Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta
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...
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BMJ Publishing Group
2023-11-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/13/11/e078164.full |
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| author | Jennifer Walsh Catriona Keerie Stuart H Ralston Christopher J Weir Muhammad K Javaid WAYNE LAM Bente L Langdahl Jannie D Hald Patricia Osborne |
| author_facet | Jennifer Walsh Catriona Keerie Stuart H Ralston Christopher J Weir Muhammad K Javaid WAYNE LAM Bente L Langdahl Jannie D Hald Patricia Osborne |
| author_sort | Jennifer Walsh |
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| description | 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.Trial registration number ISRCTN15313991. |
| format | Article |
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| institution | Kabale University |
| issn | 2044-6055 |
| language | English |
| publishDate | 2023-11-01 |
| publisher | BMJ Publishing Group |
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| series | BMJ Open |
| spelling | doaj-art-a03a4baaf65c4ff8a88d7beb27b7d67e2025-02-11T13:35:14ZengBMJ Publishing GroupBMJ Open2044-60552023-11-01131110.1136/bmjopen-2023-078164Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfectaJennifer Walsh0Catriona Keerie1Stuart H Ralston2Christopher J Weir3Muhammad K Javaid4WAYNE LAM5Bente L Langdahl6Jannie D Hald7Patricia Osborne8Metabolic Bone Centre, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UKUniversity of Edinburgh College of Medicine and Veterinary Medicine, Edinburgh, UKCentre for Genomic and Experimental Medicine, University of Edinburgh Western General Hospital, Edinburgh, UK4 Edinburgh Clinical Trials Unit, The University of Edinburgh, Usher Institute of Population Health Sciences and Informatics, Edinburgh, UKOxford NIHR Musculoskeletal Biomedical Research Unit, University of Oxford, Oxford, UKCentre for Genomic and Experimental Medicine, University of Edinburgh Western General Hospital, Edinburgh, UKDepartment of Endocrinology and Internal Medicine, Aarhus Universitet, Aarhus, DenmarkDepartment of Endocrinology and Internal Medicine, Aarhus Universitet, Aarhus, DenmarkBrittle Bone Society, Dundee, UKIntroduction 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.Trial registration number ISRCTN15313991.https://bmjopen.bmj.com/content/13/11/e078164.full |
| spellingShingle | Jennifer Walsh Catriona Keerie Stuart H Ralston Christopher J Weir Muhammad K Javaid WAYNE LAM Bente L Langdahl Jannie D Hald Patricia Osborne Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta BMJ Open |
| title | Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta |
| title_full | Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta |
| title_fullStr | Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta |
| title_full_unstemmed | Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta |
| title_short | Protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta |
| title_sort | protocol of a randomised trial of teriparatide followed by zoledronic acid to reduce fracture risk in adults with osteogenesis imperfecta |
| url | https://bmjopen.bmj.com/content/13/11/e078164.full |
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