Summary
Tocilizumab 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lists 8 registered trials linking Tocilizumab to Rheumatoid Arthritis: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT04251741). The largest enrolment is 241 participants (NCT00144547). Registration activity spans 2004 to 2020. The literature layer holds 13 publications for this pair: 5 Cochrane reviews, 2 meta-analyses, 1 randomised controlled trial publication and 5 clinical trial publications. Publication years run from 2024 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 | 78.0 (trials 28.0, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 6 completed, 2 other |
| Linked publications | 13 (5 Cochrane reviews, 5 clinical trial publications, 2 meta-analyses, 1 randomised controlled trial publication) |
| Agent type | Biologic |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT01717859 | Musculoskeletal Ultrasound in Predicting Early Dose Titration With Tocilizumab 2014 | completed | Phase 4 | 74 |
| NCT04251741 | Using Adalimumab Serum Concentration to Choose a Subsequent Biological DMARD in Rheumatoid Arthritis Patients Failing Adalimumab Treatment 2020 | status unknown | Phase 4 | 86 |
| NCT00773461 | A Study of Tocilizumab in Combination With DMARD Therapy in Patients With Active Rheumatoid Arthritis. 2008 | completed | Phase 3 | 209 |
| NCT00144547 | Long-term Treatment Study of MRA for Rheumatoid Arthritis (RA) From Study MRA012JP 2004 | completed | Phase 3 | 241 |
| NCT01587989 | A Study of RoActemra/Actemra (Tocilizumab) With or Without Methotrexate in Patients With Mild to Moderate Rheumatoid Arthritis With an Inadequate Response to Methotrexate 2012 | completed | Phase 3 | 77 |
| NCT01242488 | Efficacy and Safety of CDP6038 in Patients With Rheumatoid Arthritis With an Unsuccessful Response to Anti-Tumor Necrosis Factor (Anti-TNF) Therapy 2010 | completed | Phase 2 | 221 |
| NCT01564901 | An Observational Study of RoActemra/Actemra (Tocilizumab) in Patients With Rheumatoid Arthritis Treated With Glucocorticoids 2012 | completed | Not applicable | 23 |
| NCT03288584 | Effects of Interleukin-6 Inhibition on Vascular, Endothelial and Left Ventricular Function in Rheumatoid Arthritis 2017 | status unknown | Not applicable | 60 |
Published literature
- Clinical trial publication Efficacy and safety of filgotinib versus tocilizumab in active rheumatoid arthritis: A randomized, open-label, multicenter study with clinical and musculoskeletal ultrasound evaluation (TRANSFORM study)Shimizu T, Kawashiri SY, Koga T et al. · Drug discoveries & therapeutics · 2026 · PMID 42036316
- Clinical trial publication Certolizumab pegol, abatacept, tocilizumab or active conventional therapy in early rheumatoid arthritis: 48-week patient-reported outcomes from the NORD-STAR trialLampa J, Nordström D, van Vollenhoven R et al. · The Lancet. Rheumatology · 2026 · PMID 41881637
- Clinical trial publication Long-term safety and efficacy of tocilizumab in adult-onset Still's disease: open-label, long-term extension of the phase III trialSuzuki K, Kameda H, Ikeda K et al. · Rheumatology (Oxford, England) · 2026 · PMID 41609432
- Cochrane review Effect of targeted therapies on pulmonary function in rheumatoid arthritis-associated interstitial lung disease: a systematic review and meta-analysisHilliquin S, Vismara E, Berlengiero V et al. · RMD open · 2026 · PMID 41856563
- Cochrane review Long-term persistence of biological agents in the treatment of patients with rheumatoid arthritis: a systematic literature review and meta-analysisSchneeberger EE, Felquer MLA, Citera G et al. · Clinical rheumatology · 2026 · PMID 41160371
- Clinical trial publication Disease activity and treatment response in early rheumatoid arthritis: an exploratory metabolomic profiling in the NORD-STAR cohortFatima T, Zhang Y, Vasileiadis GK et al. · Arthritis research & therapy · 2025 · PMID 40713837
- Clinical trial publication Efficacy and Safety of Biosimilar CT-P47 Versus Reference Tocilizumab: 1-Year Results of a Randomised, Active-Controlled, Double-Blind, Phase III Study in Patients with Rheumatoid ArthritisBurmester G, Trefler J, Racewicz A et al. · Clinical drug investigation · 2025 · PMID 40650731
- Meta-analysis Efficacy and safety of biologic drugs in Still's disease: a systematic review and network meta-analysis of randomized controlled trialsKilic B, Ozturk A, Karup S et al. · Rheumatology (Oxford, England) · 2025 · PMID 38775654
- Randomized controlled trial Deep molecular profiling of synovial biopsies in the STRAP trial identifies signatures predictive of treatment response to biologic therapies in rheumatoid arthritisLewis MJ, Çubuk C, Surace AEA et al. · Nature communications · 2025 · PMID 40603860
- Cochrane review Cardiovascular safety of the class of JAK inhibitors or tocilizumab compared with TNF inhibitors in patients with rheumatoid arthritis: Systematic review and a traditional and Bayesian network meta-analysis of randomized clinical trialsPugliesi A, Oliveira DGC, Filho VAS et al. · Seminars in arthritis and rheumatism · 2024 · PMID 39383617
- Cochrane review Disease-Modifying Antirheumatic Drugs and Dementia Prevention: A Systematic Review of Observational Evidence in Rheumatoid ArthritisWu CY, Xiong LY, Wong YY et al. · The journal of prevention of Alzheimer's disease · 2024 · PMID 39350379
- Cochrane review Systematic Review and Metaanalysis of Pharmacological Interventions in Adult-Onset Still Disease and the Role of Biologic Disease-Modifying Antirheumatic DrugsRuscitti P, McGonagle D, Garcia VC et al. · The Journal of rheumatology · 2024 · PMID 38302170
- Meta-analysis Efficacy and safety of current therapies for difficult-to-treat rheumatoid arthritis: a systematic review and network meta-analysisSu QY, Luo J, Zhang Y et al. · Journal of translational medicine · 2024 · PMID 39198829
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 Tocilizumab approved for Rheumatoid Arthritis?
Tocilizumab 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 Tocilizumab in clinical trials for Rheumatoid Arthritis?
ClinicalTrials.gov lists 8 registered trials linking Tocilizumab to Rheumatoid Arthritis: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT04251741). The largest enrolment is 241 participants (NCT00144547). Registration activity spans 2004 to 2020.
What does the evidence show for Tocilizumab in Rheumatoid Arthritis?
Tocilizumab 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.