Osteogenesis imperfecta / Posted 4 years ago

TOPaZ: Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic acid 

Osteogenesis imperfecta (OI) is an inherited skeletal disorder characterised by increased risk of fragility fractures. Bisphosphonates are frequently prescribed for adult patients with OI with the aim of preventing fractures but the evidence base for efficacy is poor. Recent evidence suggests that the bone anabolic agent teriparatide (TPTD) increases bone mineral density (BMD) and may have the potential to prevent fractures in OI.

The purpose of the TOPaZ Trial is to investigate if a a two-year course of teriparatide (TPTD) followed by antiresorptive therapy with a single infusion of zoledronic acid (ZA) in adults with OI reduces the proportion of patients who experience a fracture as compared with standard care.

Adult patients with a clinical diagnosis of OI who are willing and able to give informed consent and who do not have contraindications to the study medications will be recruited from participating sites. Participants will be randomised 1:1 to receive either standard care for the duration of the trial or TPTD for 24 months followed by a single infusion of ZA, or another antiresorptive agent in the event that ZA is contraindicated.

Participants will attend recruiting centres for a Baseline/Screening visit, at 12 months, 24 months and at the end of the trial for formal study visits with telephone calls every 6 months from a site research nurse. Participants randomised to TPTD will also attend recruiting centre at regular intervals during the 24 month treatment period to collect new supplies of TPTD.

  • Inclusion Criteria : Adult patients age 18 years and above with a clinical diagnosis of OI Patients willing and able to consent and comply with the study protocol.
  • Exclusion Criteria : Contraindication to TPTD or ZA Unable or unwilling to provide informed consent Age less than 18 years Current or previous treatment with an investigational drug with effects on bone metabolism Women that are breastfeeding Women of childbearing potential not using adequate contraception(see below) Pregnancy Women of childbearing potential (WOCBP) can be enrolled into the study but will be required to use adequate contraception before and during the trial if they are being treated with TPTD or bisphosphonates, Examples of adequate contraception include: Established use of oral, injected or implanted hormonal methods of contraception. Placement of an intrauterine device (IUD) or intrauterine system. Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository. True abstinence. Periodic abstinence such as calendar, ovulation, symptothermal, post-ovulation methods and withdrawal are not acceptable methods of contraception.
  • Study start date : 22/05/2017
  • Study end date : 15/04/2023
  • Wales-Based Study Contact : Dr Kirsty Maclean
  • Principal Investigator : Prof Stuart Ralston

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