Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Prostate Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Prostate Cancer. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Prostate Cancer: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00887640). The largest enrolment is 61 participants (NCT00085566). Registration activity spans 2000 to 2014. The literature layer holds 4 publications for this pair: 1 meta-analysis and 3 clinical trial publications. Publication years run from 2015 to 2021. 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 Prostate Cancer 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: Low-grade (Gleason 6/Grade Group 1): active surveillance appropriate in 40-50% — many will never need treatment; high-grade: lethal without treatment within 5-10 years.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 51.0 (trials 21.0, literature 10.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 6 completed, 2 other |
| Linked publications | 4 (3 clinical trial publications, 1 meta-analysis) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ACP3 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00012142 | CCI-779 in Treating Patients With Prostate Cancer 2000 | completed | Phase 2 | — |
| NCT02091531 | Dual mTOR Inhibitor MLN0128 in Advanced Castration-Resistant Prostate Cancer (CRPC) Patients 2014 | completed | Phase 2 | 9 |
| NCT00887640 | Impact of Temsirolimus Therapy on Circulating Tumor Cell Biology In Men With Castration Resistant Metastatic Prostate Cancer 2009 | terminated | Phase 2 | 11 |
| NCT00943956 | Everolimus, Bicalutamide, and Leuprolide Acetate in Treating Patients Undergoing Radiation Therapy For High-Risk Locally Advanced Prostate Cancer 2009 | completed | Phase 1 | 30 |
| NCT02125084 | Enzalutamide Plus Everolimus in Men With Metastatic Castrate-Resistant Prostate Cancer 2014 | completed | Phase 1 | 38 |
| NCT00375245 | Rapamycin With Grapefruit Juice for Advanced Malignancies 2006 | completed | Phase 1 | 41 |
| NCT00512668 | Hormone Therapy and Temsirolimus in Treating Patients With Relapsed Prostate Cancer 2007 | terminated | Phase 1 | 24 |
| NCT00085566 | Everolimus and Gefitinib in Treating Patients With Progressive Glioblastoma Multiforme or Progressive Metastatic Prostate Cancer 2004 | completed | PHASE1, PHASE2 | 61 |
Published literature
- Clinical trial publication Phase I Study of Docetaxel and Temsirolimus in Refractory Solid TumorsAmin M, Gao F, Terrero G et al. · American journal of clinical oncology · 2021 · PMID 34310349
- Clinical trial publication Phase II trial of SM-88, a cancer metabolism based therapy, in non-metastatic biochemical recurrent prostate cancerGartrell BA, Roach M 3rd, Retter A et al. · Investigational new drugs · 2021 · PMID 32924093
- Clinical trial publication Everolimus combined with gefitinib in patients with metastatic castration-resistant prostate cancer: Phase 1/2 results and signaling pathway implicationsRathkopf DE, Larson SM, Anand A et al. · Cancer · 2015 · PMID 26178426
- Meta-analysis Sirolimus effects on cancer incidence after kidney transplantation: a meta-analysisYanik EL, Siddiqui K, Engels EA · Cancer medicine · 2015 · PMID 26108799
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 ACP3. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Prostate Cancer?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Prostate Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Prostate Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Prostate Cancer?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Prostate Cancer: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00887640). The largest enrolment is 61 participants (NCT00085566). Registration activity spans 2000 to 2014.
What does the evidence show for Sirolimus in Prostate Cancer?
Sirolimus has both completed registered trials and synthesis-level publications linked to Prostate 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 ACP3. 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.