Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Myelodysplastic Syndromes (MDS): 4 are active or not yet recruiting; 3 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00544115). It plans or enrolled 260 participants. Registration activity spans 2001 to 2021. The literature layer holds 2 publications for this pair: 2 randomised controlled trial publications. Publication years run from 2019 to 2023. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Myelodysplastic Syndrome 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: Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 52.0 (trials 26.0, literature 6.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 4 active / not yet recruiting, 3 completed, 1 other |
| Linked publications | 2 (2 randomised controlled 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 | BCL2 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00544115 | Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders 2001 | active, not recruiting | Phase 2 | 260 |
| NCT01244906 | Post-transplant Cyclophosphamide and Sirolimus Following Reduced Intensity Conditioning (RIC) Transplant 2010 | completed | Phase 2 | 26 |
| NCT00589563 | Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer 2007 | completed | Phase 2 | 32 |
| NCT00105001 | Tacrolimus and Mycophenolate Mofetil With or Without Sirolimus in Preventing Acute Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer 2004 | completed | Phase 2 | 210 |
| NCT01488253 | Sirolimus/Tacrolimus Combination After HLA Matched Related Peripheral Blood Stem Cell Transplants 2012 | terminated | Phase 2 | 3 |
| NCT05088356 | Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft 2021 | active, not recruiting | Phase 1 | 66 |
| NCT00819546 | RAD001 in Combination With PKC412 in Patients With Relapsed, Refractory or Poor Prognosis AML or MDS 2009 | active, not recruiting | Phase 1 | 29 |
| NCT02061800 | CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant 2013 | active, not recruiting | PHASE1, PHASE2 | 14 |
Published literature
- Randomized controlled trial Triple-Drug Graft-versus-Host Disease Prophylaxis after HLA-Matched Unrelated Donor Nonmyeloablative Allogenic Hematopoietic Stem Cell TransplantationWegener A, Andersen NS, Friis LS et al. · Transplantation and cellular therapy · 2023 · PMID 37301257
- Randomized controlled trial Effect of Sirolimus on Immune Reconstitution Following Myeloablative Allogeneic Stem Cell Transplantation: An Ancillary Analysis of a Randomized Controlled Trial Comparing Tacrolimus/Sirolimus and Tacrolimus/Methotrexate (Blood and Marrow Transplant Clinical Trials Network/BMT CTN 0402)Gooptu M, Kim HT, Howard A et al. · Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019 · PMID 31271885
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 BCL2. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Myelodysplastic Syndromes (MDS)?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Myelodysplastic Syndromes (MDS): 4 are active or not yet recruiting; 3 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00544115). It plans or enrolled 260 participants. Registration activity spans 2001 to 2021.
What does the evidence show for Sirolimus in Myelodysplastic Syndromes (MDS)?
Sirolimus has 3 completed trials and 2 linked publications for Myelodysplastic Syndromes (MDS). 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 BCL2. 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.