Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Bladder Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Bladder Cancer. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Bladder Cancer: 1 is active or not yet recruiting; 5 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT04375813). It plans or enrolled 166 participants. Registration activity spans 2006 to 2021. No published literature item is linked to Sirolimus and Bladder Cancer in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Bladder 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: NMIBC: 50-70% recurrence within 5 years; MIBC: progression to metastatic in 50% without radical treatment; spontaneous remission essentially zero.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 41.0 (trials 21.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 1 active / not yet recruiting, 5 completed, 2 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ALK |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT04375813 | Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention 2021 | active, not recruiting | Phase 2 | 166 |
| NCT01215136 | First-line Everolimus +/- Paclitaxel for Cisplatin-ineligible Patients With Advanced Urothelial Carcinoma 2010 | terminated | Phase 2 | 36 |
| NCT00933374 | Trial to Evaluate Paclitaxel Plus RAD001 in Urothelial Carcinoma 2009 | terminated | Phase 2 | 28 |
| NCT00375245 | Rapamycin With Grapefruit Juice for Advanced Malignancies 2006 | completed | Phase 1 | 41 |
| NCT01522820 | Vaccine Therapy With or Without Sirolimus in Treating Patients With NY-ESO-1 Expressing Solid Tumors 2012 | completed | Phase 1 | 18 |
| NCT02753309 | A Study of Rapamycin Combined With Intravesical BCG in Patients With Non-muscle Invasive Bladder Cancer 2016 | completed | Early Phase 1 | 33 |
| NCT01938573 | Sirolimus, Cisplatin, and Gemcitabine Hydrochloride in Treating Patients With Bladder Cancer 2013 | completed | PHASE1, PHASE2 | 21 |
| NCT01090466 | Gemcitabine Hydrochloride, Cisplatin, and Temsirolimus as First-Line Therapy in Treating Patients With Locally Advanced and/or Metastatic Transitional Cell Cancer of the Urothelium 2008 | completed | PHASE1, PHASE2 | 15 |
Published literature
No publication is linked to this pair yet.
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 ALK. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Bladder Cancer?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Bladder Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Bladder Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Bladder Cancer?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Bladder Cancer: 1 is active or not yet recruiting; 5 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT04375813). It plans or enrolled 166 participants. Registration activity spans 2006 to 2021.
What does the evidence show for Sirolimus in Bladder Cancer?
Sirolimus has 5 completed registered trials for Bladder Cancer but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. 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 ALK. 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.