Summary
Cyclosporine 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 5 registered trials linking Cyclosporine to Beta Thalassemia: 5 have completed. The most advanced is Phase 4 (NCT04009525). It plans or enrolled 823 participants. Registration activity spans 1999 to 2019. The literature layer holds 2 publications for this pair: 2 clinical trial publications. Publication years run from 2001 to 2003. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
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 | 42.0 (trials 18.0, literature 4.0, tier 15, approved bonus 5.0) |
| Registered trials | 5 total: 0 recruiting, 0 active / not yet recruiting, 5 completed, 0 other |
| Linked publications | 2 (2 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT04009525 | Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study 2019 | completed | Phase 4 | 823 |
| NCT02342145 | Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major 2015 | completed | Phase 4 | 205 |
| NCT00029380 | Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia 1999 | completed | Phase 2 | 30 |
| NCT01917708 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases 2014 | completed | Phase 1 | 10 |
| NCT02435901 | HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity 2008 | completed | PHASE1, PHASE2 | 29 |
Published literature
- Clinical trial publication Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemiaIannone R, Casella JF, Fuchs EJ et al. · Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2003 · PMID 12931121
- Clinical trial publication [Umbilical cord blood transplantation (UCBT) in thalassemia children]Huang S, Fang J, Chen C et al. · Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2001 · PMID 11877069
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 DGIdb, 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 Cyclosporine approved for Beta Thalassemia?
Cyclosporine 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 Cyclosporine in clinical trials for Beta Thalassemia?
ClinicalTrials.gov lists 5 registered trials linking Cyclosporine to Beta Thalassemia: 5 have completed. The most advanced is Phase 4 (NCT04009525). It plans or enrolled 823 participants. Registration activity spans 1999 to 2019.
What does the evidence show for Cyclosporine in Beta Thalassemia?
Cyclosporine has 5 completed trials and 2 linked publications for Beta Thalassemia. 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 DGIdb, 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.