicenter study will assess the efficacy and safety of two different vismodegib regimens in participants with multiple basal cell carcinoma. Participants will receive vismodegib 150 mg orally once daily either in an intermittent schedule of 12 weeks vismodegib followed by 8 weeks placebo (Arm A) or as 24 weeks induction followed by an intermittent schedule of 8 weeks placebo followed by 8 weeks vismodegib (Arm B). Anticipated time on study treatment is 72 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
229
Vismodegib 150 mg hard gelatin capsule orally once daily
Vismodegib placebo orally once daily
Mean Percent Change From Baseline in the Number of Clinically Evident Basal Cell Carcinomas at Week 73 (After 72 Weeks of Treatment)
The total number of clinically evident basal cell carcinomas = the total number of target and/or non-target lesions present in individual participants.
Time frame: Baseline; Week 73
Percentage of Participants Who Discontinued Study Treatment Due to Tolerability Issues
The percentage of participants who discontinued study treatment (due either to adverse event, refusal of treatment, or withdrawal of consent) was summarized by treatment group.
Time frame: Baseline to Week 73
Mean Percent Change From Baseline in Total Size of Three Target Basal Cell Carcinoma Lesions in Individual Participants at Week 73
The three target basal cell carcinoma lesions = the three largest visible lesions, at least 5 mm in the longest diameter, in individual participants.
Time frame: Baseline; Week 73
Percentage of Participants With at Least 50% Reduction in the Number of Basal Cell Carcinomas at Week 73
Time frame: Baseline; Week 73
Percentage of Participants With New Basal Cell Carcinomas at Week 73
Time frame: Baseline; Week 73
Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 85 (12 Weeks Following End of Treatment) (Recurrence Rate)
Time frame: Baseline; Week 85
Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 97 (24 Weeks Following End of Treatment) (Recurrence Rate)
Time frame: Baseline; Week 97
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Dermatology Research Associate
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
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San Diego, California, United States
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Santa Rosa, California, United States
Advanced Derm & Cosmetic Surg
Ormond Beach, Florida, United States
Skin and Cancer Associates and the Center for Cosmetic Enhancement
Plantation, Florida, United States
Emory University Clinic
Atlanta, Georgia, United States
Laser & Skin Surgery Center of Indiana
Carmel, Indiana, United States
Beverly Hospital;Oncology Center Pharmacy
Beverly, Massachusetts, United States
Saint Louis University School of Medicine; Department of Dermatology
St Louis, Missouri, United States
...and 48 more locations
Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 125 (52 Weeks Following End of Treatment) (Recurrence Rate)
Time frame: Baseline; Week 125
Percentage of Participants Experiencing Any Adverse Event
Time frame: Up to 125 weeks
Percent Change From Baseline in the Skindex-16 Symptom Domain Score at Week 73
The Skindex-16 is a patient-reported outcome health questionnaire. Participants were asked about their symptoms, and their answers were combined into a composite Symptom Domain Score. Scores range from 0 ("never bothered") to 100 ("always bothered").
Time frame: Baseline; Week 73
Percent Change From Baseline in the Skindex-16 Emotion Domain Score at Week 73
The Skindex-16 is a patient-reported outcome health questionnaire. Participants were asked about their emotional state, and their answers were combined into a composite Emotion Domain Score. Scores range from 0 ("never bothered") to 100 ("always bothered").
Time frame: Baseline; Week 73
Percent Change From Baseline in the Skindex-16 Function Domain Score at Week 73
The Skindex-16 is a patient-reported outcome health questionnaire. Participants were asked about their ability to function, and answers were combined into a composite Function Domain Score. Scores range from 0 ("never bothered") to 100 ("always bothered").
Time frame: Baseline; Week 73