Regorafenib in Patients with Refractory Metastatic Pancreatic Cancer: A Phase II Study (RESOUND)

Abstract

Aim: Regorafenib may be active in different cancer types. This Phase II trial included patients with various refractory cancer types treated with regorafenib. Here, we report the results of the pancreatic adenocarcinoma cohort. Methods: The primary end point was progression-free survival (PFS) rate at 8 weeks; further investigation of regorafenib would be warranted with a PFS rate ≥50%. Results: A total of 20 patients were enrolled. The best response was stable disease in four patients (20%). The 8-week PFS rate was 25% with a median PFS of 1.7 months (95% CI: 1.5–2.0). A total of 13 patients (65%) experienced grade 3–4 treatment-related adverse events. Conclusion: The study did not meet its primary end point. Further investigation of regorafenib monotherapy in this setting is not recommended.

Clinical Trial Registration: NCT02307500

Keywords::

• advanced
• angiogenesis
• metastatic
• pancreatic cancer
• regorafenib

Availability of data & materials

Data are available from the corresponding author upon reasonable request.

Author contributions

A Santoro conceived and designed the study, analyzed and interpreted data and prepared, edited and reviewed the manuscript. S Bozzarelli conceived and designed the study, acquired, controlled, analyzed and interpreted data and algorithms and prepared, edited and reviewed the manuscript. L Rimassa analyzed and interpreted data and prepared, edited and reviewed the manuscript. L Giordano controlled, analyzed and interpreted data and algorithms and did statistical analysis. S Sala acquired, controlled, analyzed and interpreted data and algorithms. MC Tronconi, T Pressiani, V Smiroldo, N Personeni, MG Prete and P Spaggiari helped in analysis and interpretation of data and in preparing the manuscript.

Data sharing statement

The authors certify that this manuscript reports original clinical trial data, NCT02307500. Patient characteristic and efficacy toxicity data are available along with the study protocol. The data are available now. Proposals to access data should be directed to the Corresponding Author after publication.

Financial & competing interests disclosure

Bayer SpA (Milan, Italy) provided the drug for the study and financial support. A Santoro reports personal and other fees from BMS, Servier, Gilead, Pfizer, Eisai, Bayer, MSD, Arqule, Takeda, Roche, Abbvie, Amgen, Celgene, AstraZeneca, Lilly, Sandoz and Novartis, outside the submitted work. L Rimassa reports personal fees from Lilly, Bayer, Syrtex Medical, ArQule, Exelixis, Ipsen, Celgene, Eisai, AstraZeneca, AbbVie, Gilead and Roche, outside the submitted work. N Personeni reports personal fees from AbbVie and Gilead, and other fees from ArQule, outside the submitted work. S Bozzarelli reports the participation to an advisory board sponsored by Celgene, outside the submitted work. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Editorial assistance was provided by L Giacomelli, S Di Nunzio and A Shah (Polistudium srl), funded internally.

Ethical conduct of research

This investigator-sponsored trial (IST) was approved by the local Ethics Committee of Humanitas Clinical and Research Center – IRCCS and was conducted in accordance with the Declaration of Helsinki. A written informed consent was obtained from every patient at study inclusion. This study is registered at ClinicalTrials.gov NCT02307500.

Additional information

Funding

Bayer SpA (Milan, Italy) provided the drug for the study and financial support. A Santoro reports personal and other fees from BMS, Servier, Gilead, Pfizer, Eisai, Bayer, MSD, Arqule, Takeda, Roche, Abbvie, Amgen, Celgene, AstraZeneca, Lilly, Sandoz and Novartis, outside the submitted work. L Rimassa reports personal fees from Lilly, Bayer, Syrtex Medical, ArQule, Exelixis, Ipsen, Celgene, Eisai, AstraZeneca, AbbVie, Gilead and Roche, outside the submitted work. N Personeni reports personal fees from AbbVie and Gilead, and other fees from ArQule, outside the submitted work. S Bozzarelli reports the participation to an advisory board sponsored by Celgene, outside the submitted work. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. Editorial assistance was provided by L Giacomelli, S Di Nunzio and A Shah (Polistudium srl), funded internally.