Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12614000696695
Ethics application status
Approved
Date submitted
25/06/2014
Date registered
2/07/2014
Date last updated
6/09/2022
Date data sharing statement initially provided
6/09/2022
Date results information initially provided
6/09/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Predictors of relapse in Polymyalgia Rheumatica patients treated with low-dose glucocorticoid therapy
Scientific title
Predictors of relapse in Polymyalgia Rheumatica patients treated with low-dose glucocorticoid therapy
Secondary ID [1] 284862 0
None
Universal Trial Number (UTN)
U1111-1158-3532
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polymyalgia Rheumatica 292262 0
Condition category
Condition code
Inflammatory and Immune System 292617 292617 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
This study will prospectively evaluate patients with a new diagnosis of Polymyalgia Rheumatica (PMR) as they are treated with a standard weaning course of Prednisolone (duration 46 weeks). The demographic, clinical, laboratory and radiologic characteristics (on musculoskeletal ultrasound and whole body PET/CT scan [in selected cases]) of patients whose disease relapses (defined by the PMR-Activity Score) will be compared with those in sustained disease remission. Identification of this "refractory" subset of patients will permit future treatment to be tailored to individual risk of disease relapse and prevent complications from unnecessarily prolonged glucocorticoid therapy.
Intervention code [1] 289667 0
Not applicable
Comparator / control treatment
No comparator
Control group
Uncontrolled

Outcomes
Primary outcome [1] 292457 0
Disease relapse as defined by the PMR-Activity Score (>9.35 or change in PMR-AS >6.6)
Timepoint [1] 292457 0
At week 4, week 16, week 32 and week 46
Secondary outcome [1] 308993 0
Non-response (PMR-AS >9.35) to Prednisolone 15mg daily
Timepoint [1] 308993 0
At week 4
Secondary outcome [2] 308994 0
Prednisolone dose >5mg daily
Timepoint [2] 308994 0
At week 46
Secondary outcome [3] 308995 0
Evolution of abnormalities (bursitis, tenosynovitis, synovitis and joint effusions) on musculoskeletal ultrasound with treatment and in clinical remission
Timepoint [3] 308995 0
At week 0, week 4, week 16 and week 46

Eligibility
Key inclusion criteria
New diagnosis of Polymyalgia Rheumatica as defined by the 2012 EULAR/ACR Classification Criteria:
- Age >= 50 years;
AND
- Bilateral shoulder aching;
AND
- Abnormal ESR and/or CRP;
PLUS a score of >=4 based upon a scoring algorithm:
- Morning stiffness duration >45 mins (2 points);
- Hip pain or limited range of movement (1 point);
- Negative rheumatoid factor and/or ACPA (2 points);
- Absence of peripheral joint pain (1 point).
Minimum age
50 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Inability to provide informed consent;
- Symptoms suggestive of Giant Cell Arteritis (headache, jaw claudication, scalp tenderness or visual disturbance);
- Cancer within the past 5 years;
- Neuromuscular disease;
- Active infection;
- Other inflammatory conditions eg. RA;
- Chronic pain syndromes;
- Uncontrolled psychiatric conditions, hypertension or diabetes;
- Treatment with glucocorticoids for >7 days prior to screening or a dose >15mg/day.
- Treatment with concomitant Disease Modifying Anti-Rheumatic Drugs.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
At study completion, statistical analyses will be undertaken using SPSS 20.0 to compare those PMR patients who relapsed with those who remained in remission with standardized low-dose glucocorticoid therapy. Parametric data will be compared using t-tests and one-way ANOVA, while non-parametric data will be compared using the chi-square test or Kruskall-Wallis. P-values of < 0.05 will be classified as statistically significant. A more detailed multivariable and conditional logistic regression is also planned to control for the effects of variables such as gender, BMI and smoking status.

Due to a lack of literature indicating anticipated effect size in a pilot study such as this, sample size calculation is difficult. That said, a similar study design by Cimmino et al. (2011) did achieve a statistically significant result with enrolment of 60 patients.

Cimmino, M. et al. (2011). ‘The correct Prednisolone starting dose in polymyalgia rheumatica is related to body weight but not disease severity’, BMC Musculoskeletal Disorders; 12:94.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
14/07/2014
Actual
14/07/2014
Date of last participant enrolment
Anticipated
8/02/2016
Actual
9/05/2017
Date of last data collection
Anticipated
Actual
29/03/2018
Sample size
Target
60
Accrual to date
Final
35
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 2667 0
Austin Health - Austin Hospital - Heidelberg
Recruitment postcode(s) [1] 8338 0
3084 - Heidelberg

Funding & Sponsors
Funding source category [1] 289486 0
Hospital
Name [1] 289486 0
Austin Hospital
Country [1] 289486 0
Australia
Primary sponsor type
Hospital
Name
Austin Health
Address
Rheumatology Department
300 Waterdale Road,
Heidelberg West VIC 3081
Country
Australia
Secondary sponsor category [1] 288170 0
Charities/Societies/Foundations
Name [1] 288170 0
Arthritis Australia
Address [1] 288170 0
Level 2/255 Broadway,
Glebe NSW 2037
Country [1] 288170 0
Australia
Secondary sponsor category [2] 288171 0
Charities/Societies/Foundations
Name [2] 288171 0
Austin Medical Research Foundation
Address [2] 288171 0
Austin Hospital
145 Studley Road,
Heidelberg VIC 3084
Country [2] 288171 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 291242 0
Austin Health
Ethics committee address [1] 291242 0
145 Studley Road,
Heidelberg VIC 3084
Ethics committee country [1] 291242 0
Australia
Date submitted for ethics approval [1] 291242 0
Approval date [1] 291242 0
04/06/2014
Ethics approval number [1] 291242 0
HREC/14/Austin/158

Summary
Brief summary
Despite the fact that Polymyalgia Rheumatica (PMR) is the most common inflammatory rheumatic disease of the elderly, it is poorly understood. With no diagnostic tests available, diagnosis is dependent upon a history of muscle pain and stiffness in the hip and shoulder regions, combined with raised inflammation levels in the blood. Treatment consists of Prednisolone (commonly referred to as “cortisone”) prescribed in a “one size fits all” approach. However, the way in which PMR patients’ symptoms respond is very variable; some improve almost overnight, whilst other individuals require higher doses for much longer periods of time. Unfortunately, such long-term Prednisolone use can result in many complications including osteoporosis, weight gain, high blood pressure and diabetes. Similarly, uncontrolled PMR is associated with increased risk of heart attacks and stroke. This project aims to identify the characteristics of patients that fail to respond adequately to Prednisolone treatment. It is hypothesized that this information will delineate a distinct subset of “refractory” PMR patients, thereby permitting further study of alternate therapy in this group and minimizing the side effects of Prednisolone use long-term.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 49438 0
Dr Claire Owen
Address 49438 0
Austin Health
Rheumatology Department
300 Waterdale Road,
Heidelberg West VIC 3081
Country 49438 0
Australia
Phone 49438 0
+61 3 9496 4013
Fax 49438 0
+ 61 3 9496 4012
Email 49438 0
claire.owen@austin.org.au
Contact person for public queries
Name 49439 0
Dr Claire Owen
Address 49439 0
Austin Health
Rheumatology Department
300 Waterdale Road,
Heidelberg West VIC 3081
Country 49439 0
Australia
Phone 49439 0
+61 3 9496 4013
Fax 49439 0
+ 61 3 9496 4012
Email 49439 0
claire.owen@austin.org.au
Contact person for scientific queries
Name 49440 0
Dr Claire Owen
Address 49440 0
Austin Health
Rheumatology Department
300 Waterdale Road,
Heidelberg West VIC 3081
Country 49440 0
Australia
Phone 49440 0
+61 3 9496 4013
Fax 49440 0
+ 61 3 9496 4012
Email 49440 0
claire.owen@austin.org.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
TypeIs Peer Reviewed?DOICitations or Other DetailsAttachment
Study results articleYes Owen CE, McMaster C, Liew DFL, Leung JL, Scott AM,... [More Details]
Study results articleYes Owen CE, Poon AMT, Yang V, McMaster C, Lee ST, Lie... [More Details]
Study results articleYes Owen CE, Poon AMT, Lee ST, Yap LP, Zwar RB, McMena... [More Details]

Documents added automatically
No additional documents have been identified.