Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Immune Thrombocytopaenia (ITP) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Immune Thrombocytopaenia (ITP). ClinicalTrials.gov lists 4 registered trials linking Sirolimus to Immune Thrombocytopaenia (ITP): 1 is currently recruiting; 3 have completed. The most advanced is PHASE2, PHASE3 (NCT07657793). It plans or enrolled 164 participants. Registration activity spans 2006 to 2026. No published literature item is linked to Sirolimus and Immune Thrombocytopaenia (ITP) 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 Immune Thrombocytopenia 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: Childhood ITP: spontaneous remission in 70-80% within 6 months; Adult ITP: spontaneous remission in 20-30% at 1 year without treatment; chronic ITP (>12 months) rarely resolves spontaneously.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 30.5 (trials 10.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 4 total: 1 recruiting, 0 active / not yet recruiting, 3 completed, 0 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 | ADRM1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT01672151 | Efficacy of Rapamycin Therapy With Chronic Immune Thrombocytopenia 2011 | completed | Not applicable | 35 |
| NCT00392951 | Sirolimus for Autoimmune Disease of Blood Cells 2006 | completed | PHASE1, PHASE2 | 30 |
| NCT01971684 | ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP 2013 | completed | Not applicable | 120 |
| NCT07657793 | Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia 2026 | recruiting | PHASE2, PHASE3 | 164 |
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 ADRM1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Immune Thrombocytopaenia (ITP)?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Immune Thrombocytopaenia (ITP) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Immune Thrombocytopaenia (ITP). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Immune Thrombocytopaenia (ITP)?
ClinicalTrials.gov lists 4 registered trials linking Sirolimus to Immune Thrombocytopaenia (ITP): 1 is currently recruiting; 3 have completed. The most advanced is PHASE2, PHASE3 (NCT07657793). It plans or enrolled 164 participants. Registration activity spans 2006 to 2026.
What does the evidence show for Sirolimus in Immune Thrombocytopaenia (ITP)?
Sirolimus has 3 completed registered trials for Immune Thrombocytopaenia (ITP) 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 ADRM1. 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.