Zoledronate in the Prevention of Paget's - Long term Extension
Research type
Research Study
Full title
Zoledronate in the Prevention of Paget's - Long Term Extension
IRAS ID
245197
Contact name
Stuart Ralston
Contact email
Sponsor organisation
University of Edinburgh
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Paget's disease of the bone (PDB) is a metabolic bone disorder which in some individuals can cause pain, bone deformity, arthritis and deafness, although in many patients it does not cause symptoms. Paget's disease has a strong genetic component and SQSTM1 is the most important susceptibility gene. People who inherit mutations in SQSTM1 have a high risk of developing PDB later in life. This study is an extension of the ZiPP study which was is a randomised trial currently in progress to determine if the bisphosphonate zoledronic acid (ZA) can prevent or delay the development of PDB-like bone lesions compared with a dummy treatment (placebo) in people who inherit SQSMT1 gene mutations. Although the ZiPP study will provide information on whether early ZA treatment can favourably influence bone lesion development the significance of this to the patient in terms of symptoms is unclear as yet. The aim of the extension study is to keep these individuals under surveillance for any symptoms or signs of PDB over a further 5 year period and to evaluate if there has been any progression of PDB-like lesions by bone scan at the end of this period. Since participants in the ZIPP study are at increased risk of developing PDB treatment for PDB will be offered to subjects that develop symptoms of PDB during follow up. Participants who previously received ZA in the core study will be offered further ZA treatment if they develop signs of PDB even in the absence of symptoms. Both strategies are commonly used in the treatment of early PDB in routine clinical practice The extension will provide valuable information on the natural history of PDB in people who carry SQSTM1 mutations and the effects of treatment on evolution of PDB.
REC name
East of Scotland Research Ethics Service REC 2
REC reference
18/ES/0086
Date of REC Opinion
11 Jul 2018
REC opinion
Favourable Opinion