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A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Regorafenib Versus Placebo in Advanced/Metastatic,
Richard F Riedel1, Karla V Ballman2, Yao Lu2
1Duke Cancer Institute, Duke University Medical Center, Durham, North Carolina, USA.
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.
Insights
Regorafenib did not improve progression-free survival in patients with refractory liposarcoma. Further research into new therapies is needed for this patient group with limited options.
Area of Science:
- Oncology
- Pharmacology
Background:
- Regorafenib is a multi-targeted kinase inhibitor.
- It has a distinct kinase profile compared to pazopanib, which is approved for certain soft tissue sarcomas.
Purpose of the Study:
- To evaluate the efficacy of regorafenib compared to placebo in patients with refractory liposarcoma.
- To assess progression-free survival (PFS) as the primary endpoint.
Main Methods:
- A randomized, phase II study was conducted.
- Patients with advanced or metastatic, treatment-refractory liposarcoma received either regorafenib (160 mg daily) or placebo.
- The treatment cycle was 3 weeks on, 1 week off.
Main Results:
- Forty-eight patients were enrolled, with most having dedifferentiated liposarcoma.
- Median PFS was similar between the regorafenib and placebo groups (1.87 vs. 2.07 months).
- No objective responses were observed with regorafenib; one partial response occurred with placebo.
Conclusions:
- Regorafenib did not demonstrate a significant improvement in PFS for treatment-refractory liposarcoma.
- No new safety concerns were identified with regorafenib use.
- Novel therapeutic strategies, including combination therapies, are necessary for liposarcoma patients with limited treatment alternatives.
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