Safety and Efficiency of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis

Phase 3 Completed 24 enrolled Amgen

RandomizedParallel-groupQuadruple-blindTreatment

Summary

To evaluate the effect of denosumab on lumbar spine bone mineral density (BMD) Z-score as assessed by dual-energy X-ray absorptiometry (DXA) at 12 months in children 5 to 17 year of age with Glucocorticoid (GC)-induced osteoporosis (GiOP).

Timeline

Start: 2018-05-07
Primary completion: 2021-12-13
Completion: 2023-12-20

Outcome

Missed primary endpoint

registry analysis (superiority test); Denosumab/Denosumab vs Placebo/Denosumab; p = 0.68; Least Squares Mean Difference 0.11 (95% CI -0.450 to 0.673); ANCOVA NCT03164928 ↗

Publications

Storm NE, Kubik C, Priem G, Kim M, Lin TC, Lange J, O'Connell C, Glennane A, Bradbury BD, Taisey MJ. Case Study on the Use of Real-World Evidence in a Feasibility Assessment of a Randomized Controlled Trial Design to Support the Fulfillment of Pediatric Requirements with the FDA and EMA. Ther Innov Regul Sci. 2026 Jun 19. doi: 10.1007/s43441-026-00977-1. Online ahead of print.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Denosumab	Monoclonal antibody	1 mg/kg	Subcutaneous