Summary
Epirubicin has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. ClinicalTrials.gov lists 8 registered trials linking Epirubicin to Cancer: 1 is currently recruiting; 4 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00003577). It plans or enrolled 2000 participants. Registration activity spans 1996 to 2024. The literature layer holds 15 publications for this pair: 5 Cochrane reviews, 1 meta-analysis, 2 systematic reviews, 2 randomised controlled trial publications and 5 clinical trial publications. Publication years run from 2023 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Cancer ranks 1226 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: Spontaneous regression is rare (<0.5% of cases); 5-year survival without treatment: <5% for advanced NSCLC; ~20% for stage I-II with surgery.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 74.5 (trials 24.5, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 0 active / not yet recruiting, 4 completed, 3 other |
| Linked publications | 15 (5 Cochrane reviews, 5 clinical trial publications, 2 systematic reviews, 2 randomised controlled trial publications, 1 meta-analysis) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ABL1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT02288741 | Tandem Melphalan and Autolog. SCT in MM Patients 60 to 70 Years of Age With and Without Induction Chemotherapy 2001 | completed | Phase 3 | 549 |
| NCT00003577 | Combination Chemotherapy With or Without Epirubicin in Treating Women With Stage I or Stage II Breast Cancer 1996 | completed | Phase 3 | 2,000 |
| NCT01094964 | Hyperthermia and Mitomycin C, Bacillus Calmette-Guerin, or Standard Therapy as Second-Line Therapy in Treating Patients With Recurrent Bladder Cancer 2009 | status unknown | Phase 3 | 242 |
| NCT00966706 | Cisplatin, Capecitabine, Gemcitabine and Epirubicin or Docetaxel for Patients With Stage III or IV Pancreatic Cancer 2005 | completed | Phase 2 | 105 |
| NCT02115152 | Neoadjuvant Trial of Capecitabine for Axillary Lymph Node Positive Operable Breast Cancer 2014 | status unknown | Phase 2 | 100 |
| NCT02067416 | PREDATOR: Neoadjuvant Gene Prediction for Breast Cancer 2012 | withdrawn | Phase 2 | — |
| NCT05535998 | TACE-HAIC Combined With TKIs and Immunotherapy Versus TACE Alone for Hepatocellular Carcinoma With PVTT 2021 | completed | Not applicable | 743 |
| NCT06739265 | Golidocitinib Plus CHOP in Newly Diagnosed PTCL 2024 | recruiting | PHASE1, PHASE2 | 68 |
Published literature
- Clinical trial publication Pragmatic Clinical Trial Assessing Response to Neoadjuvant Docetaxel and Trastuzumab in Nigerian Women With Human Epidermal Growth Factor Receptor 2-Positive Breast Cancer (ARETTA)Ntekim A, Popoola A, Sowunmi A et al. · JCO global oncology · 2026 · PMID 42214048
- Clinical trial publication Durvalumab in Combination With Neoadjuvant Chemotherapy in Early Triple-Negative Breast Cancer: Long-Term Analysis From the GeparNuevo TrialLoibl S, Untch M, Huober J et al. · Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2026 · PMID 42008768
- Clinical trial publication Safety and Efficacy of Epirubicin, Ifosfamide, and Nivolumab as First-Line Treatment for Patients with Undifferentiated Pleomorphic SarcomaMartin-Broto J, Martinez-Trufero J, Diaz-Beveridge R et al. · Clinical cancer research : an official journal of the American Association for Cancer Research · 2026 · PMID 41817407
- Clinical trial publication Surgical Outcomes After Neoadjuvant Pembrolizumab Plus Chemotherapy for Triple-Negative Breast Cancer: Results from the Randomized, Placebo-Controlled Phase 3 KEYNOTE-522 StudyKuemmel S, Fasching PA, Schmid P et al. · Annals of surgical oncology · 2026 · PMID 41739398
- Clinical trial publication Optimal adjuvant intravesical therapy for intermediate risk non-muscle invasive bladder cancer; oncological and patient-reported outcomes of randomized controlled trialElsawy AA, Laymon M, Ezzat O et al. · Urologic oncology · 2026 · PMID 41689868
- Cochrane review Efficacy and safety of active surveillance and chemoablation in the management of non-muscle invasive bladder cancer (NMIBC): Systematic review and pooled analysis by the European Association of Urology-Young Academic Urologists: Urothelial Carcinoma Working GroupSaouli A, Contieri R, Quhal F et al. · Actas urologicas espanolas · 2026 · PMID 41500450
- Randomized controlled trial Adjuvant Epirubicin Plus Cyclophosphamide Followed by Taxanes With or Without Carboplatin in Early-Stage Triple-Negative Breast Cancer (RJBC 1501): A Randomized Phase III TrialChen X, Huang J, Shi H et al. · Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2026 · PMID 41365333
- Cochrane review Comparison of the efficacy and safety of docetaxel plus capecitabine versus docetaxel plus epirubicin for human epidermal growth factor 2 -negative breast cancer: a meta-analysisWu J, Wang Z, Fu Y · BMC women's health · 2025 · PMID 40055784
- Randomized controlled trial Effect of adjuvant carboplatin intensified chemotherapy versus standard chemotherapy on survival in women with high risk, early stage, triple negative breast cancer (CITRINE): randomised, open label phase 3 trialLiu Y, Gong Y, Zhu XZ et al. · BMJ (Clinical research ed.) · 2025 · PMID 41436191
- Systematic review [Effect of taste perception on nutritional status in patients with breast cancer: a systematic review]Rivas-Acosta H, Vedrenne-Gutiérrez F, Ramírez Monroy MF et al. · Nutricion hospitalaria · 2025 · PMID 40504002
- Systematic review An updated systematic review about various effects of microplastics on cancer: A pharmacological and in-silico based analysisBaspakova A, Zare A, Suleimenova R et al. · Molecular aspects of medicine · 2025 · PMID 39756073
- Cochrane review The Omission of Anthracycline Chemotherapy in Women with Early HER2-Negative Breast Cancer-A Systematic Review and Meta-AnalysisGiffoni de Mello Morais Mata D, Rush MB, Smith-Uffen M et al. · Current oncology (Toronto, Ont.) · 2024 · PMID 39195318
- Cochrane review Concurrent epirubicin and trastuzumab use increases complete pathological response rate without additional cardiotoxicity in patients with human epidermal growth factor receptor 2-positive early breast cancer: A meta-regression analysisYang MH, Huang CS, Chang DY et al. · Cancer medicine · 2024 · PMID 39046067
- Cochrane review Comparison of neoadjuvant treatment and surgery first for resectable or borderline resectable pancreatic carcinoma: A systematic review and network meta-analysis of randomized controlled trialsHuan L, Yu F, Cao D et al. · PloS one · 2024 · PMID 38451955
- Meta-analysis The efficacy and safety outcomes of lower dose BCG compared to intravesical chemotherapy in non-muscle-invasive bladder cancer: A network meta-analysisKawada T, Yanagisawa T, Bekku K et al. · Urologic oncology · 2023 · PMID 37137745
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 Open Targets and ChEMBL, in this case via the biomarker or target ABL1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Epirubicin approved for Cancer?
Epirubicin has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Epirubicin in clinical trials for Cancer?
ClinicalTrials.gov lists 8 registered trials linking Epirubicin to Cancer: 1 is currently recruiting; 4 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00003577). It plans or enrolled 2000 participants. Registration activity spans 1996 to 2024.
What does the evidence show for Epirubicin in Cancer?
Epirubicin has both completed registered trials and synthesis-level publications linked to Cancer. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target ABL1. 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 2026-07-05. Page built 2026-10-07.