A Phase 2 Trial of RANKL Antibody, Denosumab, in Two Cohorts of Patients with Recurrent/Refractory Osteosarcoma, a Report from the Children’s Oncology Group

Janeway KA, Chou AJ, Buxton A, Reid J, Isakoff MS, Kopp L, Hingorani P, Lee JL, Doski J, Daldrup-Link H, Hall L, Randall RL, Reed DR, Dubois SG, Krailo M, Grier H, Gorlick R. A Phase 2 Trial of RANKL Antibody, Denosumab, in Two Cohorts of Patients with Recurrent/Refractory Osteosarcoma, a Report from the Children’s Oncology Group. Clin Cancer Res. 2026 Jan 6;32(1):36-44. doi: 10.1158/1078-0432.CCR-24-2885. PMID: 41159913; PMCID: PMC12908652.

Mouse models demonstrate a role for RANK and its ligand, RANKL, in osteosarcoma. The primary objective of this single arm open label phase 2 trial was to determine if denosumab, a RANKL monoclonal antibody, improved disease control in recurrent osteosarcoma relative to benchmarks derived from historical COG clinical trial data.