A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas
- Registration Number
- NCT01815840
- Lead Sponsor
- Hoffmann-La Roche
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 229
- Adult participants, >/= 18 years of age
- Participants with multiple basal cell carcinomas, including participants with Gorlin syndrome, with at least 6 clinically evident basal cell carcinomas at the time of randomization, of which 3 measure 5 mm or more in diameter and are considered target lesions. All other lesions are considered to be non-target lesions
- Histopathologic confirmation that at least one of the three target lesions is basal cell carcinoma
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
- Adequate renal and hepatic function and hematopoietic capacity
- Women of childbearing potential must agree to use contraception as defined by protocol during treatment and for at least 9 months after completion of study treatment
- Male participants with female partners of childbearing potential must agree to use contraception as defined by protocol during treatment and for 2 months after completion of study treatment
- Inability or unwillingness to swallow capsules
- Pregnant or breastfeeding women
- Any metastatic basal cell carcinoma
- Locally advanced basal cell carcinoma lesion that is considered to be inoperable or to have medical contraindications to surgery
- Recent (i.e., within the past 28 days prior to randomization) or current participation in another experimental drug study
- Known or suspected alcohol abuse
- One of the following known rare hereditary conditions: galactose intolerance, primary hypolactasia or glucose-galactose malabsorption
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Vismodegib Intermittent Schedule Vismodegib Vismodegib intermittent schedule of 12 weeks vismodegib followed by 8 weeks placebo, repeated 3 times with a final course of vismodegib (total 72 weeks), followed by 52 weeks treatment-free follow up Vismodegib Intermittent Schedule Placebo Vismodegib intermittent schedule of 12 weeks vismodegib followed by 8 weeks placebo, repeated 3 times with a final course of vismodegib (total 72 weeks), followed by 52 weeks treatment-free follow up Vismodegib Induction Followed by Intermittent Schedule Vismodegib Vismodegib beginning with 24 weeks induction followed by intermittent schedule 8 weeks placebo, 8 weeks vismodegib (total 72 weeks), followed by 52 weeks treatment-free follow up Vismodegib Induction Followed by Intermittent Schedule Placebo Vismodegib beginning with 24 weeks induction followed by intermittent schedule 8 weeks placebo, 8 weeks vismodegib (total 72 weeks), followed by 52 weeks treatment-free follow up
- Primary Outcome Measures
Name Time Method Mean Percent Change From Baseline in the Number of Clinically Evident Basal Cell Carcinomas at Week 73 (After 72 Weeks of Treatment) Baseline; Week 73 The total number of clinically evident basal cell carcinomas = the total number of target and/or non-target lesions present in individual participants.
- Secondary Outcome Measures
Name Time Method Percentage of Participants Who Discontinued Study Treatment Due to Tolerability Issues Baseline to Week 73 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.
Mean Percent Change From Baseline in Total Size of Three Target Basal Cell Carcinoma Lesions in Individual Participants at Week 73 Baseline; 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.
Percentage of Participants With at Least 50% Reduction in the Number of Basal Cell Carcinomas at Week 73 Baseline; Week 73 Percentage of Participants With New Basal Cell Carcinomas at Week 73 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) 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) Baseline; Week 97 Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 125 (52 Weeks Following End of Treatment) (Recurrence Rate) Baseline; Week 125 Percentage of Participants Experiencing Any Adverse Event Up to 125 weeks Percent Change From Baseline in the Skindex-16 Symptom Domain Score at Week 73 Baseline; 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").
Percent Change From Baseline in the Skindex-16 Emotion Domain Score at Week 73 Baseline; 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").
Percent Change From Baseline in the Skindex-16 Function Domain Score at Week 73 Baseline; 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").
Trial Locations
- Locations (58)
Dermatology Research Associate
🇺🇸Los Angeles, California, United States
Stanford University
🇺🇸Palo Alto, California, United States
Skin Surgery Med Group, Inc
🇺🇸San Diego, California, United States
California Pacific Medical Center Research Institute
🇺🇸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
🇺🇸Saint Louis, Missouri, United States
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