REGOSTA: A randomized, placebo-controlled, double-blinded, multicenter study evaluating the efficacy and safety of regorafenib (REGO) as maintenance therapy after first-line treatment in patients (pts) with osteosarcoma (OS) and non-osteosarcomas (non-OS) of bone (non-Ewing, non-chondrosarcomas and non-chordomas).

TPS11576 Background: Pts with OS and non-OS of bone are treated with a multimodal sequence therapy of neoadjuvant chemotherapy (CT), surgery and adjuvant CT, followed by a close surveillance until recurrence. At recurrence, the prognosis remains poor with objective response rates of 3-29%, and a median Progression-Free Survival (PFS) of less than 4 months in OS. There is a clinical need to reduce the risk of recurrence after the initial treatment sequence. The REGOBONE study reported a significant clinical benefit of regorafenib compared to placebo in patients with relapsed OS (median PFS: 16.4 versus 4.1 weeks). Methods: This multicenter trial is ongoing to study the efficacy and safety of maintenance REGO in pts > = 16 years, with complete remission after initial treatment sequence of their bone sarcoma. 168 pts will be randomly allocated in a 1:1 ratio to receive either oral REGO or its matching placebo (control arm) at a daily dose of 120mg, continuously and for a maximum of 12 months. Randomization will be stratified according to the following risk factors: metastases (mets) at diagnosis and/or poor response to neoadjuvant CT versus no mets at diagnosis and good response to neoadjuvant CT. The primary objective is to compare the efficacy (Relapse-Free Survival) between the 2 arms. The expected 3-year RFS rates are 55% in the control arm and 74.6% in the REGO arm (HR = 0.5). 66 events will provide 80% power to show significant improvement in RFS, using a 2-sided log-rank test at a 5% level. Secondary endpoints include Time to Treatment Failure, Overall Survival, Quality of Life, safety profile, and compliance to treatment. Radiological endpoints will be evaluated using the RECIST 1.1. Translational objectives will be to identify predictive biomarkers for efficacy of REGO as maintenance therapy using liquid biopsies. As of Feb 1st, 2021, 3 patients have been randomized. 15 sites of the French Sarcoma Group will participate. Clinical trial information: NCT04055220.