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)
Skin and Cancer Associates and the Center for Cosmetic Enhancement
🇺🇸Plantation, Florida, United States
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Dermetics
🇨🇦Burlington, Ontario, Canada
Long Island Skin Cancer and Dermatologic Surgery
🇺🇸Smithtown, New York, United States
Dermatology Research Associate
🇺🇸Los Angeles, California, United States
The Skin Surgery Center
🇺🇸Winston-Salem, North Carolina, United States
UBC Department of Dermatology & Skin Sciences
🇨🇦Vancouver, British Columbia, Canada
Saint Louis University School of Medicine; Department of Dermatology
🇺🇸Saint Louis, Missouri, United States
Emory University Clinic
🇺🇸Atlanta, Georgia, United States
LKH Innsbruck; Universitätsklinik für Dermatologie
🇦🇹Innsbruck, Austria
Klinik Johann Wolfgang von Goethe Uni; Klinik für Dermatologie, Venerologie und Allergologie
🇩🇪Frankfurt, Germany
CHU Amiens - Hopital Sud
🇫🇷Amiens, France
Hopital Saint Andre CHU De Bordeaux; Dermatologie
🇫🇷Bordeaux, France
Hopital Nord ; Dermatologie
🇫🇷Saint-Etienne, France
Medizinische Hochschule Hannover; Klinik für Dermatologie, Allergologie und Venerologie
🇩🇪Hannover, Germany
Laser & Skin Surgery Center of Indiana
🇺🇸Carmel, Indiana, United States
Academ Ziekenhuis Groningen; Medical Oncology
🇳🇱Groningen, Netherlands
Hopital Dupuytren; Dermatologie
🇫🇷Limoges, France
Chu Site Du Bocage;Dermatologie
🇫🇷Dijon, France
Innovaderm Research Inc.
🇨🇦Montreal, Quebec, Canada
Advanced Derm & Cosmetic Surg
🇺🇸Ormond Beach, Florida, United States
Ohio State University
🇺🇸Columbus, Ohio, United States
Hopital Timone Adultes; Dermatologie
🇫🇷Marseille, France
UNI-Klinikum Campus Kiel Klinik f.Dermatologie Tagesklinik f.Dermatologie
🇩🇪Kiel, Germany
Hospital General Universitario de Valencia; Servicio de oncologia
🇪🇸Valencia, Spain
Azienda Ospedaliera Città della Salute e della Scienza di Torino
🇮🇹Torino, Piemonte, Italy
Medizinische Universität Wien; Univ.Klinik für Dermatologie
🇦🇹Wien, Austria
Hopital Hotel Dieu Et Hme; Clinique Dermatologique
🇫🇷Nantes, France
Hopital Saint Eloi; CHU de Montpellier; Svc de Dermatologie
🇫🇷Montpellier, France
Klinikum der LMU München; Klinik und Poliklinik für Dermatologie und Allergologie
🇩🇪München, Germany
Stanford University
🇺🇸Palo Alto, California, United States
Skin Surgery Med Group, Inc
🇺🇸San Diego, California, United States
Mariwalla Dermatology
🇺🇸West Islip, New York, United States
Beverly Hospital;Oncology Center Pharmacy
🇺🇸Beverly, Massachusetts, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Dermatology and Laser Center of Charleston PA
🇺🇸Charleston, South Carolina, United States
Universitätsklinik Essen; Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie
🇩🇪Essen, Germany
Ch Francois Mitterrand; Medecine Oncologie
🇫🇷Pau, France
Hôpital Saint-Louis
🇫🇷Paris, France
Universitätsklinikum Tübingen Universitäts-Hautklinik
🇩🇪Tübingen, Germany
Ospedale San Salvatore (ASL-01); Dip. di Dermatologia U.O.S. di Dermatologia Oncol
🇮🇹L'Aquila, Abruzzo, Italy
SRH Wald-Klinikum Gera; Klinik für Hautkrankheiten und Allergologie
🇩🇪Gera, Germany
Arcispedale Santa Maria Nuova; Dermatologia
🇮🇹Reggio Emilia, Emilia-Romagna, Italy
Ospedale IOT- Palagi Dermatologia 2
🇮🇹Firenze, Toscana, Italy
Università di Brescia; Dipartimento di Dermatologia
🇮🇹Brescia, Lombardia, Italy
Hospital General Dr. Manuel Gea Gonzalez; Dermatology
🇲🇽Mexico, Mexico
VU MEDISCH CENTRUM;Afdeling Dermatologie
🇳🇱Amsterdam, Netherlands
Hospital General de México
🇲🇽Mexico City, Mexico
Blokhin Cancer Research Center; General Oncology Department
🇷🇺Moscow, Russian Federation
Maastricht University Medical Centre; Dermatologie
🇳🇱Maastricht, Netherlands
FSBI "Russian Oncology Research Center n.a. N. N. Blokhin" of Ministry of Health of the Russian Fed
🇷🇺Moscow, Russian Federation
Hospital Costa Del Sol; Servicio de Dermatologia
🇪🇸Málaga, Malaga, Spain
City Clinical Oncology Dispensary
🇷🇺Saint-Petersburg, Russian Federation
Instituto Valenciano Oncologia; Oncologia Medica
🇪🇸Valencia, Spain
Hospital Clinic i Provincial; Servicio de Farmacia
🇪🇸Barcelona, Spain
Hospital General Universitario de Guadalajara; Servicio de Dermatologia
🇪🇸Guadalajara, Spain
California Pacific Medical Center Research Institute
🇺🇸Santa Rosa, California, United States
Fachklinik Hornheide; Dermatologie
🇩🇪Münster, Germany