Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Sickle Cell Disease would be drug repurposing rather than first-in-human development. This does not mean it is approved for Sickle Cell Disease. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Sickle Cell Disease: 1 is currently recruiting; 3 are active or not yet recruiting; 1 has completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT03587272). It plans or enrolled 100 participants. Registration activity spans 2011 to 2023. No published literature item is linked to Sirolimus and Sickle Cell Disease 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 Sickle Cell Disease 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: No spontaneous remission; progressive organ damage; median survival historically 40-60 years but improving; vaso-occlusive crises (VOC) in 1-4 per year average.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 40.5 (trials 20.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 3 active / not yet recruiting, 1 completed, 3 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 | ADA |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT02105766 | Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure 2014 | active, not recruiting | Phase 2 | 56 |
| NCT03263559 | Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507) 2017 | completed | Phase 2 | 95 |
| NCT03587272 | Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease 2018 | recruiting | Phase 2 | 100 |
| NCT04362293 | Reduced Intensity Transplantation for Severe Sickle Cell Disease 2020 | suspended | Phase 2 | 40 |
| NCT02061800 | CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant 2013 | active, not recruiting | PHASE1, PHASE2 | 14 |
| NCT01499888 | Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD) 2011 | active, not recruiting | PHASE1, PHASE2 | 45 |
| NCT06078696 | Siplizumab for Sickle Cell Disease Transplant 2023 | terminated | PHASE1, PHASE2 | 1 |
| NCT01850108 | Non-Myeloablative Conditioning and Bone Marrow Transplantation 2013 | status unknown | Not applicable | 26 |
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 ADA. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Sickle Cell Disease?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Sickle Cell Disease would be drug repurposing rather than first-in-human development. This does not mean it is approved for Sickle Cell Disease. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Sickle Cell Disease?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Sickle Cell Disease: 1 is currently recruiting; 3 are active or not yet recruiting; 1 has completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT03587272). It plans or enrolled 100 participants. Registration activity spans 2011 to 2023.
What does the evidence show for Sirolimus in Sickle Cell Disease?
Sirolimus has 1 completed registered trial for Sickle Cell Disease 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 ADA. 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.