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Polymyalgia RheumaticaPhase 2NCT04799262

Tofacitinib as a GC Sparing Agent for Polymyalgia Rheumatica

A Phase 2 study testing Tofacitinib 5 MG in people with Polymyalgia Rheumatica and Effect of Drug, run by RenJi Hospital at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Glucocorticoids are the cornerstone treatment for polymyalgia rheumatica but induce adverse events. The efficacy of the candidate drug Tofacitinib has not yet been demonstrated in controlled studies. Read more about this study

Who is it for?
Ages 50 to 85 years
What is being tested?
Tofacitinib 5 MG
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
1 siteChina
Study dates
1 Jan 2021to 1 May 2022
Phase
Phase 2
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 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Female or male between 50 and 85 years old.
  • PMR according to the ACR/ EULAR 2012 PMR classification criteria.
  • Patients must have erythrocyte sedimentation rate (ESR) ≥20 mm/hr and/or CRP ≥8 mg/L associated with highly active PMR (PMR-AS>17) within 2 weeks prior to screening.
  • Patient is willing and able to take prednisone of 15 mg/day at baseline.
  • Signed written informed consent.

Who cannot join

  • Presence of any other connective tissue disease, such as but not limited to giant-cell arteritis, systemic lupus erythematosus, systemic sclerosis, vasculitis, myositis, mixed connective tissue disease, and ankylosing spondylitis.
  • Concurrent diagnosis of active fibromyalgia, rhabdomyolysis or neuropathic muscular diseases.
  • Organ transplant recipient.
  • Any prior (within the defined period below) or concurrent use of immunosuppressive therapies but not limited to any of the following: ① Any prior use of tumor necrosis factor inhibitors, anti-IL-6 agents or JAK inhibitor; ② Alkylating agents including cyclophosphamide within 6 months of baseline; ③ Cell-depletion agents (e.g. anti-CD20) without evidence of recovery of B cells to baseline level; ④ Abatacept within 8 weeks of baseline; ⑤ Any prior use of csDMARDs at unstable dose for less than 12 weeks before baseline, e.g. cyclosporine, azathioprine, mycophenolate mofetil, leflunomide, MTX; ⑥ Concurrent use of systemic GCs for conditions other than PMR.
  • Evidence (as assessed by the investigators) of active infection, such as presence of hepatitis B surface antigen (HBsAg) or hepatitis C antibody in blood, human immunodeficiency virus (HIV) positivity.
  • Patients with a history of active or recurrent herpes zoster.
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  • Patients who have had surgery within 4 weeks of screening or planned surgery during study.
  • Malignancy within 5 years prior to screening, except for non-melanoma skin cancer.
  • Pregnant or breastfeeding woman.
  • Any medical condition that could interfere with the implementation or interpretation of the study or with the safety of the patient during the study.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Ren Ji Hospital

    Shanghai, 201112

Other trials in China

About this study

Official title: Phase II Study of Efficacy and Safety of Tofacitinib in Patients With Polymyalgia Rheumatica

Glucocorticoids are the cornerstone treatment for polymyalgia rheumatica but induce adverse events. The efficacy of the candidate drug Tofacitinib has not yet been demonstrated in controlled studies. The aim of the study is to investigate the efficacy and safety of Tofacitinib as a glucocorticoid sparing agent in patients with polymyalgia rheumatica.

Read the detailed description

A two-stage, phase 2 clinical trial was conducted to test whether tofacitinib would take into effect as a glucocorticoid sparing agent in patients with polymyalgia rheumatica. Tofacitinib was given at the dose of 10mg daily through the 24 weeks. Patients were to receive prednisone in a dosage of 15mg daily (or equivalent oral GCs) at baseline and tapered to 2.5mg or less daily within 20 weeks. The primary endpoint was the response to treatment, defined as the achievement of sustained low disease activity (PMR-AS<7) with GC independence (prednisone≤2.5mg daily or equivalent oral GCs) for 4 weeks from week 20.

The trial will be conducted following a two-stage Simon minimax design. After 8 participants have completed their 24-week follow up there will be an interim analysis. If there are 3 or more failures out of these 8 then the trial will stop with the conclusion that the study of Tofacitinib should be abandoned. If there are fewer than 3 failures then the study will continue until a further 6 participants have received treatment, giving a total sample size of 14. If amongst these 14 participants there are 4 or more failures then it will be concluded that further study of Tofacitinib should be abandoned. If further study of the drug is not abandoned at either the interim of the final analysis, then a recommendation to conduct a comparative, randomized phase III trial will be made.

Being tested
Tofacitinib 5 MG
Medical subject headings
Polymyalgia Rheumatica

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Tofacitinib 5 MGDrug

    Also called Prednisone

    Oral tofacitinib at the dose of 5mg twice a day was given for 24 weeks. Prednisone (or equivalent oral GCs) of 15 mg daily at baseline willed be tapered to 2.5 mg or less within 20 weeks. Unless specifically considered by patients and physicians, the GC will be tapered followed the predefined taper regimen depending on the response to treatment judged by PMR-AS. The PMR-AS will be determined every two weeks; if<10 the daily GC dosage was decreased by 2.5mg; if>17, the GC daily dosage was increased to the previous dosage; if 10≤PMR-AS≤17, the GC daily dosage was maintained at the previous stable dose.

Study groups in NCT04799262
Study groupRoleWhat they receive
Tofacitinib+PrednisoneExperimentalTofacitinib was given at the dose of 10mg daily through the 24 weeks. Prednisone (or equivalent oral GCs) of 15 mg daily at baseline willed be tapered to 2.5 mg or less within 20 weeks. Unless specifically considered by patients and physicians, the GC will be tapered followed the predefined taper regimen depending on the response to treatment judged by PMR-AS. The PMR-AS will be determined every two weeks; if<10 the GC daily dosage was decreased by 2.5mg; if>17, the GC daily dosage was increased to the previous dosage; if 10≤PMR-AS≤17, the GC daily dosage was maintained at the previous stable dose.
Receives: Drug: Tofacitinib 5 MG

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Response to treatment

    Response to treatment is defined as the achievement of sustained low disease activity (PMR-AS<7) with GC independence (≤2.5mg/d) for 4 weeks from week 20

    Measured: 24 week

Secondary outcomes (9)

  1. Time till GC-free low disease activity within 24w

    Time till GC-free low disease activity (PMR-AS<7) within 24 weeks

    Measured: 24 week

  2. Time till first relapse within 24w

    Time till first relapse (PMR-AS ≥7) within 24w

    Measured: 24 week

  3. Cumulative GC dose at 24w

    Cumulative glucocorticoids dose over time

    Measured: 24 week

  4. Proportion of patients with sustained remission with GC independence for 4 weeks from week 20

    Proportion of patients with sustained remission (PMR-AS <1.5) with GC independence (≤2.5mg/d) for 4 weeks from week 20

    Measured: 24 week

  5. Incidence of adverse events (AEs) and Serious AEs (SAEs)

    Incidence of adverse events (AEs) and Serious AEs (SAEs) within 24 weeks

    Measured: 24 week

  6. Change of disease activity by PMR-AS

    Change of disease activity by PMR-AS within 24 weeks

    Measured: 24 week

  7. Assessment of quality of life using the MHAQ

    The Modified Health Assessment Questionnaire (MHAQ), reduced the number of items from 20 in the original HAQ to eight, and improved the feasibility in clinical practice when screening patients. The MHAQ score is calculated as the mean of the scores for each activity. Total score is between 0.0-3.0, in 0.125 increments. Higher scores indicate worse function and greater disability. MHAQ scores <0.3 are considered normal.

    Measured: 24 week

  8. Assessment of quality of life using the EQ-5D

    Health quality assessed by EuroQol five dimensions questionnaire. It is a preference-based measure that can be regarded as a continuous outcome scored on a -0.59 to 1.00 scale, with 1.00 indicating 'full health' and 0 representing dead.

    Measured: 24 week

  9. Circulating serum cytokines, immunoregulators, and inflammatory parameters

    On the circulating serum cytokines, immunoregulators (IL-6, IL-1, BLyS/BAFF, IL-6 receptor, gp130, etc.), B cells receptors, phenotype of circulating T- and B-cells will be collected at W0 and W24. On inflammatory parameters (CRP and ESR) every 4 weeks from baseline.

    Measured: 24 week

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2021
  2. Study start1 Jan 2021
  3. Primary completion16 Mar 2022
  4. Study completion1 May 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04799262
Sponsor's protocol ID
GCs Sparing Regimen in PMR
Status last verified
May 2022
First submitted
4 Mar 2021
Last updated
15 Sep 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Zhang L, Li J, Yin H, Chen D, Li Y, Gu L, Fu Y, Chen J, Chen Z, Yang S, Ye S, Li T, Lu L. Efficacy and safety of tofacitinib in patients with polymyalgia rheumatica: a phase 2 study. Ann Rheum Dis. 2023 May;82(5):722-724. doi: 10.1136/ard-2022-223562. Epub 2023 Jan 5. No abstract available.

    PubMed 36604153

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tofacitinib 5 MG in people with Polymyalgia Rheumatica and Effect of Drug, run by RenJi Hospital at 1 site in China.

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 5 inclusion and 10 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 15 Sep 2022. 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 China. 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.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

Response to treatment (time frame: 24 week). 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 NCT04799262, which also lists 1 linked publication.

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 15 Sep 2022.

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