biostudies-literatureJaneway KANational Institutes of Health (NIH)National Cancer InstituteNCI NIH HHSNational Institutes of Health36-44https://www.ebi.ac.uk/biostudies/studies/S-EPMC12908652biostudies-literatureUnknown32(1)<h4>Purpose</h4>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 whether denosumab, a RANKL mAb, improved disease control in recurrent osteosarcoma relative to benchmarks derived from historic Children's Oncology Group clinical trial data.<h4>Patients and methods</h4>Skeletally mature patients ages 11 to 49 years old with measurable disease were eligible for cohort 1, and those with complete surgical resection of all sites of disease were eligible for cohort 2. Patients received denosumab 120 mg subcutaneously every 4 weeks with calcium and vitamin D supplementation. The primary endpoints were RECIST response and remaining event-free for 4 months for cohort 1 and remaining evClinical cancer research : an official journal of the American Association for Cancer ResearchA Phase 2 Trial of RANKL Antibody, Denosumab, in Two Cohorts of Patients with Recurrent/Refractory Osteosarcoma, a Report from the Children's Oncology Group.PMC12908652U10CA180886U10 CA180886P30 CA015083Krailo MHall LGorlick RDoski JReed DRGrier HJaneway KADubois SGBuxton ADaldrup-Link HRandall RLChou AJReid JHingorani PIsakoff MSLee JLKopp LfalseA Phase 2 Trial of RANKL Antibody, Denosumab, in Two Cohorts of Patients with Recurrent/Refractory Osteosarcoma, a Report from the Children's Oncology Group.<h4>Purpose</h4>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 whether denosumab, a RANKL mAb, improved disease control in recurrent osteosarcoma relative to benchmarks derived from historic Children's Oncology Group clinical trial data.<h4>Patients and methods</h4>Skeletally mature patients ages 11 to 49 years old with measurable disease were eligible for cohort 1, and those with complete surgical resection of all sites of disease were eligible for cohort 2. Patients received denosumab 120 mg subcutaneously every 4 weeks with calcium and vitamin D supplementation. The primary endpoints were RECIST response and remaining event-free for 4 months for cohort 1 and remaining ev2026-01-01T00:00:00Z2026 Jan2026-07-16T05:09:57.552Z2026-07-10T03:09:30.327ZS-EPMC129086524115991310.1158/1078-0432.CCR-24-288510.1158/1078-0432.ccr-24-2885