Osteogenesis imperfecta Musculoskeletal Diseases Osteogenesis Imperfecta
Conditions
Interventions
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 fra
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
Sponsors
University of Edinburgh
Eligibility
Sex/Gender
All
Age
18 Years to 100 Years
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of participants experiencing a clinical fracture validated by x-ray or other imaging at the final study visit (between 36-60 months) | — |
Secondary
| Measure | Time frame |
|---|---|
| Current secondary outcome measures: 1. Total number of clinical fractures experienced by participants validated by x-ray or other imaging at the final study visit 2. Number of incident vertebral fractures assessed by imaging of the thoracic and lumbar spine at the final study visit 3. Total number of fractures experienced by participants defined as the combination of validated clinical fractures and vertebral fractures and fractures reported by participants, where imaging was not performed, not feasible or where the results were inconclusive at the final study visit 4. Bone pain is assessed by the brief pain inventory (BPI) at 12 months, 24 months and at the end of the study visit 5. Quality of life is assessed by the SF36 questionnaire at 12 months, 24 months and at the end of the study visit 6. Functional status is assessed by the health assessment questionnaire (HAQ) and EuroQol5D (EQ5D) assessment tools at 12 months, 24 months and at the end of the study visit 7. Adverse events reported by participants at 12 months, 24 months and at the end of the study visit Previous secondary outcome measures: 1. Total number of clinical fractures experienced by participants validated by x-ray or other imaging at the final study visit (between 36-60 months) 2. Number of incident vertebral fractures assessed by imaging of the thoracic and lumbar spine at the final study visit (between 36-60 months) 3. Total number of fractures experienced by participants defined as the combination validated clinical fractures and vertebral fractures and fractures reported by participants, where imaging was not performed, not feasible or where the results were inconclusive at the final study visit (between 36-60 months) 4. Bone pain is assessed by the brief pain inventory (BPI) at 12 months, 24 months and at the end of study visit (between 36-60 months) 5. Quality of life is assessed by the SF36 questionnaire at at 12 months, 24 months and at the end of study visit (between 36-60 months) 6. Fun | — |
Countries
Denmark, England, France, Ireland, Netherlands, Northern Ireland, Scotland, United Kingdom, Wales
Contacts
Public ContactLorna Dewar
Outcome results
None listed