Polymyalgia RheumaticaNCT05542316
Validation of the PMR Activity Score and Evolution of Patient-reported Outcomes in Patients With Polymyalgia Rheumatica
An observational study of Polymyalgia Rheumatica, run by Universitaire Ziekenhuizen KU Leuven at 1 site in Belgium.
Trial summary
Polymyalgia rheumatica (PMR) is a systemic inflammatory disease that affects elderly people. It is characterized by pain and morning stiffness in the shoulders, pelvic girdles and neck. Glucocorticoids are the mainstay of the treatment. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Polymyalgia Rheumatica
- How many people
- 133 participants
- Where
- 1 siteBelgium
- Study dates
- 1 Jul 2022to 9 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Diagnosis of PMR
Who may be able to join
- Diagnosis of PMR based on the judgment of an experienced clinician (affiliated with the general internal medicine or rheumatology department), taking into account all available information (clinical symptoms, biochemical, radiological, and PET results)
- Able to give informed consent
- Understanding and able to write Dutch, English or French
Who cannot join
- Concomitant diagnosis of giant cell arteritis (new diagnosis or being treated for giant cell arteritis)
- Concurrent rheumatoid arthritis, other inflammatory arthritis or other connective tissue disease
- Patients on glucocorticoids or other immunosuppressive drugs for another indication
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Validation of the Polymyalgia Rheumatica Activity Score for Determination of Disease Activity
Polymyalgia rheumatica (PMR) is a systemic inflammatory disease that affects elderly people. It is characterized by pain and morning stiffness in the shoulders, pelvic girdles and neck. Glucocorticoids are the mainstay of the treatment. In clinical practice, the disease activity of PMR and corresponding treatment changes are based on the presence of symptoms and inflammatory markers. The interpretation of these abnormalities can be surprisingly difficult, especially when they are not consistent. In 2004, Leeb and Bird developed a composite score for measurement of disease activity in PMR, called the polymyalgia rheumatica activity score. It consists of 5 domains: morning stiffness time, ability to elevate the upper limbs, physician's global assessment, pain and CRP level. However, high-quality evidence on the measurement properties is lacking and there is still no consensus on the optimal cut off point.
Based on a Delphi study with physicians and patients OMERACT defines laboratory markers of systemic inflammation, pain, stiffness and physical function as the four inner core of domains considered mandatory for clinical trials of PMR, most frequently measured by erythrocyte sedimentation rate (ESR) and/or C-reactive protein (CRP), visual analogue scale (VAS) for pain, morning stiffness time and Health Assessment Questionnaire-Disability Index (HAQ-DI) respectively. Patient's global fatigue was strongly recommended to measure in PMR as well. Recently, a PMR-specific patient-reported outcome measure was developed, called the PMR impact scale. However, outcome measures in PMR studies lack consistency and there is no high-quality evidence on the measurement properties. In addition, the evolution of these patient reported outcomes is not known.
Read the detailed description
The primary objective of this prospective observational trial is to validate the PMR-activity score and to determine the optimal cut-off point to discriminate between disease remission and active disease. The secondary objective is to assess the evolution of the patient reported outcomes during the disease course and their relation with disease activity.
The investigators intend to perform a 12-month, observational prospective trial in patients with recently diagnosed PMR. Patients will be followed and treated as standard of care. Follow-up visits will be planned at 8 weeks, 16 weeks, 26 weeks and 52 weeks. As in standard of care, additional visits can be planned if necessary, e.g. in case of symptoms suggestive of relapse. Each visit ESR, CRP, morning stiffness time, ability to elevate the upper limbs, physician's global assessment and VAS for pain as components of the PMR-activity score and VAS for global health, pain, stiffness and fatigue, HAQ-DI, Short Form Health survey (SF36) and PMR impact scale as patient-reported outcomes will be collected.
- Conditions
- Polymyalgia Rheumatica
- Medical subject headings
- Polymyalgia Rheumatica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with a final diagnosis of PMR | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of the sensitivity and specificity of the PMR-activity score and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Secondary outcomes (20)
Determination of the sensitivity and specificity of morning stiffness time and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of ability to elevate the upper limbs and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of physician's global assessment using a visual analogue scale and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of patient's assessment of pain using a visual analogue scale and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of C-reactive protein and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of the change in the PMR-activity score between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of the change in morning stiffness between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of the change in ability to elevate the upper limbs between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of the change in physician's global assessment using a visual analogue scale between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of the change in patient's assessment of pain using a visual analogue scale between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Determination of the sensitivity and specificity of the change in C-reactive protein between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Measured: 52 weeks
Evolution of Health Assessment Questionnaire-Disability Index (HAQ-DI) over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of the ability to elevate the upper limbs over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of the patient's assessment of pain using a visual analogue scale over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of the patient's assessment of global health using a visual analogue scale over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of the patient's assessment of fatigue using a visual analogue scale over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of the patient's assessment of stiffness using a visual analogue scale over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of morning stiffness time over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of 36-item Short Form Health Survey (SF36) over the disease course and the relation with disease activity
Measured: 52 weeks
Evolution of the PMR impact scale over the disease course and the relation with disease activity
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2022
- Study start1 Jul 2022
- Primary completion9 Dec 2024
- Study completion9 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Polymyalgia RheumaticaStudies of the same condition, open and under way.
- More trials run by Universitaire Ziekenhuizen KU LeuvenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 133 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universitaire Ziekenhuizen KU Leuven
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Steven Vanderschueren, MD, PhD, Principal investigator, Universitaire Ziekenhuizen KU Leuven
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05542316
- Sponsor's protocol ID
- S66638
- Status last verified
- Mar 2025
- First submitted
- 7 Sep 2022
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Leeb BF, Bird HA. A disease activity score for polymyalgia rheumatica. Ann Rheum Dis. 2004 Oct;63(10):1279-83. doi: 10.1136/ard.2003.011379.
PubMed 15361387Mackie SL, Twohig H, Neill LM, Harrison E, Shea B, Black RJ, Kermani TA, Merkel PA, Mallen CD, Buttgereit F, Mukhtyar C, Simon LS, Hill CL; OMERACT PMR Working Group. The OMERACT Core Domain Set for Outcome Measures for Clinical Trials in Polymyalgia Rheumatica. J Rheumatol. 2017 Oct;44(10):1515-1521. doi: 10.3899/jrheum.161109. Epub 2017 Aug 1.
PubMed 28765246Twohig H, Owen C, Muller S, Mallen CD, Mitchell C, Hider S, Hill C, Shea B, Mackie SL. Outcomes Measured in Polymyalgia Rheumatica and Measurement Properties of Instruments Considered for the OMERACT Core Outcome Set: A Systematic Review. J Rheumatol. 2021 Jun;48(6):883-893. doi: 10.3899/jrheum.200248. Epub 2020 Aug 1.
PubMed 32739892Twohig H, Mitchell C, Mallen CD, Muller S. Development and psychometric evaluation of the PMR-Impact Scale: a new patient reported outcome measure for polymyalgia rheumatica. Rheumatology (Oxford). 2023 Feb 1;62(2):758-765. doi: 10.1093/rheumatology/keac317.
PubMed 35639659
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of the Polymyalgia Rheumatica Activity Score for Determination of Disease Activity
- ClinicalTrials.gov ID
- NCT05542316
- Acronym / sponsor ID
- S66638
- Conditions
- Polymyalgia Rheumatica
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 133 participants (actual)
- Lead sponsor
- Universitaire Ziekenhuizen KU LeuvenUniversity, hospital or other
- Study start
- 1 Jul 2022 (actual)
- Primary completion
- 9 Dec 2024 (actual)
- Study completion
- 9 Dec 2024 (actual)
- First posted
- 15 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Polymyalgia rheumatica (PMR) is a systemic inflammatory disease that affects elderly people. It is characterized by pain and morning stiffness in the shoulders, pelvic girdles and neck. Glucocorticoids are the mainstay of the treatment. In clinical practice, the disease activity of PMR and corresponding treatment changes are based on the presence of symptoms and inflammatory markers. The interpretation of these abnormalities can be surprisingly difficult, especially when they are not consistent. In 2004, Leeb and Bird developed a composite score for measurement of disease activity in PMR, called the polymyalgia rheumatica activity score. It consists of 5 domains: morning stiffness time, ability to elevate the upper limbs, physician's global assessment, pain and CRP level. However, high-quality evidence on the measurement properties is lacking and there is still no consensus on the optimal cut off point.
Based on a Delphi study with physicians and patients OMERACT defines laboratory markers of systemic inflammation, pain, stiffness and physical function as the four inner core of domains considered mandatory for clinical trials of PMR, most frequently measured by erythrocyte sedimentation rate (ESR) and/or C-reactive protein (CRP), visual analogue scale (VAS) for pain, morning stiffness time and Health Assessment Questionnaire-Disability Index (HAQ-DI) respectively. Patient's global fatigue was strongly recommended to measure in PMR as well. Recently, a PMR-specific patient-reported outcome measure was developed, called the PMR impact scale. However, outcome measures in PMR studies lack consistency and there is no high-quality evidence on the measurement properties. In addition, the evolution of these patient reported outcomes is not known.
Detailed description
The primary objective of this prospective observational trial is to validate the PMR-activity score and to determine the optimal cut-off point to discriminate between disease remission and active disease. The secondary objective is to assess the evolution of the patient reported outcomes during the disease course and their relation with disease activity.
The investigators intend to perform a 12-month, observational prospective trial in patients with recently diagnosed PMR. Patients will be followed and treated as standard of care. Follow-up visits will be planned at 8 weeks, 16 weeks, 26 weeks and 52 weeks. As in standard of care, additional visits can be planned if necessary, e.g. in case of symptoms suggestive of relapse. Each visit ESR, CRP, morning stiffness time, ability to elevate the upper limbs, physician's global assessment and VAS for pain as components of the PMR-activity score and VAS for global health, pain, stiffness and fatigue, HAQ-DI, Short Form Health survey (SF36) and PMR impact scale as patient-reported outcomes will be collected.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with a final diagnosis of PMR | – | – |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Diagnosis of PMR
Key inclusion criteria (3 listed)
- Diagnosis of PMR based on the judgment of an experienced clinician (affiliated with the general internal medicine or rheumatology department), taking into account all available information (clinical symptoms, biochemical, radiological, and PET results)
- Able to give informed consent
- Understanding and able to write Dutch, English or French
Key exclusion criteria (3 listed)
- Concomitant diagnosis of giant cell arteritis (new diagnosis or being treated for giant cell arteritis)
- Concurrent rheumatoid arthritis, other inflammatory arthritis or other connective tissue disease
- Patients on glucocorticoids or other immunosuppressive drugs for another indication
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Determination of the sensitivity and specificity of the PMR-activity score and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
20 secondary outcomes
Determination of the sensitivity and specificity of morning stiffness time and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of ability to elevate the upper limbs and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of physician's global assessment using a visual analogue scale and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of patient's assessment of pain using a visual analogue scale and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of C-reactive protein and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of the change in the PMR-activity score between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of the change in morning stiffness between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of the change in ability to elevate the upper limbs between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of the change in physician's global assessment using a visual analogue scale between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of the change in patient's assessment of pain using a visual analogue scale between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Determination of the sensitivity and specificity of the change in C-reactive protein between two visits and the optimal cut-off point to discriminate between disease remission and active disease
Time frame: 52 weeks
Evolution of Health Assessment Questionnaire-Disability Index (HAQ-DI) over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of the ability to elevate the upper limbs over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of the patient's assessment of pain using a visual analogue scale over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of the patient's assessment of global health using a visual analogue scale over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of the patient's assessment of fatigue using a visual analogue scale over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of the patient's assessment of stiffness using a visual analogue scale over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of morning stiffness time over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of 36-item Short Form Health Survey (SF36) over the disease course and the relation with disease activity
Time frame: 52 weeks
Evolution of the PMR impact scale over the disease course and the relation with disease activity
Time frame: 52 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
- Background: PubMed 15361387Leeb BF, Bird HA. A disease activity score for polymyalgia rheumatica. Ann Rheum Dis. 2004 Oct;63(10):1279-83. doi: 10.1136/ard.2003.011379.
- Background: PubMed 28765246Mackie SL, Twohig H, Neill LM, Harrison E, Shea B, Black RJ, Kermani TA, Merkel PA, Mallen CD, Buttgereit F, Mukhtyar C, Simon LS, Hill CL; OMERACT PMR Working Group. The OMERACT Core Domain Set for Outcome Measures for Clinical Trials in Polymyalgia Rheumatica. J Rheumatol. 2017 Oct;44(10):1515-1521. doi: 10.3899/jrheum.161109. Epub 2017 Aug 1.
- Background: PubMed 32739892Twohig H, Owen C, Muller S, Mallen CD, Mitchell C, Hider S, Hill C, Shea B, Mackie SL. Outcomes Measured in Polymyalgia Rheumatica and Measurement Properties of Instruments Considered for the OMERACT Core Outcome Set: A Systematic Review. J Rheumatol. 2021 Jun;48(6):883-893. doi: 10.3899/jrheum.200248. Epub 2020 Aug 1.
- Background: PubMed 35639659Twohig H, Mitchell C, Mallen CD, Muller S. Development and psychometric evaluation of the PMR-Impact Scale: a new patient reported outcome measure for polymyalgia rheumatica. Rheumatology (Oxford). 2023 Feb 1;62(2):758-765. doi: 10.1093/rheumatology/keac317.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven Vanderschueren, MD, PhD principal investigator
Universitaire Ziekenhuizen KU Leuven
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polymyalgia Rheumatica, run by Universitaire Ziekenhuizen KU Leuven at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determination of the sensitivity and specificity of the PMR-activity score and the optimal cut-off point to discriminate between disease remission and active disease (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05542316, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025.