Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12616001084471
Ethics application status
Approved
Date submitted
3/08/2016
Date registered
11/08/2016
Date last updated
7/09/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
Characterization of patients with established polymyalgia rheumatica
Scientific title
Characterization of patients with established polymyalgia rheumatica treated
with long-term glucocorticoid therapy
Secondary ID [1] 289837 0
None
Universal Trial Number (UTN)
U1111-1186-0234
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polymyalgia rheumatica 299767 0
Condition category
Condition code
Inflammatory and Immune System 299701 299701 0 0
Autoimmune diseases
Musculoskeletal 299785 299785 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Patients with an established diagnosis of PMR attending the Austin Hospital or referred by primary care providers and Rheumatologists in the Hospital’s catchment area (north-eastern suburbs of Melbourne, Victoria) will be invited to partake. After appropriate consent, demographic data, a range of questionnaires on symptoms and prednisolone-related treatment complications, and a general physical examination will be carried out as part of the initial visit (performed in the Rheumatology Department, Heidelberg Repatriation Hospital. time commitment 2 hours). Participants will then have a set of blood tests performed, along with the following imaging tests:
- Bilateral hand x-rays (performed during initial visit);
- Ultrasound of the wrists and shoulders (performed during initial visit);
- Whole body Positron Emission Tomography (PET)/Computed Tomography (CT) scan (performed in Molecular Imaging and Therapy at Austin Hospital, time commitment 2 hours);
- Whole body composition DEXA scan (performed in Bone and Mineral Research Unit, time commitment 1 hour).

A subsequent visit will be arranged to the Centre for Physical Activity and Nutrition at Deakin University (time commitment 2 hours), Burwood Campus where participants will undergo a range of muscle strength, power and function tests in a dedicated laboratory. A peripheral quantitative CT scan (a test capable of measuring muscle size and bone strength) will also be performed during this visit.

In selected participants, targeted MRI may also be performed at a later date in order to permit correlation of whole body PET/CT findings (performed in the Radiology Department at Austin Hospital, time commitment 1 hour).

At the conclusion of the study (no longer than one month from enrolment to completion of subsequent visit), the characteristics of participants with established PMR will be compared with those from a prior study at Austin Health of patients with newly diagnosed disease. It is hypothesized that this information will further delineate a distinct subset of patients refractory to the “one size fits all” cortisone approach, thereby permitting further study of alternate therapy in this group. In addition, the complications of long-term prednisolone use in PMR will be better appreciated.
Intervention code [1] 295519 0
Not applicable
Comparator / control treatment
Patients with newly diagnosed PMR from a prior study at Austin Health (ACTRN12614000696695) will be the comparator group (data collected from June 2014 to June 2017).
Control group
Historical

Outcomes
Primary outcome [1] 299161 0
Disease activity, using PMR – Activity Score (PMR-AS):
- comprises patient visual analogue scale, physician visual analogue scale, C-reactive protein, duration of early morning stiffness and elevation of upper limb score.

Timepoint [1] 299161 0
Initial visit
Secondary outcome [1] 326362 0
Quality of life, using 36-Item Short Form Survey (SF36)
Timepoint [1] 326362 0
Initial visit
Secondary outcome [2] 326585 0
Quality of life, using Health Assessment Questionnaire – Disability Index (HAQ-DI)
Timepoint [2] 326585 0
Initial visit
Secondary outcome [3] 326586 0
Mood, using Hospital Anxiety and Depression Scale (HADS)
Timepoint [3] 326586 0
Initial visit
Secondary outcome [4] 326587 0
Sleep, using Pittsburgh Sleep Quality Index (PSQI)
Timepoint [4] 326587 0
Initial visit
Secondary outcome [5] 326588 0
Fatigue, using Bristol Rheumatoid Arthritis Fatigue Multidimensional Questionnaire (BRAF-MDQ)
Timepoint [5] 326588 0
Initial visit
Secondary outcome [6] 326589 0
Sarcopaenia, using SARC-F questionnaire
Timepoint [6] 326589 0
Subsequent visit (within 1 month of initial visit)
Secondary outcome [7] 326590 0
Physical activity, using Habitual physical activity questionnaire
Timepoint [7] 326590 0
Subsequent visit (within 1 month of initial visit)
Secondary outcome [8] 326591 0
Cytokine profile, using multiplex assay
Timepoint [8] 326591 0
Initial visit
Secondary outcome [9] 326592 0
Abnormalities on ultrasound of bilateral wrists and shoulders
Timepoint [9] 326592 0
Initial visit
Secondary outcome [10] 326593 0
Distribution, incidence and intensity of joint and vascular FDG uptake on whole body PET/CT
Timepoint [10] 326593 0
Initial visit
Secondary outcome [11] 326594 0
Lean, fat and bone mass on whole body composition DEXA scan
Timepoint [11] 326594 0
Initial visit
Secondary outcome [12] 326595 0
Muscle cross-sectional area, subcutaneous fat cross-sectional area, muscle density, cortical bone thickness, cortical bone density and bone strength strain index on peripheral quantitative CT scan of radius, femur and tibia
Timepoint [12] 326595 0
Subsequent visit (within 1 month of initial visit)
Secondary outcome [13] 326596 0
Muscle power, using upper and lower limb maximal muscle power and strength, bilateral maximal isometric grip strength and bilateral maximal isometric dorsi-flexion strength, using Keiser pneumatic resistance training equipment fitted with A420 electronics, hand-held dynamometer and dorsiflexion dynamometer
Timepoint [13] 326596 0
Subsequent visit (within 1 month of initial visit)
Secondary outcome [14] 326597 0
Physical activity, using Short Physical Performance Battery (SPPB)
Timepoint [14] 326597 0
Subsequent visit (within 1 month of initial visit)
Secondary outcome [15] 326598 0
Balance and coordination, using Choice Stepping Reaction Time (CSRT) and Four-Step Square Test (FSST)
Timepoint [15] 326598 0
Subsequent visit (within 1 month of initial visit)

Eligibility
Key inclusion criteria
Diagnosis of PMR (as defined by the 2012 European League Against Rheumatism/American College of Rheumatology [EULAR/ACR] Classification Criteria) at least 18 months earlier;
AND
Long-term treatment with low-dose glucocorticoid therapy (stable prednisolone dose greater than or equal to 5mg and less than or equal to 15mg oral daily for at least 2 months duration).
Minimum age
50 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Prior diagnosis or current symptoms suggestive of Giant Cell Arteritis (GCA [headache, jaw claudication, scalp tenderness or sudden visual disturbance]);
Active malignancy;
Active infection;
Active neuromuscular disease;
Other inflammatory conditions eg. Rheumatoid Arthritis (RA);
Chronic pain syndromes;
Treatment with concomitant Disease Modifying Anti-Rheumatic Drugs (DMARD).

Study design
Purpose
Natural history
Duration
Cross-sectional
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
At completion of the study period, statistical analyses will be undertaken using Stata 13.0 to compare the characteristics of those patients with established PMR with those from a newly diagnosed population from a prior study. Parametric data will be compared using t-tests, while non-parametric data will be compared using the chi-square test or Kruskall-Wallis one-way analysis of variance. 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.

To the author’s knowledge, no study has previously attempted to characterize the demographic, clinical, laboratory and radiologic features of patients with established PMR (as distinct from newly diagnosed PMR). In the absence of such pilot data, sample size calculation is difficult given the expected proportion of participants with a given outcome is unknown. With that said, the minimum sample size is anticipated to be similar to that enrolled in a prior study at Austin Health of newly diagnosed patients (~30 participants), in order to enable direct comparison of the two groups.

Recruitment
Recruitment status
Withdrawn
Reason for early stopping/withdrawal
Other reasons/comments
Other reasons
Significant change of study design
Date of first participant enrolment
Anticipated
15/08/2016
Actual
Date of last participant enrolment
Anticipated
31/12/2017
Actual
Date of last data collection
Anticipated
31/12/2017
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 6373 0
Austin Health - Austin Hospital - Heidelberg
Recruitment hospital [2] 6374 0
Austin Health - Heidelberg Repatriation Hospital - Heidelberg West
Recruitment postcode(s) [1] 13923 0
3084 - Heidelberg
Recruitment postcode(s) [2] 13924 0
3081 - Heidelberg West

Funding & Sponsors
Funding source category [1] 294217 0
Hospital
Name [1] 294217 0
Austin Health
Country [1] 294217 0
Australia
Funding source category [2] 294218 0
Charities/Societies/Foundations
Name [2] 294218 0
Arthritis Australia
Country [2] 294218 0
Australia
Primary sponsor type
Hospital
Name
Austin Health
Address
Rheumatology Department,
Repatriation Campus,
Level 1, North Wing,
300 Waterdale Road,
Heidelberg West VIC 3081.
Country
Australia
Secondary sponsor category [1] 293050 0
Charities/Societies/Foundations
Name [1] 293050 0
Arthritis Australia
Address [1] 293050 0
Level 2/255 Broadway,
Glebe NSW 2037.
Country [1] 293050 0
Australia
Other collaborator category [1] 279141 0
University
Name [1] 279141 0
Deakin University
Address [1] 279141 0
Centre for Physical Activity and Nutrition Research,
Burwood Campus,
Building J,
221 Burwood Highway,
Burwood VIC 3125.
Country [1] 279141 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 295626 0
Austin Health
Ethics committee address [1] 295626 0
145 Studley Road,
Heidelberg VIC 3084.
Ethics committee country [1] 295626 0
Australia
Date submitted for ethics approval [1] 295626 0
30/03/2016
Approval date [1] 295626 0
25/07/2016
Ethics approval number [1] 295626 0
HREC/16/Austin/77

Summary
Brief summary
Despite the fact that Polymyalgia Rheumatica (PMR) is the most common inflammatory rheumatic disease of the elderly, it is under-researched and poorly understood. With no diagnostic tests available, diagnosis is dependent upon a history of muscle pain and stiffness in the shoulder and hip 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 that lasts ~12 months in duration. 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. This project therefore aims to identify the characteristics of PMR patients that have failed standard cortisone treatment and remain on long-term prednisolone. In addition, prednisolone-related treatment complications will be documented.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 67990 0
A/Prof Russell Buchanan
Address 67990 0
Austin Health
Rheumatology Department,
Repatriation Campus,
Level 1, North Wing,
300 Waterdale Road,
Heidelberg West VIC 3081.
Country 67990 0
Australia
Phone 67990 0
+613 9496 4013
Fax 67990 0
+613 9496 4012
Email 67990 0
[email protected]
Contact person for public queries
Name 67991 0
Dr Jessica Leung
Address 67991 0
Austin Health
Rheumatology Department,
Repatriation Campus,
Level 1, North Wing,
300 Waterdale Road,
Heidelberg West VIC 3081.
Country 67991 0
Australia
Phone 67991 0
+613 9496 4013
Fax 67991 0
+613 9496 4012
Email 67991 0
[email protected]
Contact person for scientific queries
Name 67992 0
Dr Jessica Leung
Address 67992 0
Austin Health
Rheumatology Department,
Repatriation Campus,
Level 1, North Wing,
300 Waterdale Road,
Heidelberg West VIC 3081.
Country 67992 0
Australia
Phone 67992 0
+613 9496 4013
Fax 67992 0
+613 9496 4012
Email 67992 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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