Open AccessA Randomized, Double‐Blind , Placebo‐Controlled , Phase II Study of Regorafenib Versus Placebo in Advanced/Metastatic, Treatment‐Refractory Liposarcoma: Results from the SARC024 Study
Author(s) -
Riedel Richard F.,
Ballman Karla V.,
Lu Yao,
Attia Steven,
Loggers Elizabeth T.,
Ganjoo Kristen N.,
Livingston Michael B.,
Chow Warren,
Wright Jennifer,
Ward John H.,
Rushing Daniel,
Okuno Scott H.,
Reed Damon R.,
Liebner David A.,
Keedy Vicki L.,
Mascarenhas Leo,
Davis Lara E.,
Ryan Christopher,
Reinke Denise K.,
Maki Robert G.
Publication year - 2020
Publication title -
the oncologist
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.176
H-Index - 164
eISSN - 1549-490X
pISSN - 1083-7159
DOI - 10.1634/theoncologist.2020-0679
Subject(s) - regorafenib , medicine , placebo , pazopanib , hazard ratio , clinical endpoint , population , soft tissue sarcoma , liposarcoma , surgery , gastroenterology , randomized controlled trial , oncology , sarcoma , confidence interval , colorectal cancer , pathology , sunitinib , renal cell carcinoma , cancer , soft tissue , alternative medicine , environmental health
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.