Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Melanoma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Melanoma. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Melanoma: 2 are active or not yet recruiting; 3 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT01014351). The largest enrolment is 136 participants (NCT01614301). Registration activity spans 2006 to 2027. The literature layer holds 1 publication for this pair: 1 Cochrane review. The dated items are from 2014. 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 Melanoma ranks 50 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 regression documented in ~0.4% of metastatic melanoma cases; early-stage surgical resection: 90%+ 5-year survival for Stage I; Stage IV (metastatic): historically <10% 5-year survival.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 45.0 (trials 20.0, literature 5.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 2 active / not yet recruiting, 3 completed, 3 other |
| Linked publications | 1 (1 Cochrane review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | AKT1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT01014351 | Study of Everolimus With Paclitaxel and Carboplatin in Patients With Metastatic Melanoma 2010 | completed | Phase 2 | 70 |
| NCT07477548 | A Study to Evaluate the Efficacy and Safety of Everolimus in Patients With Teratment-refractory Vascular Anomalies 2026 | not yet recruiting | Phase 2 | 67 |
| NCT06708455 | Phase 2b of RAPA-201 Cell Therapy in Post-PD-(L)-1 Melanoma 2027 | not yet recruiting | Phase 2 | 65 |
| NCT01166126 | Temsirolimus/AZD 6244 for Treatment-naive With BRAF Mutant Unresectable Stage IV 2010 | terminated | Phase 2 | 4 |
| NCT01960829 | Everolimus in Selected Patients With Metastatic Melanoma: Efficacy and Safety Study 2013 | status unknown | Phase 2 | 60 |
| NCT00375245 | Rapamycin With Grapefruit Juice for Advanced Malignancies 2006 | completed | Phase 1 | 41 |
| NCT01596140 | Vemurafenib in Combination With Everolimus or Temsirolimus With Advanced Cancer 2012 | completed | Phase 1 | 27 |
| NCT01614301 | Phase I and Consecutive Phase II, Two-arm, Randomized Multi-center Trial in Patients With Advanced Melanoma 2012 | status unknown | PHASE1, PHASE2 | 136 |
Published literature
- Cochrane review Effect of sirolimus on malignancy and survival after kidney transplantation: systematic review and meta-analysis of individual patient dataKnoll GA, Kokolo MB, Mallick R et al. · BMJ (Clinical research ed.) · 2014 · PMID 25422259
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 AKT1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Melanoma?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Melanoma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Melanoma. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Melanoma?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Melanoma: 2 are active or not yet recruiting; 3 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT01014351). The largest enrolment is 136 participants (NCT01614301). Registration activity spans 2006 to 2027.
What does the evidence show for Sirolimus in Melanoma?
Sirolimus has both completed registered trials and synthesis-level publications linked to Melanoma. 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 AKT1. 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 2026-07-06. Page built 2026-10-07.