Polymyalgia Rheumatica (PMR)NCT05479448
Predictive Factors for Treatment Response in Patients With Newly-diagnosed Polymyalgia Rheumatica and Giant Cell Arteritis
An observational study of Polymyalgia Rheumatica (PMR) and Giant Cell Arteritis (GCA), run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
Trial summary
This prospective study is to explore different predictive factors for response to steroid treatment in patients with PMR and/or GCA. It evaluates the association of endogenous GC suppression (plasma and urinary cortisol and cortisone) to the responsiveness of PMR/GCA to GCs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polymyalgia Rheumatica (PMR), Giant Cell Arteritis (GCA)
- What is being tested?
- Data collection for cellular analyses (Immune subset composition, GCR expression, in vitro steroid responsiveness), Data collection for exploratory analyses of endogenous steroid hormones and Data collection for correlation between clinical defined and lab defined GC responsivness
- How many people
- 30 participants
- Where
- 1 siteSwitzerland
- Study dates
- 3 Jun 2022to Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- Adult, Older adult
Study population: Patients presenting in the University Hospital Basel with newly diagnosed PMR according to the 2012 provisional classification criteria ) and patients with newly diagnosed GCA will be screened for inclusion. Final diagnosis of GCA is made either (i) if temporal artery biopsy is positive, (ii) if patients fulfill the 1989 ACR criteria, or (iii) if they fulfill at least 2/5 ACR criteria in combination with typical vasculitic ultrasound findings or vasculitic findings in other imaging methods.
Who may be able to join
- Patients with diagnosis of PMR according to the 2012 provisional classification criteria and GCA according to published criteria
- Consent to participate in the SCQM database
- Treatment according to our standardized regimes
Who cannot join
- Treatment with Tocilizumab, MTX or other disease modifying medications at inclusion
- History of GCA and PMR in the past
- Inability to give informed consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Switzerland1 site, 1 recruiting
- Recruiting
Department of Rheumatology University Hospital Basel
Basel, 4031
Thomas Daikeler, Prof. Dr. med., +41 61 265 27 09, Thomas.Daikeler@usb.ch
Thomas Daikeler, Prof. Dr. med.
About this study
This prospective study is to explore different predictive factors for response to steroid treatment in patients with PMR and/or GCA. It evaluates the association of endogenous GC suppression (plasma and urinary cortisol and cortisone) to the responsiveness of PMR/GCA to GCs.
Read the detailed description
Polymyalgia rheumatica (PMR) and giant cell arteritis (GCA) are closely related inflammatory rheumatic diseases. The first-line treatment of both PMR and GCA are glucocorticoids (GC). In PMR, initial oral prednisone equivalent doses in between 10 and 25 mg/day are given. In contrast, GCA is usually treated with significantly higher steroid doses (starting dose 1 mg/kg body-weight) to prevent vascular complications.
The dose and duration of steroid treatment needed to control disease in patients with PMR and GCA vary and about half of the patients experience relapses, early upon GC dose tapering or after discontinuation of treatment. The reasons for the inter-individual differences in steroid-response are not known. Data from this controlled prospective study will help to identify subjects with GC resistance which would allow to use intensified treatment strategies (higher dose or alternative immune modulatory therapy) to overcome resistance. On the other hand, strong responsiveness to GC would provide a rational for rapid steroid tapering or treatment with lower doses, both resulting in reduced risk for GC related adverse events such as osteoporosis, infections, diabetes and mood disorders. Detailed understanding of the relation of individual GC signaling and GC metabolism with patients' response to steroid treatment will help to define steroid responder profiles. This prospective study is to explore different predictive factors for response to steroid treatment in patients with PMR and/or GCA.
At inclusion and at all follow-up visits, the clinical evaluation will be documented in the SCQM database. All participants with PMR will be treated according to our local treatment protocol: starting dose is 15 mg prednisone/d, tapered by 2.5 mg every second week once symptoms are controlled. After tapering to 10 mg/d, prednisone dose is further reduced by 2.5 mg every month.
All patients with GCA are treated according to published guidelines with prednisone starting at 1mg/kg body-weight followed by reduction to 0 mg at week 26 (GIACTA protocol).
- Being tested
- Data collection for cellular analyses (Immune subset composition, GCR expression, in vitro steroid responsiveness), Data collection for exploratory analyses of endogenous steroid hormones, Data collection for correlation between clinical defined and lab defined GC responsivness, Data collection for Prednisone metabolism
- Keywords
- Large vessel vasculitis (LVV), Glucocorticoid receptor (GCR), glucocorticoid (GC), steroid-response, GCR expression levels, GCR sensitivity/responsiveness, prednisolone, prednisone, Swiss Clinical Quality Management in Rheumatic Diseases (SCQM), Glucocorticoid resistance, endogenous GC suppression
- Medical subject headings
- Polymyalgia Rheumatica, Giant Cell Arteritis, Glucocorticoid Receptor Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Data collection for cellular analyses (Immune subset composition, GCR expression, in vitro steroid responsiveness)Other
Biological material will be sampled at three time-points. The first time-point will be when patients have been treated with 15 mg of prednisone per day for at least 5 days. A second time-point will be after prednisone was successfully tapered and maintained at 5 mg per day for at least 5 days, a third time point will be 4 weeks after the stop of prednisone. Biosampling is done for cellular analyses, pharmacokinetics and hormone measurements.
Data collection for exploratory analyses of endogenous steroid hormonesOther
Concentrations of GC, MC, androgens and progestins will be determined and the suppression of cortisol and cortisone upon prednisone treatment will be analyzed.
Data collection for correlation between clinical defined and lab defined GC responsivnessOther
The time to first relapse, the cumulative steroid dose at 1 year after diagnosis, the need for treatment with steroid sparing agents and the GTI after 1 year will be correlated to the percentage of in vitro inhibition of cytokine concentration by dexamethasone treatment, to the prednisone/prednisolone ratio in plasma, to the percentage of endogenous GC suppression (plasma and urinary cortisol and cortisone) by prednisone treatment. Furthermore, correlations of steroid sensitivity with Mineralocorticoid (MC) and androgens will be investigated.
Data collection for Prednisone metabolismOther
Plasma concentrations of prednisone and its active metabolite prednisolone will be quantified by liquid chromatography-tandem mass spectrometry (LC-MS/MS). If changes on prednisone/prednisolone are observed, the quantification of the 6-hydroxylated prednisone/prednisolone metabolites in 24 h urine samples will be performed to estimate their metabolism as well as the ratio of inactive to active GC.
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse (no/yes) of PMR/ GCA
Relapse (no/yes) of PMR/ GCA (associated with endogenous cortisol levels under stable doses of 15 mg of prednisone). Relapse of GCA and or PMR is defined as intensification of immune-suppressive treatment due to symptoms, signs or laboratory values judged by the caring physician to be due to PMR or GCA.
Measured: Within one year after PMR/GCA diagnosis
Secondary outcomes (5)
Cumulative steroid dose at 1 year after diagnosis
Cumulative steroid dose at 1 year after diagnosis
Measured: At 1 year after diagnosis
Number of patients with Tocilizumab or other immunosuppressive/ biological treatment started within 1 year after diagnosis.
Number of patients with Tocilizumab or other immunosuppressive/ biological treatment started within 1 year after diagnosis.
Measured: Within one year after PMR/GCA diagnosis
Number of patients with Methotrexate (MTX) treatment started within 1 year after diagnosis
Number of patients with Methotrexate (MTX) treatment started within 1 year after diagnosis
Measured: Within one year after PMR/GCA diagnosis
Prednisone/prednisolone ratio in plasma
Prednisone/prednisolone ratio in plasma
Measured: Within one year after PMR/GCA diagnosis
Glucocorticoid Toxicity Index (GTI) at 1 year after diagnosis
Glucocorticoid Toxicity Index (GTI) at 1 year after diagnosis. The GTI is composed of 9 domains and measures the change in glucocorticoid toxicity between 2 points in time. The GTI can measure not only the worsening of glucocorticoid toxicity but also its improvement. The minimal clinically important difference for the GTI scores is 10.
Measured: At 1 year after diagnosis
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2022
- Study start3 Jun 2022
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polymyalgia Rheumatica (PMR)Studies of the same condition, open and under way.
- 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
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Collaborators
- Schweizerische Stiftung für die Erforschung der Muskelkrankheiten, Novartis
- Responsible party
- Sponsor
- Investigators
- Thomas Daikeler, Prof. Dr. med., Principal investigator, Department of Rheumatology University Hospital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05479448
- Sponsor's protocol ID
- 2022-00788; mu22Daikeler
- Status last verified
- Mar 2025
- First submitted
- 26 Jul 2022
- Last updated
- 1 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predictive Factors for Treatment Response in Patients With Newly-diagnosed Polymyalgia Rheumatica and Giant Cell Arteritis
- ClinicalTrials.gov ID
- NCT05479448
- Acronym / sponsor ID
- 2022-00788; mu22Daikeler
- Keywords
- Large vessel vasculitis (LVV), Glucocorticoid receptor (GCR), glucocorticoid (GC), steroid-response, GCR expression levels, GCR sensitivity/responsiveness, prednisolone, prednisone
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Collaborators
- Schweizerische Stiftung für die Erforschung der Muskelkrankheiten, Novartis
- Study start
- 3 Jun 2022 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 29 Jul 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.
This prospective study is to explore different predictive factors for response to steroid treatment in patients with PMR and/or GCA. It evaluates the association of endogenous GC suppression (plasma and urinary cortisol and cortisone) to the responsiveness of PMR/GCA to GCs.
Detailed description
Polymyalgia rheumatica (PMR) and giant cell arteritis (GCA) are closely related inflammatory rheumatic diseases. The first-line treatment of both PMR and GCA are glucocorticoids (GC). In PMR, initial oral prednisone equivalent doses in between 10 and 25 mg/day are given. In contrast, GCA is usually treated with significantly higher steroid doses (starting dose 1 mg/kg body-weight) to prevent vascular complications.
The dose and duration of steroid treatment needed to control disease in patients with PMR and GCA vary and about half of the patients experience relapses, early upon GC dose tapering or after discontinuation of treatment. The reasons for the inter-individual differences in steroid-response are not known. Data from this controlled prospective study will help to identify subjects with GC resistance which would allow to use intensified treatment strategies (higher dose or alternative immune modulatory therapy) to overcome resistance. On the other hand, strong responsiveness to GC would provide a rational for rapid steroid tapering or treatment with lower doses, both resulting in reduced risk for GC related adverse events such as osteoporosis, infections, diabetes and mood disorders. Detailed understanding of the relation of individual GC signaling and GC metabolism with patients' response to steroid treatment will help to define steroid responder profiles. This prospective study is to explore different predictive factors for response to steroid treatment in patients with PMR and/or GCA.
At inclusion and at all follow-up visits, the clinical evaluation will be documented in the SCQM database. All participants with PMR will be treated according to our local treatment protocol: starting dose is 15 mg prednisone/d, tapered by 2.5 mg every second week once symptoms are controlled. After tapering to 10 mg/d, prednisone dose is further reduced by 2.5 mg every month.
All patients with GCA are treated according to published guidelines with prednisone starting at 1mg/kg body-weight followed by reduction to 0 mg at week 26 (GIACTA protocol).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Patients presenting in the University Hospital Basel with newly diagnosed PMR according to the 2012 provisional classification criteria ) and patients with newly diagnosed GCA will be screened for inclusion. Final diagnosis of GCA is made either (i) if temporal artery biopsy is positive, (ii) if patients fulfill the 1989 ACR criteria, or (iii) if they fulfill at least 2/5 ACR criteria in combination with typical vasculitic ultrasound findings or vasculitic findings in other imaging methods.
Key inclusion criteria (3 listed)
- Patients with diagnosis of PMR according to the 2012 provisional classification criteria and GCA according to published criteria
- Consent to participate in the SCQM database
- Treatment according to our standardized regimes
Key exclusion criteria (3 listed)
- Treatment with Tocilizumab, MTX or other disease modifying medications at inclusion
- History of GCA and PMR in the past
- Inability to give informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Data collection for cellular analyses (Immune subset composition, GCR expression, in vitro steroid responsiveness)Biological material will be sampled at three time-points. The first time-point will be when patients have been treated with 15 mg of prednisone per day for at least 5 days. A second time-point will be after prednisone was successfully tapered and maintained at 5 mg per day for at least 5 days, a third time point will be 4 weeks after the stop of prednisone. Biosampling is done for cellular analyses, pharmacokinetics and hormone measurements. | Other | – |
| Data collection for exploratory analyses of endogenous steroid hormonesConcentrations of GC, MC, androgens and progestins will be determined and the suppression of cortisol and cortisone upon prednisone treatment will be analyzed. | Other | – |
| Data collection for correlation between clinical defined and lab defined GC responsivnessThe time to first relapse, the cumulative steroid dose at 1 year after diagnosis, the need for treatment with steroid sparing agents and the GTI after 1 year will be correlated to the percentage of in vitro inhibition of cytokine concentration by dexamethasone treatment, to the prednisone/prednisolone ratio in plasma, to the percentage of endogenous GC suppression (plasma and urinary cortisol and cortisone) by prednisone treatment. Furthermore, correlations of steroid sensitivity with Mineralocorticoid (MC) and androgens will be investigated. | Other | – |
| Data collection for Prednisone metabolismPlasma concentrations of prednisone and its active metabolite prednisolone will be quantified by liquid chromatography-tandem mass spectrometry (LC-MS/MS). If changes on prednisone/prednisolone are observed, the quantification of the 6-hydroxylated prednisone/prednisolone metabolites in 24 h urine samples will be performed to estimate their metabolism as well as the ratio of inactive to active GC. | Other | – |
Primary outcome
Relapse (no/yes) of PMR/ GCA
Time frame: Within one year after PMR/GCA diagnosis
Relapse (no/yes) of PMR/ GCA (associated with endogenous cortisol levels under stable doses of 15 mg of prednisone). Relapse of GCA and or PMR is defined as intensification of immune-suppressive treatment due to symptoms, signs or laboratory values judged by the caring physician to be due to PMR or GCA.
5 secondary outcomes
Cumulative steroid dose at 1 year after diagnosis
Time frame: At 1 year after diagnosis
Number of patients with Tocilizumab or other immunosuppressive/ biological treatment started within 1 year after diagnosis.
Time frame: Within one year after PMR/GCA diagnosis
Number of patients with Methotrexate (MTX) treatment started within 1 year after diagnosis
Time frame: Within one year after PMR/GCA diagnosis
Prednisone/prednisolone ratio in plasma
Time frame: Within one year after PMR/GCA diagnosis
Glucocorticoid Toxicity Index (GTI) at 1 year after diagnosis
Time frame: At 1 year after diagnosis
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2025.
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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.
Thomas Daikeler, Prof. Dr. med. principal investigator
Department of Rheumatology University Hospital Basel
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 (PMR) and Giant Cell Arteritis (GCA), run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
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: looking for participants now, as of the sponsor's last update on 1 Apr 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 Switzerland. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Thomas Daikeler, Prof. Dr. med. (+41 61 265 27 09, Thomas.Daikeler@usb.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relapse (no/yes) of PMR/ GCA (time frame: Within one year after PMR/GCA diagnosis). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Apr 2025.