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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03859895




Registration number
NCT03859895
Ethics application status
Date submitted
31/01/2019
Date registered
1/03/2019
Date last updated
19/04/2019

Titles & IDs
Public title
Zoledronate In the Prevention of Paget's Disease: Long Term Extension
Scientific title
Zoledronate In the Prevention of Paget's Disease: Long Term Extension
Secondary ID [1] 0 0
245197
Secondary ID [2] 0 0
AC18051
Universal Trial Number (UTN)
Trial acronym
ZiPP-LTE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paget Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Observational (without bone scan) - Former Observational Arm participants of the ZiPP trial.

Observational (with bone scan) - Former Interventional Arm participants of the ZiPP trial.

Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Primary Endpoint (former ZiPP interventional arm): The proportion of patients that develop PDB-like bone lesions - The proportion of patients in each of the randomisation groups that develop PDB-like bone lesions by the end of study assessed by radionuclide bone scan.
Timepoint [1] 0 0
5 year time-point
Primary outcome [2] 0 0
Primary Endpoint (former ZiPP observational arm): proportion of individuals that develop abnormalities suggestive of PDB - The primary endpoint will be to evaluate the proportion of individuals that develop biochemical or clinical abnormalities suggestive of PDB over a the 10-year duration of follow up.
Timepoint [2] 0 0
During follow-up period
Secondary outcome [1] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to the number of new bone lesions assessed by radionuclide bone scan.
Timepoint [1] 0 0
5 year time-point
Secondary outcome [2] 0 0
Secondary Endpoint (former ZiPP interventional arm): Evaluate differences between ZiPP treatment and placebo groups for change in bone lesion activity by semi-quantitative analysis of radionuclide bone scans (method described by Patel et al (1995)).
Timepoint [2] 0 0
5 year time-point
Secondary outcome [3] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to SF36 (36-Item Short Form Survey) scores.
Timepoint [3] 0 0
5 year time-point
Secondary outcome [4] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to HAQ (Health Assessment Questionnaire) scores.
Timepoint [4] 0 0
5 year time-point
Secondary outcome [5] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to EQ5D (EuroQol five dimension scale) scores.
Timepoint [5] 0 0
5 year time-point
Secondary outcome [6] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to IPAQ (International Physical Activity Questionnaire) scores.
Timepoint [6] 0 0
5 year time-point
Secondary outcome [7] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to BPI (Brief Pain Inventory Scale) scores.
Timepoint [7] 0 0
5 year time-point
Secondary outcome [8] 0 0
Secondary Endpoint (former ZiPP interventional arm): Differences between ZiPP trial treatment and placebo groups with regard to development of PDB-related skeletal events (PRSE). - Defined as new bone lesions thought to be due to PDB on imaging OR complications of PDB such as pathological fractures, bone deformity, deafness, and joint replacement surgery OR administration of treatment for PDB because of pain localised to an affected site in a patient with metabolically active disease.
Timepoint [8] 0 0
5 year time-point
Secondary outcome [9] 0 0
Secondary Endpoint (former ZiPP observational arm): Health-related quality of life in ZiPP trial observational arm participants assessed using EQ5D.
Timepoint [9] 0 0
5 year time-point
Secondary outcome [10] 0 0
Secondary Endpoint (former ZiPP observational arm): Health-related quality of life in ZiPP trial observational arm participants assessed using SF36.
Timepoint [10] 0 0
5 year time-point
Secondary outcome [11] 0 0
Secondary Endpoint (former ZiPP observational arm): Health-related quality of life in ZiPP trial observational arm participants assessed using BPI.
Timepoint [11] 0 0
5 year time-point
Secondary outcome [12] 0 0
Secondary Endpoint (former ZiPP observational arm): Health-related quality of life in ZiPP trial observational arm participants assessed using HAQ.
Timepoint [12] 0 0
5 year time-point
Secondary outcome [13] 0 0
Secondary Endpoint (former ZiPP observational arm): Health-related quality of life in ZiPP trial observational arm participants assessed using IPAQ.
Timepoint [13] 0 0
5 year time-point
Secondary outcome [14] 0 0
Secondary Endpoint (former ZiPP observational arm): Pain in ZiPP trial observational arm participants assessed using EQ5D.
Timepoint [14] 0 0
5 year time-point
Secondary outcome [15] 0 0
Secondary Endpoint (former ZiPP observational arm): Pain in ZiPP trial observational arm participants assessed using SF36.
Timepoint [15] 0 0
5 year time-point
Secondary outcome [16] 0 0
Secondary Endpoint (former ZiPP observational arm): Pain in ZiPP trial observational arm participants assessed using BPI.
Timepoint [16] 0 0
5 year time-point
Secondary outcome [17] 0 0
Secondary Endpoint (former ZiPP observational arm): Anxiety in ZiPP trial observational arm participants assessed using EQ5D.
Timepoint [17] 0 0
5 year time-point
Secondary outcome [18] 0 0
Secondary Endpoint (former ZiPP observational arm): Anxiety in ZiPP trial observational arm participants assessed using SF36.
Timepoint [18] 0 0
5 year time-point
Secondary outcome [19] 0 0
Secondary Endpoint (former ZiPP observational arm): Depression in ZiPP trial observational arm participants assessed using SF-36.
Timepoint [19] 0 0
5 year time-point
Secondary outcome [20] 0 0
Secondary Endpoint (former ZiPP observational arm): Depression in ZiPP trial observational arm participants assessed using BPI.
Timepoint [20] 0 0
5 year time-point
Secondary outcome [21] 0 0
Secondary Endpoint (former ZiPP observational arm): Depression in ZiPP trial observational arm participants assessed using EQ5D.
Timepoint [21] 0 0
5 year time-point

Eligibility
Key inclusion criteria
- Subject that participated in ZiPP

- Participant willing and able to consent and comply with the study protocol.
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Unable or unwilling to provide informed consent

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
University Hospital Geelong - Geelong
Recruitment hospital [2] 0 0
Sir Charles Gardner Hospital - Nedlands
Recruitment hospital [3] 0 0
Royal Newcastle Centre - Newcastle
Recruitment hospital [4] 0 0
University of Sydney - Sydney
Recruitment hospital [5] 0 0
University of Queensland - Toowoomba
Recruitment postcode(s) [1] 0 0
3220 - Geelong
Recruitment postcode(s) [2] 0 0
6009 - Nedlands
Recruitment postcode(s) [3] 0 0
NSW 2305 - Newcastle
Recruitment postcode(s) [4] 0 0
- Sydney
Recruitment postcode(s) [5] 0 0
QLD 4350 - Toowoomba
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Ireland
State/province [2] 0 0
Dublin
Country [3] 0 0
Italy
State/province [3] 0 0
Florence
Country [4] 0 0
Italy
State/province [4] 0 0
Siena
Country [5] 0 0
Italy
State/province [5] 0 0
Turin
Country [6] 0 0
New Zealand
State/province [6] 0 0
Auckland
Country [7] 0 0
New Zealand
State/province [7] 0 0
Christchurch
Country [8] 0 0
Spain
State/province [8] 0 0
Barcelona
Country [9] 0 0
Spain
State/province [9] 0 0
Salamanca
Country [10] 0 0
United Kingdom
State/province [10] 0 0
England
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Scotland
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Wales

Funding & Sponsors
Primary sponsor type
Other
Name
University of Edinburgh
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
European Research Council
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief 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 (Zoledronate in the
Prevention of Paget's) study which was is 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.
Trial website
https://clinicaltrials.gov/show/NCT03859895
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Stuart Ralston, Prof
Address 0 0
Univeristy of Edinburgh
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Elaine Kinsella, PhD
Address 0 0
Country 0 0
Phone 0 0
01316519916
Fax 0 0
Email 0 0
ZIPP-LTE@ed.ac.uk
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03859895