A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Regorafenib Versus Placebo in Advanced/Metastatic, Treatment-Refractory Liposarcoma: Results from the SARC024 Study
Year: 2020
Type: article
Abstract: LESSONS LEARNED: The results from the liposarcoma cohort of SARC024 confirm previously published data and do not support the routine use of regorafenib in this patient population. Continued exploration of novel therapies, including combination approaches, is warranted for a patient population in whom limited treatment options exist. BACKGROUND: Regorafenib is a multitargeted kinase inhibitor with a kinase profile overlapping, but distinct from, pazopanib, an agent approved for recurrent and metastatic non-gastrointestinal stromal tumor (GIST), non-adipocytic soft tissue sarcoma. We conducted a randomized, phase II study of regorafenib versus placebo in refractory liposarcoma patients. METHODS: Patients with advanced or metastatic, treatment-refractory liposarcoma were randomized 1:1 to receive regorafenib 160 mg or placebo once daily (3 weeks on, 1 week off). Patients with well-differentiated liposarcoma only were excluded. Crossover for placebo was allowed upon progression. The primary endpoint was progression-free survival (PFS), according to RECIST version 1.1. RESULTS: Forty-eight subjects with liposarcoma (34 dedifferentiated, 12 myxoid/round cell, 2 pleomorphic) were enrolled. Median PFS was 1.87 (95% confidence interval [CI], 0.92-3.67) months for regorafenib versus 2.07 (95% CI, 1.64-3.44) months for placebo; stratified hazard ratio [HR], 0.85 (95% CI, 0.46, 1.58), p = .62. No responses were seen on regorafenib. One PR was observed on placebo. Median overall survival was 6.46 (95% CI, 4.16-23.48) months for regorafenib and 4.89 (95% CI, 3.02-9.77) months for placebo, stratified HR, 0.66 (95% CI, 0.31-1.40), p = .28). Treatment-related adverse events were similar to the known safety profile of regorafenib. CONCLUSION: Regorafenib did not appear to improve PFS in treatment-refractory liposarcoma. No new significant safety signals were observed.
Source: The Oncologist
Authors: Richard F. Riedel, Karla V. Ballman, Yao Lu, Steven Attia, Elizabeth T. Loggers, Kristen N. Ganjoo, Michael B. Livingston, Warren Chow, Jennifer Wright, John H. Ward, Daniel Rushing, Scott H. Okuno, Damon R. Reed, David A. Liebner, Vicki L. Keedy, Leo Mascarenhas, Lara E. Davis, Christopher Ryan, Denise K. Reinke, Robert G. Maki
Institutions: Duke Medical Center, Duke Cancer Institute, Weill Cornell Medicine, Mayo Clinic, Mayo Clinic in Florida, Fred Hutch Cancer Center, Stanford Cancer Institute, Stanford University, Levine Cancer Institute, City Of Hope National Medical Center, City of Hope, Eli Lilly (United States), Huntsman Cancer Institute, Indiana University Indianapolis, Indiana University Melvin and Bren Simon Comprehensive Cancer Center, Moffitt Cancer Center, The Ohio State University, Vanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center, University of Southern California, Children's Hospital Los Angeles, Oregon Health & Science University, OHSU Knight Cancer Institute, Sarcoma Alliance for Research through Collaboration, Abramson Cancer Center, University of Pennsylvania
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $1,500
OpenAlex ID: W3045044516