Denosumab for the Treatment of Osteoporosis in Children: A Pilot Study

Osteoporosis Clinical Trial

Official title: A Single-Blinded, Randomized, Controlled, Phase 2 Pilot Study to Evaluate the Safety and Efficacy of Denosumab Compared to Zoledronic Acid for the Treatment of Osteoporosis in Children

Status Completed

Clinical Trial Summary

The aim of this study is to acquire preliminary, pilot data over a 2-year period on the safety and efficacy of subcutaneous denosumab versus the current CHEO standard-of-care (intravenous zoledronic acid) for the treatment of osteoporosis in children. Both denosumab (1.0mg/kg) and zoledronic acid (0.025mg/kg) will be given as four doses separated by a six month interval (i.e. at baseline, 6 months, 12 months and 18 months), with follow-up to 2 years.

Clinical Trial Description

Bone fractures without significant trauma (also known as osteoporosis) are a frequent complication of chronic childhood diseases (for example, Duchenne muscular dystrophy and inflammatory disorders) and genetic disorders (such as osteogenesis imperfecta). To date, the only effective therapy for the treatment of osteoporosis in children is a class of medications known as bisphosphonates (which include pamidronate and zoledronic acid). Unfortunately, these medications have the disadvantage of frequent side effects with the first dose (including fever, nausea, vomiting and bone pain), as well as intravenous administration (delivery through a vein, similar to a bloodtest). Recently, a new medication has been proven effective in the treatment of adults with osteoporosis - this medication is called denosumab, and it has a distinct advantage over intravenous bisphosphonates since it is administered sub-cutaneously (through a small needle injected into the skin), plus an excellent safety profile. To date, denosumab has been used with success in a few children with rare forms of osteoporosis and other bone disorders. The purpose of this study is to further test the safety and benefits of denosumab in children with fractures due to osteoporosis, by comparing this new agent to the current standard treatment at CHEO, zoledronic acid. This will be a one year, pilot study in 20 children with a variety of forms of osteoporosis. Children enrolled in the study will be randomly assigned to receive zoledronic acid or denosumab every 6 months (for two doses), with tests carried out every few months over the course of the year to study the effects of these therapies on fractures, bone density and various safety parameters as measured by bloodtests. Overall, the results of this study will provide us with important information on the potential for denosumab to provide children with a safe, effective and convenient new osteoporosis treatment. ;

Related Conditions & MeSH terms

• Osteoporosis

• NCT number: NCT02632916
• Study type: Interventional
• Source: Children's Hospital of Eastern Ontario
• Status: Completed
• Phase: Phase 2
• Start date: August 2016
• Completion date: February 3, 2020