Disease × agent evidence record

Sirolimus for Cancer: evidence, trials and status

Sirolimus has both completed registered trials and synthesis-level publications linked to Cancer. 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.

8 registered trials 2 recruiting 19 publications Evidence tier A · Strong Score 75.0
Research Tracker › Pairs › Cancer › Sirolimus
Research map, not treatment advice. This page aggregates registry and literature records. It does not evaluate efficacy, dosing or safety for any individual. Discuss any treatment decision with a qualified clinician.

Summary

Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Cancer: 2 are currently recruiting; 4 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT05476939). It plans or enrolled 433 participants. Registration activity spans 2006 to 2022. The literature layer holds 19 publications for this pair: 5 Cochrane reviews, 2 meta-analyses, 3 systematic reviews, 4 randomised controlled trial publications and 5 clinical trial publications. Publication years run from 2021 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 Cancer ranks 1226 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 is rare (<0.5% of cases); 5-year survival without treatment: <5% for advanced NSCLC; ~20% for stage I-II with surgery.

Open in Repurposing ExplorerCancer hubChEMBLOpen Targets

Evidence table

Evidence tierA · Strong
Evidence score75.0 (trials 25.0, literature 30.0, tier 15, approved bonus 5.0)
Registered trials8 total: 2 recruiting, 0 active / not yet recruiting, 4 completed, 2 other
Linked publications19 (5 Cochrane reviews, 5 clinical trial publications, 4 randomised controlled trial publications, 3 systematic reviews, 2 meta-analyses)
Agent typeDrug (Small molecule)
Development stage (any indication)Approved
Mechanism / classNot recorded
Data sourcesOpen Targets, ChEMBL
Linked via biomarker / targetABL1
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 IDTitleStatusPhaseEnrolment
NCT05476939Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0
2022
recruitingPhase 3433
NCT00337545Phase II Trial of RAD001 in Refractory Colorectal Cancer
2006
completedPhase 222
NCT00516412Everolimus in Treating Patients With Relapsed or Refractory Mantle Cell Lymphoma
2007
completedPhase 235
NCT06482086Efficacy of Organoid-Based Drug Screening to Guide Treatment for Locally Advanced Thyroid Cancer
2021
recruitingPhase 275
NCT04591431The Rome Trial From Histology to Target: the Road to Personalize Target Therapy and Immunotherapy
2020
status unknownPhase 2400
NCT00943956Everolimus, Bicalutamide, and Leuprolide Acetate in Treating Patients Undergoing Radiation Therapy For High-Risk Locally Advanced Prostate Cancer
2009
completedPhase 130
NCT01334502Everolimus, Rituximab, and Combination Chemotherapy in Treating Patients With Newly Diagnosed Untreated Diffuse Large B-Cell Lymphoma
2012
completedPhase 126
NCT00671112Everolimus and Bortezomib in Treating Patients With Relapsed or Refractory Lymphoma
2008
terminatedPhase 130

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Published literature

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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 ABL1. The tier describes how well the drug-disease link is documented, not how well the drug works.

Frequently asked questions

Is Sirolimus approved for Cancer?

Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.

Is Sirolimus in clinical trials for Cancer?

ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Cancer: 2 are currently recruiting; 4 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT05476939). It plans or enrolled 433 participants. Registration activity spans 2006 to 2022.

What does the evidence show for Sirolimus in Cancer?

Sirolimus has both completed registered trials and synthesis-level publications linked to Cancer. 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 ABL1. The tier describes how well the drug-disease link is documented, not how well the drug works.

Cite this page

Open Source Medicine Foundation. Sirolimus for Cancer: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-05. https://research.opensourcemed.info/pairs/cancer/sirolimus.html

Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.

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This is a research map, not treatment advice. Evidence tiers and scores summarise what has been studied, not whether a treatment works or is safe for you.