Original articlePivotal ERIVANCE basal cell carcinoma (BCC) study: 12-month update of efficacy and safety of vismodegib in advanced BCC
Section snippets
Study design
The ERIVANCE BCC methods have been reported previously.7 Briefly, ERIVANCE BCC is a pivotal, phase-II, single-arm, 2-cohort, multicenter clinical trial to evaluate the efficacy and safety of vismodegib in patients with advanced BCC (ClinicalTrials.gov number NCT00833417). The study was conducted in accordance with FDA regulations and the International Conference on Harmonization E6 Guideline for Good Clinical Practice. The protocol was approved by an independent review board or ethics committee
Patient demographics and baseline characteristics
A total of 104 patients (mBCC n = 33, laBCC n = 71) were enrolled at 31 sites in the United States, Europe, and Australia. Eight patients in the laBCC cohort were excluded from efficacy analysis because they did not have histologically confirmed BCC by tumor biopsy performed at enrollment; however, all patients were considered safety evaluable (see Consolidated Standards of Reporting Trials [CONSORT] diagram [eFig 1]). Patient baseline characteristics have been previously described.7
Treatment exposure
At the data
Discussion
Vismodegib, an oral, selective inhibitor of the Hedgehog signaling pathway, which is critical in BCC pathogenesis,1, 5, 6 has been approved by the FDA as a treatment option for adult patients with a BCC.8, 9 Approval was based on results from the primary analysis of the pivotal ERIVANCE BCC study.7 The results presented here, after an additional 12 months of patient follow-up, confirm the results of the primary analysis and demonstrate durable responses to vismodegib treatment. At the
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This study was funded by F. Hoffmann-LaRoche and was designed by the investigators and representatives of the sponsor. The sponsor also was responsible for data collection and analysis. Medical writing assistance was provided by Saema Magre, PhD, Tony Serino, PhD, and David Gibson, PhD, CMPP, of ApotheCom (Yardley, PA) and was funded by F. Hoffmann-La Roche.
Dr Sekulic serves as a consultant for F. Hoffman-LaRoche (uncompensated). Dr Migden serves as a consultant for Genentech, Novartis, and Lilly and has received honoraria from Genentech, Novartis, and Lilly. Dr Lewis serves as a consultant for F. Hoffman-LaRoche, has received honoraria from F. Hoffman-LaRoche, and his institution has received grant funding for clinical trials from F. Hoffman-LaRoche. Dr Hainsworth's institution has received grant funding for clinical trials from Genentech. Dr Solomon is a consultant for Genentech, has received honoraria from Genentech, and his institution has received grant funding for clinical trials from Genentech. Dr Yoo is a consultant for Genentech, has received honoraria from Genentech, and his institution has received grant funding for clinical trials from Genentech. Dr Arron serves as a consultant for Genentech, Kythera, Allergan, Anacor, Lilly, and F. Hoffmann-LaRoche, has received salary from Genentech, Kythera, Allergan, Anacor, and Lilly, and has received honoraria from F. Hoffmann-LaRoche. Dr Friedlander serves as a consultant for Genentech and Bristol-Myers, has received honoraria from Genentech and Bristol-Myers Squibb, and his institution has received grant funding from Genentech. Dr Marmur serves as a consultant for Allergan and Dusa Pharmaceuticals. Dr Rudin serves as a consultant for Novartis and Lilly and has received honoraria from Novartis and Lilly. Dr Chang has received grants for clinical trials from F. Hoffmann-La Roche, Novartis, and Lilly. Dr Dirix declared no conflicts of interest. Dr Hou is an employee of Genentech and has received salary, stock options, and stock from Genentech. Dr Yue is an employee of F. Hoffmann-La Roche and has received salary, stock options, and stock from F. Hoffmann-LaRoche. Dr Hauschild serves as a consultant to Amgen, Bristol-Myers Squibb, Celgene, Eisai, GlaxoSmithKline, MedImmune, MelaSciences, Merck Serono, Merck, Novartis, Oncosec, and F. Hoffmann-LaRoche, received honoraria from Amgen, Bristol-Myers Squibb, Celgene, Eisai, GlaxoSmithKline, MedImmune, MelaSciences, Merck Serono, Merck, Novartis, Oncosec, and F. Hoffmann-LaRoche, and grants for clinical trials from Amgen, Bristol-Myers Squibb, Celgene, Eisai, GlaxoSmithKline, MelaSciences, Merck Serono, Merck, Novartis, Oncosec, and F. Hoffmann-LaRoche.
Supplementary data, eTables, and eFigures are available at http://www.jaad.org.