Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta

Jannie D Hald, Catriona Keerie, Christopher J Weir, M Kassim Javaid, Wayne Lam, Patricia Osborne, Jennifer Walsh, Bente Lomholt Langdahl, Stuart H Ralston*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract / Description of output

INTRODUCTION: Osteogenesis imperfecta (OI) is a rare genetic disease associated with multiple fractures throughout life. It is often treated with osteoporosis medications but their effectiveness at preventing fractures is unknown. The Treatment of Osteogenesis Imperfecta with Parathyroid Hormone and Zoledronic Acid trial will determine if therapy with teriparatide (TPTD) followed by zoledronic acid (ZA) can reduce the risk of clinical fractures in OI.

METHODS AND ANALYSIS: Individuals aged ≥18 years with a clinical diagnosis of OI are eligible to take part. At baseline, participants will undergo a spine X-ray, and have bone mineral density (BMD) measured by dual-energy X-ray absorptiometry (DXA) at the spine and hip. Information on previous fractures and previous bone targeted treatments will be collected. Questionnaires will be completed to assess pain and other aspects of health-related quality of life (HRQoL). Participants will be randomised to receive a 2-year course of TPTD injections 20 µg daily followed by a single intravenous infusion of 5 mg ZA, or to receive standard care, which will exclude the use of bone anabolic drugs. Participants will be followed up annually, have a repeat DXA at 2 years and at the end of study. Spine X-rays will be repeated at the end of study. The duration of follow-up will range between 2 and 8 years. The primary endpoint will be new clinical fractures confirmed by X-ray or other imaging. Secondary endpoints will include participant reported fractures, BMD and changes in pain and HRQoL.

ETHICS AND DISSEMINATION: The study received ethical approval in December 2016. Following completion of the trial, a manuscript will be submitted to a peer-reviewed journal. The results will inform clinical practice by determining if TPTD/ZA can reduce the risk of fractures in OI compared with standard care.


Original languageEnglish
Article numbere078164
Pages (from-to)e078164
Number of pages9
JournalBMJ Open
Issue number11
Publication statusPublished - 22 Nov 2023

Keywords / Materials (for Non-textual outputs)

  • Adolescent
  • Adult
  • Bone Density
  • Bone Density Conservation Agents/therapeutic use
  • Fractures, Bone/prevention & control
  • Humans
  • Osteogenesis Imperfecta/complications
  • Pain/drug therapy
  • Quality of Life
  • Randomized Controlled Trials as Topic
  • Teriparatide/therapeutic use
  • Zoledronic Acid/therapeutic use


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