Summary
Daunorubicin 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 Daunorubicin Hydrochloride to Myelodysplastic Syndromes (MDS): 1 is currently recruiting; 2 are active or not yet recruiting; 4 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00046930). It plans or enrolled 449 participants. Registration activity spans 1993 to 2025. No published literature item is linked to Daunorubicin 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 | 49.0 (trials 29.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 2 active / not yet recruiting, 4 completed, 1 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT02521493 | Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome 2015 | active, not recruiting | Phase 3 | 280 |
| NCT00046930 | Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia 2002 | completed | Phase 3 | 449 |
| NCT00002517 | Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome 1993 | completed | Phase 3 | — |
| NCT00002833 | Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia 1994 | completed | Phase 2 | 53 |
| NCT06050941 | Reduced Intensive Idarubicin and Cytarabine Plus Venetoclax as First-line Treatment for Adults AML and MDS 2024 | not yet recruiting | Phase 2 | 60 |
| NCT07046078 | Combination Chemotherapy (FLAG-Ida) Followed Immediately by Reduced-Intensity Total Body Radiation Therapy and Donor Hematopoietic Cell Transplant for the Treatment of Adults Age 60 and Older With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia or Other High-Grade Myeloid Cancer 2025 | recruiting | Phase 2 | 20 |
| NCT01342692 | Best Promising Drug Association With Azacitidine in Higher Risk Myelodysplastic Syndromes 2011 | status unknown | Phase 2 | 320 |
| NCT01831232 | Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes 2013 | completed | Not applicable | 24 |
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 Daunorubicin Hydrochloride approved for Myelodysplastic Syndromes (MDS)?
Daunorubicin 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 Daunorubicin Hydrochloride in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Daunorubicin Hydrochloride to Myelodysplastic Syndromes (MDS): 1 is currently recruiting; 2 are active or not yet recruiting; 4 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00046930). It plans or enrolled 449 participants. Registration activity spans 1993 to 2025.
What does the evidence show for Daunorubicin Hydrochloride in Myelodysplastic Syndromes (MDS)?
Daunorubicin Hydrochloride has 4 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.