Summary
Mitoxantrone Hydrochloride has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Breast Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Breast Cancer. ClinicalTrials.gov lists 8 registered trials linking Mitoxantrone Hydrochloride to Breast Cancer: 1 is currently recruiting; 2 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00002544). The largest enrolment is 716 participants (NCT05582499). Registration activity spans 1992 to 2023. The literature layer holds 5 publications for this pair: 5 clinical trial publications. Publication years run from 2001 to 2026. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Breast Cancer ranks 1462 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 documented but rare (<0.5%); 5-year survival stage I: ~99%; stage IV: ~28% overall, improving with modern therapy.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 50.5 (trials 20.5, literature 10.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 0 active / not yet recruiting, 2 completed, 5 other |
| Linked publications | 5 (5 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | BRCA1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00002544 | Mitoxantrone With or Without Docetaxel in Treating Women With Metastatic Breast Cancer 1993 | completed | Phase 3 | 300 |
| NCT00003068 | High-Dose Chemotherapy and Autologous Blood Cell Transplantation in Treating Patients With Primary, Locally Advanced, or Stage IV Breast Cancer 1997 | completed | Phase 2 | 30 |
| NCT05582499 | Fudan University Shanghai Cancer Center Breast Cancer Precision Platform Series Study- Neoadjuvant Therapy 2022 | recruiting | Phase 2 | 716 |
| NCT04927481 | A Study of Mitoxantrone Hydrochloride Liposome Injection in Patients With Advanced HER2 Negative Breast Cancer 2021 | terminated | Phase 2 | 28 |
| NCT02596373 | A Study of Mitoxantrone Hydrochloride Liposome Injection in Advanced Recurrent or Metastatic Breast Cancer 2015 | status unknown | Phase 2 | 60 |
| NCT00002498 | Combination Chemotherapy Compared With Mitoxantrone in Treating Older Patients With Advanced Breast Cancer 1992 | status unknown | Phase 2 | — |
| NCT00010075 | Combination Chemotherapy in Treating Older Women With Metastatic Breast Cancer 2000 | status unknown | Phase 2 | — |
| NCT06156761 | Mitoxantrone Hydrochloride Liposome Combined With Capecitabine in Patients With HER-2 Negative Advanced Breast Cancer 2023 | status unknown | Not applicable | 24 |
Published literature
- Clinical trial publication Mitoxantrone hydrochloride liposome (Lipo-MIT) combined with capecitabine in HER2-negative advanced breast cancer: a dose-escalation, phase I studyLiu J, Jiang M, Zhang M et al. · BMC medicine · 2026 · PMID 42050512
- Clinical trial publication Discovery of fluorescent properties in mitoxantrone hydrochloride injection for tracing: application for sentinel lymph node biopsy in breast cancerLi X, Wu Y, Liang Y et al. · World journal of surgical oncology · 2026 · PMID 41668045
- Clinical trial publication A comparative study of mitoxantrone hydrochloride injection and technetium-99 m for tracing axillary sentinel nodes in patients with breast cancerJiao D, Chen X, Sun X et al. · BMC cancer · 2025 · PMID 39915736
- Clinical trial publication Efficacy and safety of mitoxantrone hydrochloride liposome injection in Chinese patients with advanced breast cancer: a randomized, open-label, active-controlled, single-center, phase II clinical trialWang L, Cao J, Li C et al. · Investigational new drugs · 2022 · PMID 34633576
- Clinical trial publication 5'-deoxy-5-fluorouridine, medroxyprogestrone acetate and mitoxantrone hydrochloride for advanced or recurrent breast cancerSato K, Hiraide H, Tamakuma S et al. · Breast cancer (Tokyo, Japan) · 2001 · PMID 11180767
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 BRCA1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Mitoxantrone Hydrochloride approved for Breast Cancer?
Mitoxantrone Hydrochloride has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Breast Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Breast Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Mitoxantrone Hydrochloride in clinical trials for Breast Cancer?
ClinicalTrials.gov lists 8 registered trials linking Mitoxantrone Hydrochloride to Breast Cancer: 1 is currently recruiting; 2 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00002544). The largest enrolment is 716 participants (NCT05582499). Registration activity spans 1992 to 2023.
What does the evidence show for Mitoxantrone Hydrochloride in Breast Cancer?
Mitoxantrone Hydrochloride has 2 completed trials and 5 linked publications for Breast Cancer. 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 BRCA1. 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.