Denosumab for the Treatment of Osteoporosis in Children: A Pilot Study
Condition: Osteoporosis
MeSH terms: Osteoporosis
Study type: Interventional
Phase: Phase 2

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.
    

Detailed 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.
    

Intervention name(s): Zoledronic Acid; Denosumab
Intervention type(s): Drug; Drug
Intervention description(s): Intravenous zoledronic acid 0.025mg/kg at baseline, 6 months, 12 months and 18 months; Subcutaneous denosumab 1.0mg/kg at baseline, 6 months, 12 months and 18 months
Intervention MeSH terms: Zoledronic Acid; Denosumab
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Treatment