Summary
Azathioprine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Rheumatoid Arthritis would be drug repurposing rather than first-in-human development. This does not mean it is approved for Rheumatoid Arthritis. ClinicalTrials.gov lists 8 registered trials linking Azathioprine to Rheumatoid Arthritis: 1 is currently recruiting; 1 is active or not yet recruiting; 5 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03449758). The largest enrolment is 6393 participants (NCT01646385). Registration activity spans 2005 to 2025. The literature layer holds 18 publications for this pair: 5 Cochrane reviews, 4 meta-analyses, 3 systematic reviews, 1 randomised controlled trial publication and 5 clinical trial publications. Publication years run from 1996 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Rheumatoid Arthritis ranks 544 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: 11% achieve drug-free remission (DFR) at median 74 months; most cohorts show 10-15% DFR ceiling.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 80.0 (trials 30.0, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 1 active / not yet recruiting, 5 completed, 1 other |
| Linked publications | 18 (5 Cochrane reviews, 5 clinical trial publications, 4 meta-analyses, 3 systematic reviews, 1 randomised controlled trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT03449758 | Effect of Sarilumab on Patient-reported Outcomes in Patients With Active Rheumatoid Arthritis 2018 | completed | Phase 4 | 84 |
| NCT02281799 | Thiopurine Induced Pancreatitis in IBD Patients 2015 | withdrawn | Phase 4 | — |
| NCT00531817 | A Study of Tocilizumab in Combination With DMARDs in Patients With Moderate to Severe Rheumatoid Arthritis 2007 | completed | Phase 3 | 619 |
| NCT01941095 | A Study of Subcutaneous Tocilizumab as Monotherapy and/or in Combination With Non-Biologic Disease Modifying Anti-Rheumatic Drugs (DMARDs) in Participants With Rheumatoid Arthritis 2013 | completed | Phase 3 | 100 |
| NCT03414502 | Treatment of Rheumatoid Arthritis With DMARDs: Predictors of Response 2007 | recruiting | Phase 3 | 400 |
| NCT01033656 | Treatment of Refractory Adult-onset Still's Disease With Anakinra: a Randomized Study 2005 | completed | Phase 2 | 23 |
| NCT07138898 | Immunosuppressant Management in Rheumatology Patients Undergoing Elective Total Shoulder Arthroplasty 2025 | not yet recruiting | Phase 2 | 80 |
| NCT01646385 | Study to Assess the Safety and Efficacy of Etanercept in Patients Treated Over the Long-term in Real-world Clinical Practice, Using Data Collected by the British Society of Rheumatology Biologics Registry 2012 | completed | Not applicable | 6,393 |
Published literature
- Meta-analysis Effect of monotherapy with conventional synthetic disease-modifying anti-rheumatic drugs or glucocorticoids on radiographic progression in rheumatoid arthritis: a network meta-analysis of 64 treatment arms from 31 randomized controlled trialsGuski LS, Pedder H, Andersn SE et al. · Scandinavian journal of rheumatology · 2026 · PMID 41858241
- Meta-analysis Progression of tuberculosis among patients with rheumatic diseases - A systematic review and meta-analysisSundaram K, Vajravelu LK, Velayutham R et al. · The Indian journal of tuberculosis · 2025 · PMID 40518207
- Clinical trial publication Immunogenicity of adalimumab reference product and adalimumab-adbm in patients with rheumatoid arthritis, Crohn's disease and chronic plaque psoriasis: a pooled analysis of the VOLTAIRE trialsStrand V, McCabe D, Bender S · BMJ open · 2024 · PMID 39551590
- Systematic review Overlap syndrome of anti-aquaporin 4 positive neuromyelitis optica spectrum disorder and primary Sjögren's syndrome: a systematic review of individual patient dataPrasad CB, Kopp CR, Naidu G et al. · Rheumatology international · 2024 · PMID 37500817
- Clinical trial publication Treatment Outcomes for Rheumatoid Arthritis-Associated Interstitial Lung Disease: A Real-World, Multisite Study of the Impact of Immunosuppression on Pulmonary Function TrajectoryMatson SM, Baqir M, Moua T et al. · Chest · 2023 · PMID 36470416
- Meta-analysis Cost-utility analysis of biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) combined with methotrexate for Thai rheumatoid arthritis patients with high disease activityPrawjaeng J, Leelahavarong P, Budtarad N et al. · BMC health services research · 2023 · PMID 37259090
- Systematic review Treatment of relapsing polychondritis: a systematic reviewPetitdemange A, Sztejkowski C, Damian L et al. · Clinical and experimental rheumatology · 2022 · PMID 35238756
- Systematic review Immunosuppressive treatment for peripheral neuropathies in Sjogren's syndrome - a systematic reviewHumă AC, Kecskeş EM, Tulbă D et al. · Romanian journal of internal medicine = Revue roumaine de medecine interne · 2020 · PMID 31527298
- Cochrane review To stop the erosion of hope: the DMARD category and the place of semantics in modern rheumatologyBuer JK · Inflammopharmacology · 2017 · PMID 28194545
- Cochrane review Methotrexate monotherapy and methotrexate combination therapy with traditional and biologic disease modifying anti-rheumatic drugs for rheumatoid arthritis: A network meta-analysisHazlewood GS, Barnabe C, Tomlinson G et al. · The Cochrane database of systematic reviews · 2016 · PMID 27571502
- Meta-analysis Preoperative Use of Methotrexate and the Risk of Early Postoperative Complications in Patients with Inflammatory Bowel DiseaseAfzali A, Park CJ, Zhu K et al. · Inflammatory bowel diseases · 2016 · PMID 27057681
- Cochrane review Methotrexate for treating rheumatoid arthritisLopez-Olivo MA, Siddhanamatha HR, Shea B et al. · The Cochrane database of systematic reviews · 2014 · PMID 24916606
- Cochrane review Interstitial lung diseases induced or exacerbated by DMARDS and biologic agents in rheumatoid arthritis: a systematic literature reviewRoubille C, Haraoui B · Seminars in arthritis and rheumatism · 2014 · PMID 24231065
- Clinical trial publication [The clinical observation of different therapeutic strategies in combined primary biliary cirrhosis and Sjögren syndrome]Gao LX, Zhang FC, Wang L et al. · Zhonghua nei ke za zhi · 2012 · PMID 23291020
- Cochrane review Current evidence for the management of rheumatoid arthritis with synthetic disease-modifying antirheumatic drugs: a systematic literature review informing the EULAR recommendations for the management of rheumatoid arthritisGaujoux-Viala C, Smolen JS, Landewé R et al. · Annals of the rheumatic diseases · 2010 · PMID 20447954
- Clinical trial publication Radiological and clinical results of longterm treatment of rheumatoid arthritis with methotrexate and azathioprineKerstens PJ, Boerbooms AM, Jeurissen ME et al. · The Journal of rheumatology · 2000 · PMID 10813280
- Clinical trial publication A double blind placebo controlled trial of azathioprine in the treatment of primary Sjögren's syndromePrice EJ, Rigby SP, Clancy U et al. · The Journal of rheumatology · 1998 · PMID 9598887
- Randomized controlled trial Combination treatment of rheumatoid arthritis using azathioprine and methotrexate: a 48 week controlled clinical trialWillkens RF, Stablein D · The Journal of rheumatology. Supplement · 1996 · PMID 8833056
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Azathioprine approved for Rheumatoid Arthritis?
Azathioprine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Rheumatoid Arthritis would be drug repurposing rather than first-in-human development. This does not mean it is approved for Rheumatoid Arthritis. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Azathioprine in clinical trials for Rheumatoid Arthritis?
ClinicalTrials.gov lists 8 registered trials linking Azathioprine to Rheumatoid Arthritis: 1 is currently recruiting; 1 is active or not yet recruiting; 5 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03449758). The largest enrolment is 6393 participants (NCT01646385). Registration activity spans 2005 to 2025.
What does the evidence show for Azathioprine in Rheumatoid Arthritis?
Azathioprine has both completed registered trials and synthesis-level publications linked to Rheumatoid Arthritis. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.