Summary
Fludarabine 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 8 registered trials linking Fludarabine to Beta Thalassemia: 1 is currently recruiting; 2 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03171831). The largest enrolment is 100 participants (NCT01962415). Registration activity spans 2001 to 2017. The literature layer holds 4 publications for this pair: 4 clinical trial publications. Publication years run from 2003 to 2025. 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 | 45.5 (trials 17.5, literature 8.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 0 active / not yet recruiting, 2 completed, 5 other |
| Linked publications | 4 (4 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT03171831 | Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major 2017 | status unknown | Phase 4 | 30 |
| NCT01049854 | CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant 2011 | completed | Phase 2 | 20 |
| NCT01962415 | Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT 2014 | recruiting | Phase 2 | 100 |
| NCT00034528 | Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia 2001 | terminated | Phase 2 | 2 |
| NCT00408447 | Stem Cell Transplant in Sickle Cell Disease and Thalassemia 2004 | status unknown | Phase 2 | 53 |
| NCT02435901 | HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity 2008 | completed | PHASE1, PHASE2 | 29 |
| NCT00578292 | Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO 2004 | terminated | Not applicable | 10 |
| NCT02179359 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies 2014 | terminated | Not applicable | 38 |
Published literature
- Clinical trial publication Early Engraftment and Immune Kinetics Following Allogeneic Transplant Using a Novel Reduced-Toxicity Transplant Strategy in Children/Adolescents with High-Risk Transfusion-Dependent Thalassemia: Early Results of the ThalFAbS TrialRaffa EH, Harris TM, Choed-Amphai C et al. · Transplantation and cellular therapy · 2025 · PMID 39722321
- Clinical trial publication Unrelated Donor Peripheral Blood Stem Cell Transplantation for Patients with β-Thalassemia Major Based on a Novel Conditioning RegimenSun L, Wang N, Chen Y et al. · Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019 · PMID 30951841
- Clinical trial publication [Pretreatment doses of antithymocyte globubin-fresenius for allogeneic hematopoietic stem cell transplantation for beta-thalassemia major]Li C, Wang Y, Wu X et al. · Nan fang yi ke da xue xue bao = Journal of Southern Medical University · 2012 · PMID 22588927
- 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
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 ADA. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Fludarabine approved for Beta Thalassemia?
Fludarabine 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 Fludarabine in clinical trials for Beta Thalassemia?
ClinicalTrials.gov lists 8 registered trials linking Fludarabine to Beta Thalassemia: 1 is currently recruiting; 2 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03171831). The largest enrolment is 100 participants (NCT01962415). Registration activity spans 2001 to 2017.
What does the evidence show for Fludarabine in Beta Thalassemia?
Fludarabine has 2 completed trials and 4 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 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 2026-07-18. Page built 2026-10-07.