Edinburgh Clinical Trials

ZiPP-LTE

Zoledronate In the Prevention of Paget’s Disease: Long Term Extension

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.

 

Chief Investigator: Prof Stuart Ralston

Number and location of participating sites / geographical scope (by region/ country: Total 20 sites.  UK 7 sites, Australia 5 sites, Italy 3 sites, Spain 2 sites, New Zealand 2 sites, Ireland 1 site, Belgium 1 site.

 

ISRCTN number: Clinicaltrials.gov - NCT03859895

 

Funder: European Research Council and University of Edinburgh

Start and End date

Of grant award: 08/2018 – 07/2023

Of recruitment

 

Current Status: Currently recruiting

 

If you have participated in this study and would like to read how the study complies with GDPR, please click on this link:

 

Sponsor:  ACCORD, QMRI, 47 Little France Crescent, Edinburgh, EH16 4TJ, 0131 242 3326, resgov@accord.scot

Chief Investigator: Prof Stuart Ralston, Arthritis Research UK Professor of Rheumatology, Centre for Genomic and Experimental Medicine, IGC, University of Edinburgh, Western General Hospital, Edinburgh, EH4 2XU, 0131-651-8743  (Direct) / 0131-651-8741 (administrator), Stuart.Ralston@ed.ac.uk

Trial Management:  Institute of Genetics and Cancer,  University of Edinburgh, Western General Hospital, Edinburgh EH4 2XU Jonathan.Phillips@ed.ac.uk or ZIPP-LTE@ed.ac.uk

  

ECTU Involvement: Statistics