Clinical trial NCT01815840: A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas
Official title: A Randomized, Double-blinded, Regimen-controlled, Phase II, Multicenter Study to Assess the Efficacy and Safety of Two Different Vismodegib Regimens in Patients With Multiple Basal Cell Carcinomas
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Basal Cell Carcinoma
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2013-03-21
Enrollment: 229
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Mean Percent Change From Baseline in the Number of Clinically Evident Basal Cell Carcinomas at Week 73 (After 72 Weeks of Treatment)
timeFrame: Baseline; Week 73
Secondary outcomes:
measure: Percentage of Participants Who Discontinued Study Treatment Due to Tolerability Issues
timeFrame: Baseline to Week 73
measure: Mean Percent Change From Baseline in Total Size of Three Target Basal Cell Carcinoma Lesions in Individual Participants at Week 73
timeFrame: Baseline; Week 73
measure: Percentage of Participants With at Least 50% Reduction in the Number of Basal Cell Carcinomas at Week 73
timeFrame: Baseline; Week 73
measure: Percentage of Participants With New Basal Cell Carcinomas at Week 73
timeFrame: Baseline; Week 73
measure: Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 85 (12 Weeks Following End of Treatment) (Recurrence Rate)
timeFrame: Baseline; Week 85
measure: Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 97 (24 Weeks Following End of Treatment) (Recurrence Rate)
timeFrame: Baseline; Week 97
measure: Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 125 (52 Weeks Following End of Treatment) (Recurrence Rate)
timeFrame: Baseline; Week 125
measure: Percentage of Participants Experiencing Any Adverse Event
timeFrame: Up to 125 weeks
measure: Percent Change From Baseline in the Skindex-16 Symptom Domain Score at Week 73
timeFrame: Baseline; Week 73
measure: Percent Change From Baseline in the Skindex-16 Emotion Domain Score at Week 73
timeFrame: Baseline; Week 73
measure: Percent Change From Baseline in the Skindex-16 Function Domain Score at Week 73
timeFrame: Baseline; Week 73
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Dermatology Research Associate
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: Skin Surgery Med Group, Inc
state: California
city: Santa Rosa
country: United States
facility: California Pacific Medical Center Research Institute
state: California
city: Ormond Beach
country: United States
facility: Advanced Derm & Cosmetic Surg
state: Florida
city: Plantation
country: United States
facility: Skin and Cancer Associates and the Center for Cosmetic Enhancement
state: Florida
city: Atlanta
country: United States
facility: Emory University Clinic
state: Georgia
city: Carmel
country: United States
facility: Laser & Skin Surgery Center of Indiana
state: Indiana
city: Beverly
country: United States
facility: Beverly Hospital;Oncology Center Pharmacy
state: Massachusetts
city: Saint Louis
country: United States
facility: Saint Louis University School of Medicine; Department of Dermatology
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Smithtown
country: United States
facility: Long Island Skin Cancer and Dermatologic Surgery
state: New York
city: West Islip
country: United States
facility: Mariwalla Dermatology
state: New York
city: Winston-Salem
country: United States
facility: The Skin Surgery Center
state: North Carolina
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Dermatology and Laser Center of Charleston PA
state: South Carolina
city: Innsbruck
country: Austria
facility: LKH Innsbruck; Universitätsklinik für Dermatologie
city: Wien
country: Austria
facility: Medizinische Universität Wien; Univ.Klinik für Dermatologie
city: Vancouver
country: Canada
facility: UBC Department of Dermatology & Skin Sciences
state: British Columbia
city: Burlington
country: Canada
facility: Dermetics
state: Ontario
city: Montreal
country: Canada
facility: Innovaderm Research Inc.
state: Quebec
city: Amiens
country: France
facility: CHU Amiens - Hopital Sud
city: Bordeaux
country: France
facility: Hopital Saint Andre CHU De Bordeaux; Dermatologie
city: Dijon
country: France
facility: Chu Site Du Bocage;Dermatologie
city: Limoges
country: France
facility: Hopital Dupuytren; Dermatologie
city: Marseille
country: France
facility: Hopital Timone Adultes; Dermatologie
city: Montpellier
country: France
facility: Hopital Saint Eloi; CHU de Montpellier; Svc de Dermatologie
city: Nantes
country: France
facility: Hopital Hotel Dieu Et Hme; Clinique Dermatologique
city: Paris
country: France
facility: Hôpital Saint-Louis
city: Pau
country: France
facility: Ch Francois Mitterrand; Medecine Oncologie
city: Saint-Etienne
country: France
facility: Hopital Nord ; Dermatologie
city: Essen
country: Germany
facility: Universitätsklinik Essen; Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie
city: Frankfurt
country: Germany
facility: Klinik Johann Wolfgang von Goethe Uni; Klinik für Dermatologie, Venerologie und Allergologie
city: Gera
country: Germany
facility: SRH Wald-Klinikum Gera; Klinik für Hautkrankheiten und Allergologie
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover; Klinik für Dermatologie, Allergologie und Venerologie
city: Kiel
country: Germany
facility: UNI-Klinikum Campus Kiel Klinik f.Dermatologie Tagesklinik f.Dermatologie
city: München
country: Germany
facility: Klinikum der LMU München; Klinik und Poliklinik für Dermatologie und Allergologie
city: Münster
country: Germany
facility: Fachklinik Hornheide; Dermatologie
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen Universitäts-Hautklinik
city: L'Aquila
country: Italy
facility: Ospedale San Salvatore (ASL-01); Dip. di Dermatologia U.O.S. di Dermatologia Oncol
state: Abruzzo
city: Reggio Emilia
country: Italy
facility: Arcispedale Santa Maria Nuova; Dermatologia
state: Emilia-Romagna
city: Brescia
country: Italy
facility: Università di Brescia; Dipartimento di Dermatologia
state: Lombardia
city: Torino
country: Italy
facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino
state: Piemonte
city: Firenze
country: Italy
facility: Ospedale IOT- Palagi Dermatologia 2
state: Toscana
city: Mexico City
country: Mexico
facility: Hospital General de México
city: Mexico
country: Mexico
facility: Hospital General Dr. Manuel Gea Gonzalez; Dermatology
city: Amsterdam
country: Netherlands
facility: VU MEDISCH CENTRUM;Afdeling Dermatologie
city: Groningen
country: Netherlands
facility: Academ Ziekenhuis Groningen; Medical Oncology
city: Maastricht
country: Netherlands
facility: Maastricht University Medical Centre; Dermatologie
city: Moscow
country: Russian Federation
facility: Blokhin Cancer Research Center; General Oncology Department
city: Moscow
country: Russian Federation
facility: FSBI "Russian Oncology Research Center n.a. N. N. Blokhin" of Ministry of Health of the Russian Fed
city: Saint-Petersburg
country: Russian Federation
facility: City Clinical Oncology Dispensary
city: Málaga
country: Spain
facility: Hospital Costa Del Sol; Servicio de Dermatologia
state: Malaga
city: Barcelona
country: Spain
facility: Hospital Clinic i Provincial; Servicio de Farmacia
city: Guadalajara
country: Spain
facility: Hospital General Universitario de Guadalajara; Servicio de Dermatologia
city: Valencia
country: Spain
facility: Hospital General Universitario de Valencia; Servicio de oncologia
city: Valencia
country: Spain
facility: Instituto Valenciano Oncologia; Oncologia Medica
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Jacobsen AA, Kydd AR, Strasswimmer J. Practical management of the adverse effects of Hedgehog pathway inhibitor therapy for basal cell carcinoma. J Am Acad Dermatol. 2017 Apr;76(4):767-768. doi: 10.1016/j.jaad.2016.04.063. No abstract available.
citation: Dreno B, Kunstfeld R, Hauschild A, Fosko S, Zloty D, Labeille B, Grob JJ, Puig S, Gilberg F, Bergstrom D, Page DR, Rogers G, Schadendorf D. Two intermittent vismodegib dosing regimens in patients with multiple basal-cell carcinomas (MIKIE): a randomised, regimen-controlled, double-blind, phase 2 trial. Lancet Oncol. 2017 Mar;18(3):404-412. doi: 10.1016/S1470-2045(17)30072-4. Epub 2017 Feb 8.