Summary
Pentostatin 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 1 registered trial linking Pentostatin to Beta Thalassemia: 1 is active or not yet recruiting. The most advanced is Phase 2 (NCT02105766). It plans or enrolled 56 participants. Registered activity dates to 2014. No published literature item is linked to Pentostatin 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 | 24.0 (trials 4.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 0 recruiting, 1 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | inhibitor; inhibitor |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| 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 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
Recorded mechanism or class: inhibitor; inhibitor.
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 Pentostatin approved for Beta Thalassemia?
Pentostatin 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 Pentostatin in clinical trials for Beta Thalassemia?
ClinicalTrials.gov lists 1 registered trial linking Pentostatin to Beta Thalassemia: 1 is active or not yet recruiting. The most advanced is Phase 2 (NCT02105766). It plans or enrolled 56 participants. Registered activity dates to 2014.
What does the evidence show for Pentostatin in Beta Thalassemia?
Trials of Pentostatin in Beta Thalassemia are registered but none has completed, so there is no outcome evidence from those studies yet; the record is a signal of research interest. 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.