TOPaZ study

  • Research type

    Research Study

  • Full title

    Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic acid

  • IRAS ID

    207747

  • Contact name

    Stuart Ralston

  • Contact email

    stuart.ralston@ed.ac.uk

  • Eudract number

    2016-003228-22

  • ISRCTN Number

    ISRCTN15313991

  • Duration of Study in the UK

    6 years, 5 months, 4 days

  • Research summary

    Osteogenesis Imperfecta (OI) is an inherited disorder known as "brittle bone disease" which is associated with a greatly increased risk of fractures. People with OI may suffer tens or hundreds of fractures during their lifetime and often become disabled as a result of this. There is no curative treatment for OI. Many doctors treat OI patients with drugs called bisphosphonates which are also used in osteoporosis, but it’s not clear if they are effective at preventing fractures. While clinical trials have been performed with other osteoporosis treatments in OI, no trials have been performed to determine if these treatments reduce the risk of fracture. The aim of the present study is to determine if a two year spell of treatment with a drug called teriparatide (TPTD) followed by treatment with another drug called zoledronic acid to maintain the effect of TPTD on the skeleton is effective at reducing the risk of fractures in adults with OI. These treatments will be compared with standard care which might consist of no active treatment or bisphosphonates which are prescribed to about 30% of patients with OI in the UK. This is first trial which has been designed to determine if drug treatment can reduce the risk of fractures in OI. If positive, it could be life-changing for those affected. If negative it would signal the need to investigate alternative approaches to reduce fracture burden in the rare but disabling condition.

  • REC name

    East of Scotland Research Ethics Service REC 2

  • REC reference

    16/ES/0110

  • Date of REC Opinion

    12 Sep 2016

  • REC opinion

    Favourable Opinion