MITOXANTRONE HYDROCHLORIDE
Mitoxantrone hydrochloride is an anthracenedione antineoplastic agent indicated for several distinct conditions. In neurology, it is used to reduce neurologic disability and the frequency of clinical relapses in patients with secondary (chronic) progressive, progressive relapsing, or worsening relapsing-remitting multiple sclerosis; it is not indicated for primary progressive MS. In oncology, mitoxantrone is indicated in combination with corticosteroids for the initial treatment of patients with pain related to advanced hormone-refractory prostate cancer. It is also indicated, in combination with other approved drugs, for the initial therapy of acute nonlymphocytic leukemia (ANLL) in adults, including myelogenous, promyelocytic, monocytic, and erythroid subtypes.
How MITOXANTRONE HYDROCHLORIDE Works
Mitoxantrone is a DNA-reactive agent and type II topoisomerase inhibitor. It intercalates into DNA through hydrogen bonding, resulting in cross-links and strand breaks. It also interferes with the action of topoisomerase II, an enzyme required for DNA uncoiling and repair. These mechanisms collectively inhibit DNA and RNA synthesis, leading to cell death in rapidly dividing cells. In multiple sclerosis, the drug acts as an immunosuppressant by inhibiting the proliferation of T cells, B cells, and macrophages, which reduces the immune-mediated destruction of myelin.
Development Insights
Details
- Status
- Discontinued
- First Approved
- 2006-04-11
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
MITOXANTRONE HYDROCHLORIDE Approval History
What MITOXANTRONE HYDROCHLORIDE Treats
3 indicationsMITOXANTRONE HYDROCHLORIDE is approved for 3 conditions since its original approval in 2006. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Multiple Sclerosis
- Prostate Cancer
- Acute Nonlymphocytic Leukemia
MITOXANTRONE HYDROCHLORIDE Boxed Warning
WARNING Mitoxantrone Injection, USP (concentrate) should be administered under the supervision of a physician experienced in the use of cytotoxic chemotherapy agents. Mitoxantrone Injection, USP (concentrate) should be given slowly into a freely flowing intravenous infusion. It must never be given subcutaneously, intramuscularly, or intra-arterially. Severe local tissue damage may occur if there is extravasation during administration (see ADVERSE REACTIONS, General, Cutaneous and DOSAGE AND ADMI...
WARNING Mitoxantrone Injection, USP (concentrate) should be administered under the supervision of a physician experienced in the use of cytotoxic chemotherapy agents. Mitoxantrone Injection, USP (concentrate) should be given slowly into a freely flowing intravenous infusion. It must never be given subcutaneously, intramuscularly, or intra-arterially. Severe local tissue damage may occur if there is extravasation during administration (see ADVERSE REACTIONS, General, Cutaneous and DOSAGE AND ADMINISTRATION, Preparation and Administration Precautions ). NOT FOR INTRATHECAL USE. Severe injury with permanent sequelae can result from intrathecal administration (see WARNINGS, General ). Except for the treatment of acute nonlymphocytic leukemia, mitoxantrone therapy generally should not be given to patients with baseline neutrophil counts of less than 1,500 cells/mm 3 . In order to monitor the occurrence of bone marrow suppression, primarily neutropenia, which may be severe and result in infection, it is recommended that frequent peripheral blood cell counts be performed on all patients receiving mitoxantrone. Cardiotoxicity: Congestive heart failure (CHF), potentially fatal, may occur either during therapy with mitoxantrone or months to years after termination of therapy. Cardiotoxicity risk increases with cumulative mitoxantrone dose and may occur whether or not cardiac risk factors are present. Presence or history of cardiovascular disease, radiotherapy to the mediastinal/pericardial area, previous therapy with other anthracyclines or anthracenediones, or use of other cardiotoxic drugs may increase this risk. In cancer patients, the risk of symptomatic CHF was estimated to be 2.6% for patients receiving up to a cumulative dose of 140 mg/m 2 . To mitigate the cardiotoxicity risk with mitoxantrone, prescribers should consider the following: All Patients: All patients should be assessed for cardiac signs and symptoms by history, physical examination, and ECG prior to start
MITOXANTRONE HYDROCHLORIDE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in MITOXANTRONE HYDROCHLORIDE's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications MITOXANTRONE HYDROCHLORIDE treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to MITOXANTRONE HYDROCHLORIDE
3 of 20FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
91 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03983824 | NCI-2019-03607 NCI-2019-03607, PHI-103 | Ph 1 | active not recruiting | Testing the Addition of an Anti-cancer Drug, M3814, to the Usual Treatment (Mitoxantrone, Etoposide, and Cytarabine) for Relapsed or Refractory Acute Myeloid Leukemia |
| NCT04293562 | AAML1831 NCI-2020-00546, AAML1831 | Ph 3 | recruiting | A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations |
| NCT03182244 results posted | 2215-CL-0303 CTR20170326 | Ph 3 | active not recruiting | A Study of ASP2215 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase 3 (FLT3) Mutation |
| NCT02101853 results posted | NCI-2014-00631 NCI-2014-00631, s15-00970 | Ph 3 | active not recruiting | Blinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia |
| NCT03591510 | CPKC412A2218 2017-004830-28 | Ph 2 | active not recruiting | A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML |
| NCT03164057 | AML16 NCI-2017-00928 | Ph 2 | active not recruiting | A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT05955261 | AML23 NCI-2023-04138 | Ph 2 | suspended | A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia |
| NCT05848687 | IRB-68271 PEDSHEMALL0015, NCI-2023-04129 | Ph 1, Ph 2 | recruiting | TINI 2: Total Therapy for Infants With Acute Lymphoblastic Leukemia II |
| NCT04797767 | RG1121403 NCI-2021-01379, 10793 | Ph 1, Ph 2 | recruiting | Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms |
| NCT03250338 | ARO-013 2017-001600-29 | Ph 3 | completed | Study Investigating the Efficacy of Crenolanib With Chemotherapy vs Chemotherapy Alone in R/R FLT3 Mutated AML |
| NCT04375631 | RG1006914 NCI-2020-02616, RG1006914 | Ph 1 | recruiting | CLAG-M or FLAG-Ida Chemotherapy and Reduced-Intensity Conditioning Donor Stem Cell Transplant for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia |
| NCT02339740 results posted | AAML1331 NCI-2014-02266, PAAML1331_A01PAMDREVW0 | Ph 3 | active not recruiting | Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia |
| NCT02523976 | IIT2015001 | Ph 2 | completed | Dasatinib Combined With Chemotherapy in Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia |
| NCT03926624 | D18-11141 | Ph 3 | terminated | Trial of DFP-10917 vs Non-Intensive or Intensive Reinduction for AML Patients in 2nd/3rd/4th Salvage |
| NCT06429449 | 24-0178.cc NCI-2024-04761 | Ph 1 | recruiting | Mitoxantrone for Venetoclax Resistant Acute Myeloid Leukemia |
| NCT02553460 results posted | TINI NCI-2015-01493 | Ph 1, Ph 2 | active not recruiting | Total Therapy for Infants With Acute Lymphoblastic Leukemia (ALL) I |
| NCT01371981 results posted | NCI-2011-02670 NCI-2011-02670, CDR0000701850 | Ph 3 | active not recruiting | Bortezomib and Sorafenib Tosylate in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT03118466 results posted | 16-574 | Ph 2 | completed | Mitoxantrone, Etoposide, and Cytarabine (MEC) Plus Lenalidomide for Relapsed or Refractory Acute Myeloid Leukemia |
| NCT03860844 ISAKIDS results posted | ACT15378 PIP - 2018-002697-45, U1111-1202-1096 | Ph 2 | terminated | Isatuximab in Combination With Chemotherapy in Pediatric Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia or Acute Myeloid Leukemia |
| NCT06262438 CHIP-AML22/Q | MH22CAQ 2022-002886-14, 2023-505000-27 | Ph 2 | recruiting | CHIP-AML22/Quizartinib: Quizartinib + Chemotherapy in Newly Diagnosed Pediatric FLT3-ITD+ and NPM1wt AML Patients |
| NCT02521493 results posted | AAML1531 NCI-2015-00324, AAML1531 | Ph 3 | active not recruiting | Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome |
| NCT04195945 results posted | RG1005577 NCI-2019-07640, 10330 | Ph 2 | active not recruiting | CPX-351 or CLAG-M Regimen for the Treatment of Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms in Medically Less-Fit Patients |
| NCT02303821 results posted | CFZ008 2014-001633-84 | Ph 1 | completed | Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia |
| NCT02632708 | AG120-221-C-001 2015-004290-33 | Ph 1 | active not recruiting | Safety Study of AG-120 or AG-221 in Combination With Induction and Consolidation Therapy in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) With an IDH1 and/or IDH2 Mutation |
| NCT04778410 results posted | GS-US-546-5920 2021-003833-13 | Ph 2 | terminated | Study of Magrolimab Combinations in Participants With Myeloid Malignancies |
| NCT02688140 TUD-APOLLO-064 | TUD-APOLLO-064 | Ph 3 | completed | Study for Patients With Newly Diagnosed, High-risk Acute Promyelocytic Leukemia |
| NCT03441048 | PRO00031633 | Ph 1 | completed | Lintuzumab-Ac225 in Combination with Cladribine + Cytarabine + Filgastrim + Mitoxantrone (CLAG-M) for Relapsed/Refractory Acute Myeloid Leukemia |
| NCT02484391 results posted | IRB00033779 NCI-2015-01002, IRB00033779 | Ph 1 | completed | CPI-613, Cytarabine, and Mitoxantrone Hydrochloride in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Granulocytic Sarcoma |
| NCT06007911 | PRO00050186 | Ph 1 | withdrawn | Venetoclax-Navitoclax With Cladribine-based Salvage Therapy in Patients With Relapsed/Refractory Acute Myeloid Leukemia |
| NCT02400281 results posted | ARO-010 | Ph 1, Ph 2 | completed | Study of Crenolanib Combined With Chemotherapy in FLT3-mutated Acute Myeloid Leukemia Patients |
| NCT04196010 | RG1005551 NCI-2019-07696, RG1005551 | Ph 1 | terminated | Continuous Infusion Chemotherapy (CI-CLAM) for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms |
| NCT02626338 results posted | ARO-011 | Ph 1, Ph 2 | completed | Pilot Study of Crenolanib Combined With Standard Salvage Chemotherapy in Subjects With R/R AML |
| NCT02520011 results posted | TPI-ALV-201 | Ph 2 | terminated | Alvocidib Biomarker-driven Phase 2 AML Study |
| NCT02583893 | 15D.377 2013-087, NCI-2015-01507 | Ph 2 | completed | Biomarkers in Predicting Treatment Response to Sirolimus and Chemotherapy in Patients With High-Risk Acute Myeloid Leukemia |
| NCT03531918 results posted | 10000 NCI-2018-00776, 10000 | Ph 1, Ph 2 | completed | Gemtuzumab Ozogamicin With G-CSF, Cladribine, Cytarabine & Mitoxantrone for Untreated AML & High-Grade Myeloid Neoplasm |
| NCT02728050 results posted | 9510 NCI-2016-00286, 9510 | Ph 1, Ph 2 | completed | Filgrastim, Cladribine, Cytarabine, and Mitoxantrone With Sorafenib in Treating Patients With Newly-Diagnosed, Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome |
| NCT02299518 | OSU 14089 NCI-2014-02229 | Ph 1 | completed | Selinexor and Chemotherapy in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia |
| NCT05330377 | MCC-21-LEUK-18-PMC | Ph 1 | withdrawn | GM-CLAG in Relapsed/Refractory FLT3-mutated AML |
| NCT02937285 MITOX-REBIF | 35RC10_8918 2004-001601-10 | Ph 3 | completed | National Multicenter, Controlled, Single-blind Study With Two Parallel Groups Evaluating the Safety and Efficacy of Sequential Treatment With Mitoxantrone and Interferon Versus Interferon Alone in Patients With Strong Risk of Progression in the Initial Phase of Multiple Sclerosis |
| NCT01154439 AML1208 | AML1208 GIMEMA-AML1208, 2008-007666-28 | Ph 1 | completed | Everolimus MICE-regimen in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT01842672 MITCL | NYMC 542 L 10, 819 | Ph 1, Ph 2 | completed | Mitoxantrone and Clofarabine for Treatment of Recurrent NHL or Acute Leukemia |
| NCT00866918 results posted | AAML0631 NCI-2011-01904, CDR0000637184 | Ph 3 | completed | Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Promyelocytic Leukemia |
| NCT01700946 results posted | ALLR18 NCI-2012-00587 | Ph 2 | completed | Therapy for Pediatric Relapsed or Refractory Precursor B-Cell Acute Lymphoblastic Leukemia and Lymphoma |
| NCT02200978 | 2010001 | Ph 4 | completed | A Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia |
| NCT03563560 AML | DC850101 | Ph 1 | completed | A Study of DSP-2033 (Alvocidib) in Patients With Acute Myeloid Leukemia |
| NCT03012672 results posted | 9759 NCI-2016-02051, 9759 | Ph 2 | completed | Higher or Lower Dose Cladribine, Cytarabine, and Mitoxantrone in Treating Medically Less Fit Patients With Newly Diagnosed Acute Myeloid Leukemia or Myeloid Neoplasm |
| NCT02756572 results posted | 9567 NCI-2016-00477, 9567 | Ph 2 | completed | Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms |
| NCT02724163 Myechild01 | RG_14-088 2014-005066-30 | Ph 3 | recruiting | International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia |
| NCT01483690 results posted | T2009-003 | Ph 1, Ph 2 | terminated | A Pilot Study of Decitabine and Vorinostat With Chemotherapy for Relapsed ALL |
| NCT01839240 | 12-0111 NCI-2012-02028 | Ph 1 | completed | Azacitidine, Cytarabine, and Mitoxantrone Hydrochloride in Treating Patients With High-Risk Acute Myeloid Leukemia |
Showing 50 of 91 trials
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
MITOXANTRONE HYDROCHLORIDE FDA Label Details
Indications & Usage
MITOXANTRONE HYDROCHLORIDE is indicated for the treatment of Multiple Sclerosis; Prostate Cancer; Acute Nonlymphocytic Leukemia.
WARNING Mitoxantrone Injection, USP (concentrate) should be administered under the supervision of a physician experienced in the use of cytotoxic chemotherapy agents. Mitoxantrone Injection, USP (concentrate) should be given slowly into a freely flowing intravenous infusion. It must never be given s...
Looking for the branded version?
NOVANTRONE
Full clinical data, patents, trials, and competitive landscape for mitoxantrone hydrochloride.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment