Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Systemic Lupus Erythematosus (SLE) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Systemic Lupus Erythematosus (SLE). ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Systemic Lupus Erythematosus (SLE): 2 are currently recruiting; 2 are active or not yet recruiting; 3 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT07077486). The largest enrolment is 1016 participants (NCT01914770). Registration activity spans 2006 to 2026. The literature layer holds 2 publications for this pair: 1 Cochrane review and 1 clinical trial publication. Publication years run from 2018 to 2020. 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 Systemic Lupus Erythematosus ranks 276 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: Spontaneous drug-free remission: 2-10% in some cohorts over 5 years; disease-free survival in 15-20% at 20 years; renal lupus (LN) progression to ESRD in 10-20% within 20 years on standard care.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 52.5 (trials 25.5, literature 7.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 2 recruiting, 2 active / not yet recruiting, 3 completed, 1 other |
| Linked publications | 2 (1 Cochrane review, 1 clinical trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ATRIP |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT07077486 | Effects of Telitacicept vs Cyclophosphamide on Lupus Related Interstitial Lung Disease 2025 | recruiting | Phase 4 | 100 |
| NCT04582136 | Efficacy and Safety of Sirolimus in Active Systemic Lupus Erythematosus 2021 | active, not recruiting | Phase 3 | 146 |
| NCT00779194 | Prospective Study of Rapamycin for the Treatment of SLE 2008 | completed | Phase 2 | 99 |
| NCT04736953 | Sirolimus Treatment Of Patients With SLE 2025 | not yet recruiting | Phase 2 | 220 |
| NCT01914770 | Effectiveness of Belimumab Treatment in a Subpopulation of Systemic Lupus Erythematosus (SLE) Patients: a Pooled Analysis of BLISS-52 and BLISS-76 2009 | completed | Not applicable | 1,016 |
| NCT00392951 | Sirolimus for Autoimmune Disease of Blood Cells 2006 | completed | PHASE1, PHASE2 | 30 |
| NCT07657793 | Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia 2026 | recruiting | PHASE2, PHASE3 | 164 |
| NCT04892212 | Sirolimus in Treatment of Proteinuric Flares of Lupus Nephritis 2021 | status unknown | PHASE2, PHASE3 | 20 |
Published literature
- Cochrane review Efficacy and safety of sirolimus in patients with systemic lupus erythematosus: A systematic review and meta-analysisJi L, Xie W, Zhang Z · Seminars in arthritis and rheumatism · 2020 · PMID 32911286
- Clinical trial publication Sirolimus in patients with clinically active systemic lupus erythematosus resistant to, or intolerant of, conventional medications: a single-arm, open-label, phase 1/2 trialLai ZW, Kelly R, Winans T et al. · Lancet (London, England) · 2018 · PMID 29551338
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, in this case via the biomarker or target ATRIP. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Systemic Lupus Erythematosus (SLE)?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Systemic Lupus Erythematosus (SLE) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Systemic Lupus Erythematosus (SLE). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Systemic Lupus Erythematosus (SLE)?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Systemic Lupus Erythematosus (SLE): 2 are currently recruiting; 2 are active or not yet recruiting; 3 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT07077486). The largest enrolment is 1016 participants (NCT01914770). Registration activity spans 2006 to 2026.
What does the evidence show for Sirolimus in Systemic Lupus Erythematosus (SLE)?
Sirolimus has both completed registered trials and synthesis-level publications linked to Systemic Lupus Erythematosus (SLE). 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, in this case via the biomarker or target ATRIP. 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.