Dexrazoxane

Summary

Dexrazoxane (CHEMBL1738) is an approved small-molecule chelator (ATC V03AF02); indicated across 15 conditions including acute lymphoblastic leukemia and hepatoblastoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: V03AF02
• Indications: 15 conditions
• Clinical trials: 52
• Chemistry: 268.27 Da · C11H16N4O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1738
Name	Dexrazoxane
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	71384
ChEBI	CHEBI:50223
ATC	V03AF02
Molecular formula	C11H16N4O4
Molecular weight	268.27
InChIKey	BMKDZUISNHGIBY-ZETCQYMHSA-N

SMILES: C[C@@H](CN1CC(=O)NC(=O)C1)N2CC(=O)NC(=O)C2

IUPAC name: 4-[(2S)-2-(3,5-dioxopiperazin-1-yl)propyl]piperazine-2,6-dione

Pharmacological roles (ChEBI): chelator, antineoplastic agent, cardiovascular drug, immunosuppressive agent.

Also known as: ADR-529, Cardioxane, Dexrazoxane, Dexrazoxano, ICRF-187, NSC-169780, Razoxane (+)-form, Razoxane, (s)-, Razoxane, d-, dexrazoxane, SID442425

Parent form; salt/anhydrous children: CHEMBL1200778

Patent coverage: 8,149 distinct patent families (34,186 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: DNA topoisomerase 2-alpha, Prostaglandin G/H synthase 1, DNA topoisomerase 2-beta.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 4 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
PTGS1	5.67	AC50	2160	nM	CHEMBL_ACT_25205122
PTGS1	5.22	AC50	5980	nM	CHEMBL_ACT_25206055

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

15 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
acute lymphoblastic leukemia	3	MONDO:0004967	EFO:0000220
hepatoblastoma	3	MONDO:0018666	EFO:1000292
diffuse large B-cell lymphoma	3	MONDO:0018905	EFO:0000403
leukemia	3	MONDO:0005059	EFO:0000565
Ewing sarcoma	2	MONDO:0012817	EFO:0000174
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
rhabdomyosarcoma	2	MONDO:0005212	EFO:0002918
neuroblastoma	2	MONDO:0005072	EFO:0000621
ganglioneuroblastoma	2	MONDO:0005035	EFO:0000502
soft tissue sarcoma	2	MONDO:0018078	EFO:1001968
multiple sclerosis	2	MONDO:0005301	MONDO:0005301
osteosarcoma	1	MONDO:0009807	EFO:0000637
congenital heart disease	1	MONDO:0005453	HP:0030680
neoplasm	1	MONDO:0005070	MONDO:0004992

Clinical trials

Total trials: 52.

Phase distribution

Phase	Trials
PHASE2	18
PHASE3	16
PHASE1	13
PHASE2/PHASE3	2
PHASE1/PHASE2	2
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00980460	PHASE3	ACTIVE_NOT_RECRUITING	Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer
NCT03007147	PHASE3	ACTIVE_NOT_RECRUITING	Imatinib Mesylate and Combination Chemotherapy in Treating Patients With Newly Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
NCT03020030	PHASE3	ACTIVE_NOT_RECRUITING	Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents
NCT03126916	PHASE3	RECRUITING	Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
NCT04293562	PHASE3	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
NCT06220032	PHASE3	RECRUITING	Prevention of Anthracycline-Induced Cardiac Dysfunction With Dexrazoxane in Patients With Diffuse Large-B Cell Lymphoma
NCT06647953	PHASE3	RECRUITING	Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma
NCT00002827	PHASE3	COMPLETED	Chemotherapy Followed by Radiation Therapy in Treating Young Patients With Newly Diagnosed Hodgkin’s Disease
NCT00003937	PHASE3	COMPLETED	Combination Chemotherapy Plus Dexrazoxane in Treating Patients With Newly Diagnosed Nonmetastatic Osteosarcoma
NCT00005578	PHASE3	COMPLETED	Combination Chemotherapy With or Without Dexrazoxane in Treating Children With Hodgkin’s Disease
NCT00016276	PHASE3	TERMINATED	Combination Chemotherapy, Surgery, and Radiation Therapy With or Without Dexrazoxane and Trastuzumab in Treating Women With Stage III or Stage IV Breast Cancer
NCT00165087	PHASE3	TERMINATED	Treatment of Childhood Acute Lymphoblastic Leukemia
NCT00334867	PHASE3	WITHDRAWN	Combination Chemotherapy With or Without Topotecan in Treating Patients With Newly Diagnosed Localized Ewing’s Sarcoma
NCT00400946	PHASE3	COMPLETED	05-001: Treatment of Acute Lymphoblastic Leukemia in Children
NCT00548561	PHASE2/PHASE3	COMPLETED	Efficacy of TopotectTM (Dexrazoxane) for Accidental Extravasation of Anthracyclines
NCT00548704	PHASE2/PHASE3	COMPLETED	A Clinical Trial on Topotect® (Dexrazoxane) in the Treatment of Accidental Extravasation of Anthracyclines
NCT01230983	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia or Advanced Lymphoblastic Non-Hodgkin’s Lymphoma
NCT01231906	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Non-Metastatic Extracranial Ewing Sarcoma
NCT00077285	PHASE2	ACTIVE_NOT_RECRUITING	Irinotecan and Carboplatin as Upfront Window Therapy in Treating Patients With Newly Diagnosed Intermediate-Risk or High-Risk Rhabdomyosarcoma
NCT01864109	PHASE2	ACTIVE_NOT_RECRUITING	Irinotecan and Temozolomide in Combination With Existing High Dose Alkylator Based Chemotherapy for Treatment of Patients With Newly Diagnosed Ewing Sarcoma
NCT01871766	PHASE2	ACTIVE_NOT_RECRUITING	Risk-Adapted Focal Proton Beam Radiation and/or Surgery in Patients With Low, Intermediate and High Risk Rhabdomyosarcoma Receiving Standard or Intensified Chemotherapy
NCT03164057	PHASE2	ACTIVE_NOT_RECRUITING	A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT03589729	PHASE2	RECRUITING	Dexrazoxane Hydrochloride in Preventing Heart-Related Side Effects of Chemotherapy in Participants With Blood Cancers
NCT03786783	PHASE2	ACTIVE_NOT_RECRUITING	Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
NCT06262438	PHASE2	RECRUITING	CHIP-AML22/Quizartinib: Quizartinib + Chemotherapy in Newly Diagnosed Pediatric FLT3-ITD+ and NPM1wt AML Patients
NCT00001335	PHASE2	COMPLETED	New Therapeutic Strategies for Patients With Ewing’s Sarcoma Family of Tumors, High Risk Rhabdomyosarcoma, and Neuroblastoma
NCT00003667	PHASE2	COMPLETED	Combination Chemotherapy and Biological Therapy in Treating Patients With High-Risk Ewing’s Sarcoma
NCT00019864	PHASE2	TERMINATED	Combination Chemotherapy Before and After Surgery in Treating Patients With Osteosarcoma
NCT00038142	PHASE2	TERMINATED	Vincristine, Doxorubicin, Cyclophosphamide and Dexrazoxane (VACdxr) in High Risk Ewing’s Sarcoma Patients
NCT00084838	PHASE2	COMPLETED	Chemotherapy Combined With Radiation Therapy for Newly Diagnosed CNS AT/RT
NCT00098839	PHASE1/PHASE2	COMPLETED	Chemoimmunotherapy With Epratuzumab in Relapsed Acute Lymphoblastic Leukemia (ALL)
NCT00544778	PHASE2	TERMINATED	Combination Chemotherapy and Dexrazoxane Followed by Surgery and Radiation Therapy in Treating Patients With Advanced Soft Tissue Sarcoma or Recurrent Bone Sarcoma
NCT00955890	PHASE2	TERMINATED	Dexrazoxane as a Protective Agent in Anthracycline Treated Breast Cancer
NCT01596088	PHASE1/PHASE2	COMPLETED	A Phase I/II Study of KDX-0811(Dexrazoxane) in the Treatment of Accidental Extravasation of Anthracycline Anti-cancer Agents
NCT02584309	PHASE2	COMPLETED	Doxorubicin With Upfront Dexrazoxane for the Treatment of Advanced or Metastatic Soft Tissue Sarcoma
NCT02953184	PHASE2	UNKNOWN	Marched Pair Study Conventional Doxorubicin(DOX) Versus Pegylated Liposomal Doxorubicin(PLD) Neoadjuvant Chemotherapy
NCT03698227	PHASE2	TERMINATED	OlaReDo - Olaratumab and Rechallenge With Doxorubicin in Soft Tissue Sarcoma Patients
NCT07502521	PHASE2	COMPLETED	Dexrazoxane to Protect Against Hemorrhagic STEMI
NCT05292664	PHASE1	RECRUITING	Venetoclax Basket Trial for High Risk Hematologic Malignancies
NCT00039481	PHASE1	COMPLETED	Oblimersen Plus Combination Chemotherapy and Dexrazoxane in Treating Children and Adolescents With Relapsed or Refractory Solid Tumors

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 1 clinical and 3 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).