Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Type 1 Diabetes would be drug repurposing rather than first-in-human development. This does not mean it is approved for Type 1 Diabetes. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Type 1 Diabetes: 3 are active or not yet recruiting; 4 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00679042). The largest enrolment is 170 participants (NCT00533442). Registration activity spans 2000 to 2008. The literature layer holds 2 publications for this pair: 2 clinical trial publications. Publication years run from 2000 to 2005. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Type 1 Diabetes Mellitus ranks 324 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: Essentially zero for established T1DM (honeymoon phase occurs in 50-60% after diagnosis but lasts 3-12 months then ends); the disease is permanent autoimmune destruction of beta cells.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 49.0 (trials 25.0, literature 4.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 3 active / not yet recruiting, 4 completed, 1 other |
| Linked publications | 2 (2 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ANG |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00679042 | Islet Transplantation in Type 1 Diabetic Patients Using the University of Illinois at Chicago (UIC) Protocol 2007 | active, not recruiting | Phase 3 | 21 |
| NCT00464555 | Strategies to Improve Islet Survival 2006 | completed | Phase 2 | 5 |
| NCT00533442 | Rapamycin Versus Mycophenolate Mofetil in Kidney-Pancreas Recipients 2000 | completed | Phase 2 | 170 |
| NCT00706420 | Islet Transplantation Alone (ITA) in Patients With Difficult to Control Type I Diabetes Mellitus Using a Glucocorticoid-free Immunosuppressive Regimen 2004 | active, not recruiting | Phase 1 | 17 |
| NCT00708604 | Islet After Kidney Transplantation (IAK) in Patients With Type 1 Diabetes 2008 | completed | Phase 1 | 3 |
| NCT00160732 | Allogenic Islet Cell Transplantation 2003 | active, not recruiting | PHASE1, PHASE2 | 50 |
| NCT00286624 | Anti-Thymocyte Globulin, Cyclosporine, and RAD in Islet Transplantation 2003 | completed | PHASE1, PHASE2 | 6 |
| NCT01309022 | Immunosuppressive Medications for Participants in ITN005CT (NCT00014911) | no_longer_available | Not applicable | — |
Published literature
- Clinical trial publication Islet transplantation in type 1 diabetes mellitus using cultured islets and steroid-free immunosuppression: Miami experienceFroud T, Ricordi C, Baidal DA et al. · American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons · 2005 · PMID 15996257
- Clinical trial publication Islet transplantation in seven patients with type 1 diabetes mellitus using a glucocorticoid-free immunosuppressive regimenShapiro AM, Lakey JR, Ryan EA et al. · The New England journal of medicine · 2000 · PMID 10911004
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 ANG. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Type 1 Diabetes?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Type 1 Diabetes would be drug repurposing rather than first-in-human development. This does not mean it is approved for Type 1 Diabetes. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Type 1 Diabetes?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Type 1 Diabetes: 3 are active or not yet recruiting; 4 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00679042). The largest enrolment is 170 participants (NCT00533442). Registration activity spans 2000 to 2008.
What does the evidence show for Sirolimus in Type 1 Diabetes?
Sirolimus has 4 completed trials and 2 linked publications for Type 1 Diabetes. Completed trials may or may not have posted results; follow the NCT links to check. 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 ANG. 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.