Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic acid
Osteogenesis imperfecta
Current interventions as of 14/07/2023: Participants are randomised to one of two groups in a 1:1 ratio using minimisation to balance the groups for the following prognostic variables: 1. Clinical fracture during the two years prior to randomisation 2. Clinical subtype of OI (type I or others) 3. Gender 4. Lowest BMD T score at spine or hip (or Z-score aged 18-21) ≤-2.5; or >-2.5. 5. Age (≤50 years; >50 years) 6. Bisphosphonate at entry or within 2 years prior to randomisation Intervention group: Participants will receive a 2-year course of teriparatide 20mcg daily by subcutaneous injection. At the end of this period, participants will be given a single intravenous infusion of zoledronic acid 5mg. Control group: Participants will receive standard care, which may involve no bone-specific treatment or treatment with bisphosphonates, depending on what the specialist that is normally responsible for treating participants’ osteogenesis imperfecta feels is most appropriate. Bone anabolic drugs such as teriparatide and romosozumab will be prohibited in the standard care group. In the active group, romosozumab will be prohibited. Investigational drugs will be prohibited in both groups. This is an event-driven study which will go on until 149 clinical fractures have occurred. Based on published data, this is expected to have occurred an average of 60 months after the patient has enrolled on the study. Participants are reviewed at 12 months, 24 months and again at the end of the study. This will on average be 60 months after enrollment but it may vary between 24 and 84 months. At each visit, the patient will get blood checked and complete questionnaires. At baseline, 24 months and the study end, a DEXA will be done. At baseline and the study end a spine-x-ray will be done. Previous interventions: Participants are randomised to one of two groups using minimisation to balance the groups for the following prognostic variables: 1. Clinical fracture during the two years p
1. Adult patients age 18 years and over with a clinical diagnosis of osteogenesis imperfecta 2. Patients willing and able to consent and comply with the study protocol
Current exclusion criteria as of 07/02/2017: 1. Current or previous treatment with an investigational (non-licensed) drug with effects on bone metabolism 2. Contraindication to TPTD or ZA 3. Women of childbearing potential not using highly effective methods of contraception 4. Pregnancy 5. Women that are breastfeeding 6. Age <18 years Previous exclusion criteria: 1. Contraindication to TPTD or ZA 2. Current or previous treatment with an investigational drug with effects on bone metabolism 3. Women of childbearing potential not using adequate contraception 4. Pregnancy