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
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