Summary
Doxorubicin Hydrochloride has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). ClinicalTrials.gov lists 8 registered trials linking Doxorubicin Hydrochloride to Myelodysplastic Syndromes (MDS): 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00074490). It plans or enrolled 442 participants. Registration activity spans 1998 to 2020. No published literature item is linked to Doxorubicin Hydrochloride and Myelodysplastic Syndromes (MDS) 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 Myelodysplastic Syndrome 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: Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 37.5 (trials 17.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 5 completed, 3 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | BLM |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00074490 | Donor Stem Cell Transplant With No or Low-Intensity Chemotherapy Using Sirolimus and Treated Immune Cells to Treat Blood and Lymph Cancers 2004 | terminated | Phase 2 | 442 |
| NCT03059615 | A Phase 2a, Open-Label, Two Stage Study of Nerofe or Nerofe With Doxorubicin in Subjects With AML or MDS 2018 | withdrawn | Phase 2 | — |
| NCT00036790 | Motexafin Gadolinium and Doxorubicin in Treating Patients With Advanced Cancer 2002 | completed | Phase 1 | — |
| NCT00003243 | Combination Chemotherapy Plus Infusion of White Blood Cells in Treating Patients With Hematologic Cancer 1998 | completed | Phase 1 | — |
| NCT03878524 | Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial 2020 | terminated | Phase 1 | 2 |
| NCT00237627 | Doxorubicin Hydrochloride Liposome and Bortezomib in Treating Patients With Refractory Hematologic Cancer or Malignant Solid Tumor or Metastatic Breast Cancer 2001 | completed | PHASE1, PHASE2 | 107 |
| NCT02356159 | Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation 2015 | completed | PHASE1, PHASE2 | 34 |
| NCT00520130 | Chemotherapy and Unrelated Donor Stem Cell Transplantation for Patients With Cancers of the Blood and Immune System 2007 | completed | PHASE1, PHASE2 | 92 |
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 DGIdb, in this case via the biomarker or target BLM. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Doxorubicin Hydrochloride approved for Myelodysplastic Syndromes (MDS)?
Doxorubicin Hydrochloride has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Doxorubicin Hydrochloride in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Doxorubicin Hydrochloride to Myelodysplastic Syndromes (MDS): 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00074490). It plans or enrolled 442 participants. Registration activity spans 1998 to 2020.
What does the evidence show for Doxorubicin Hydrochloride in Myelodysplastic Syndromes (MDS)?
Doxorubicin Hydrochloride has 5 completed registered trials for Myelodysplastic Syndromes (MDS) 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 DGIdb, in this case via the biomarker or target BLM. 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.