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Prospective, Multicenter, Single-arm Study to Evaluate Efficacy, Safety and Pharmacokinetics of Denosumab in Children with Osteogenesis Imperfecta

Principal Investigator:
Jay
Shapiro

This is a prospective, multicenter single-arm study in children 2 to 17 years of age with osteogenesis imperfecta (OI). Approximately 150 subjects will be enrolled at 40 institutions including at least 30 subjects less than 7 years of age, and 5 at the KKI. All subjects will receive denosumab 1 mg/kg (up to a maximun of 60 mg) given subcutaneously every 6 months for 36 months. Enrollment is gated by age: 11-17 years as the first cohort, and 7-10 years as second cohort and the 2-6 year cohort therafter.

The primary endpoint is a change in baseline lumbar spine BMD Z-score as assessed by DXA over first 12 months. The secondary endpoints include lumbar and proximal femur DXA at 6, 18, 24 and 36 months, and occurrence of vertebral and non-vertebral fractures. Patient satisfaction parameters are also measured.

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