Phase 2 Study of Regorafenib in Combination with Immune Checkpoint Inhibitor Nivolumab in Patients with Refractory or Recurrent Osteosarcoma

Pediatric Sarcoma
Fariba Navid, MD
Children's Hospital Los Angeles


Osteosarcoma is the most common malignant bone tumor in children and young adults, with a peak incidence between 13 and 16 years of age. The mainstay of treatment is intensive multi-agent cytotoxic chemotherapy but despite these aggressive measures, 40% of patients with localized disease and 70% with metastatic disease will ultimately succumb to their illness. Intensification of active drugs or adding additional cytotoxic chemotherapy agents has not improved patient outcomes over the last 3 decades. Thus, there is an urgent need for new and innovative therapies for this disease. Vascular endothelial growth factor (VEGF) are signal proteins that play a key role in tumor angiogenesis (creation of blood supply to cancer cells) and immune evasion. High levels of VEGF is correlated with worse outcomes. Regorafenib is a drug that targets VEGF and studies suggest that inhibition VEGF might enhance the antitumor activity of immune checkpoint inhibitors. Thus, this phase II study will determine the 4-month progression-free survival (PFS) rate (the length of time after starting treatment the cancer does not grow or spread) of patients with relapsed or refractory (resistant) Osteosarcoma who receive regorafenib in combination with nivolumab, an immune checkpoint inhibitor.

Trial Registration: Identifier: NCT04803877