Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Beta Thalassemia would be drug repurposing rather than first-in-human development. This does not mean it is approved for Beta Thalassemia. ClinicalTrials.gov lists 7 registered trials linking Sirolimus to Beta Thalassemia: 4 are active or not yet recruiting; 1 has completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT02105766). The largest enrolment is 130 participants (NCT00061568). Registration activity spans 2004 to 2022. No published literature item is linked to Sirolimus and Beta Thalassemia 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 Beta Thalassemia 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.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 39.5 (trials 19.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 7 total: 0 recruiting, 4 active / not yet recruiting, 1 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 | ABCB4 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
| NCT03877809 | Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients 2019 | completed | Phase 2 | 26 |
| NCT04247750 | Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) 2021 | status unknown | Phase 2 | 45 |
| NCT05357482 | Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia 2022 | active, not recruiting | PHASE1, PHASE2 | 40 |
| NCT00977691 | Haploidentical PBMC Transplant for Severe Congenital Anemias 2009 | active, not recruiting | PHASE1, PHASE2 | 23 |
| NCT02061800 | CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant 2013 | active, not recruiting | PHASE1, PHASE2 | 14 |
| NCT00061568 | Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias 2004 | status unknown | PHASE1, PHASE2 | 130 |
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 ABCB4. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Beta Thalassemia?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Beta Thalassemia would be drug repurposing rather than first-in-human development. This does not mean it is approved for Beta Thalassemia. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Beta Thalassemia?
ClinicalTrials.gov lists 7 registered trials linking Sirolimus to Beta Thalassemia: 4 are active or not yet recruiting; 1 has completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT02105766). The largest enrolment is 130 participants (NCT00061568). Registration activity spans 2004 to 2022.
What does the evidence show for Sirolimus in Beta Thalassemia?
Sirolimus has 1 completed registered trial for Beta Thalassemia 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 ABCB4. 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 2026-07-18. Page built 2026-10-07.