Arsenic Trioxide

drug
On this page

Also known as Arseneous anhydrideArseneous oxideArsenic oxideArsenic oxide (as4o6)Arsenic sesquioxideArsenic trioxide accordArsenic trioxide medacArsenic trioxide mylanArsenicum albumArsenous oxideDiarsenic trioxideNSC-759274NSC-92859Tetraarsenic hexaoxideTrisenoxWhite arsenicSID26758061SID144205808Arsenic(III) oxide

Summary

Arsenic Trioxide (CHEMBL5483015) is an approved small molecule (ATC L01XX27); indicated across 42 conditions including acute promyelocytic leukemia and neoplasm; with CIViC clinical evidence for 6 variant-indication associations (e.g. PML::RARA Fusion in acute promyelocytic leukemia).

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01XX27
  • Indications: 42 conditions
  • Clinical trials: 111
  • Precision-oncology evidence (CIViC): 6 variant–indication associations

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL5483015
NameArsenic Trioxide
TypeSmall molecule
Max phase4
ATCL01XX27

Also known as: Arseneous anhydride, Arseneous oxide, Arsenic oxide, Arsenic oxide (as4o6), Arsenic sesquioxide, Arsenic trioxide, Arsenic trioxide accord, Arsenic trioxide medac, Arsenic trioxide mylan, Arsenicum album, Arsenous oxide, Diarsenic trioxide

Targets

Targets

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Prelamin-A/C, Somatostatin receptor type 2, Somatostatin receptor type 1.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
P286467.28Kd52.48nMCHEMBL_ACT_2033146
LMNA5.15Potency7080nMCHEMBL_ACT_3642248

Target pathways

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

Indications & clinical

Indications

42 indications (3 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).

IndicationTrial phaseMONDOEFO
acute promyelocytic leukemia4MONDO:0012883EFO:0000224
neoplasm4MONDO:0005070EFO:0000616
leukemia3MONDO:0005059EFO:0000565
acute myeloid leukemia3MONDO:0018874EFO:0000222
myelodysplastic syndrome3MONDO:0018881EFO:0000198
hepatocellular carcinoma2MONDO:0007256EFO:0000182
exocrine pancreatic carcinoma2MONDO:0005192EFO:0002618
small cell lung carcinoma2MONDO:0008433EFO:0000702
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
lymphoma2MONDO:0005062EFO:0000574
plasma cell myeloma2MONDO:0009693EFO:0001378
endometrial carcinoma2MONDO:0002447EFO:1001512
neuroblastoma2MONDO:0005072EFO:0000621
kidney cancer2MONDO:0002367MONDO:0002367
breast neoplasm2MONDO:0021100MONDO:0007254
ureter cancer2MONDO:0008627MONDO:0008627
lung neoplasm2MONDO:0021117MONDO:0008903
systemic lupus erythematosus2MONDO:0007915MONDO:0007915
plasma cell neoplasm2MONDO:0004959EFO:0000200
lung carcinoma2MONDO:0005138EFO:0001071
chronic myeloid leukemia1MONDO:0011996EFO:0000339
gliosarcoma1MONDO:0016681EFO:1001465
essential thrombocythemia1MONDO:0005029EFO:0000479
acquired polycythemia vera1MONDO:0009891EFO:0002429
primary myelofibrosis1MONDO:0009692EFO:0002430
metastatic melanoma1MONDO:0005191EFO:0002617
non-small cell lung carcinoma1MONDO:0005233EFO:0003060
AIDS1MONDO:0012268EFO:0000765
central nervous system neoplasm1MONDO:0006130EFO:1000158
brain cancer1MONDO:0001657MONDO:0001657
basal cell carcinoma1MONDO:0020804MONDO:0005341
colorectal neoplasm1MONDO:0005335MONDO:0005575
glioblastoma1MONDO:0018177MONDO:0020690

9 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 111.

Phase distribution

PhaseTrials
PHASE260
PHASE123
PHASE1/PHASE211
PHASE39
Not specified3
PHASE42
EARLY_PHASE12
PHASE2/PHASE31

Top trials by phase / activity

NCTPhaseStatusTitle
NCT00196768PHASE4UNKNOWNTreatment Protocol for Relapsed Acute Promyelocytic Leukemia (APL) With Arsenic
NCT00504764PHASE4COMPLETEDTreatment of Relapsed Promyelocytic Leukemia With Arsenic Trioxide (ATO)
NCT02339740PHASE3ACTIVE_NOT_RECRUITINGTretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia
NCT00003934PHASE3COMPLETEDTretinoin, Cytarabine, and Daunorubicin Hydrochloride With or Without Arsenic Trioxide Followed by Tretinoin With or Without Mercaptopurine and Methotrexate in Treating Patients With Acute Promyelocytic Leukemia
NCT00378365PHASE3UNKNOWNAcute Promyelocytic Leukemia 2006 (APL)
NCT00454480PHASE2/PHASE3COMPLETEDCombination Chemotherapy With or Without Gemtuzumab Ozogamicin or Tipifarnib in Treating Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes
NCT00482833PHASE3COMPLETEDPhase III Trial in Acute Promyelocytic Leukemia Patients
NCT00513305PHASE3TERMINATEDCytarabine in Combination With Arsenic Trioxide vs. Cytarabine Alone in Elderly Patients With Acute Myeloid Leukemia
NCT00866918PHASE3COMPLETEDCombination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Promyelocytic Leukemia
NCT02688140PHASE3COMPLETEDStudy for Patients With Newly Diagnosed, High-risk Acute Promyelocytic Leukemia
NCT02899169PHASE3UNKNOWNTreatment of Non-high-risk Acute Promyelocytic Leukemia (APL) With Realgar-Indigo Naturalis Formula (RIF)
NCT03377725PHASE3WITHDRAWNDecitabine and Arsenic Trioxide for Myelodysplastic Syndrome(MDS)
NCT01409161PHASE2RECRUITINGTretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia
NCT03031249PHASE1/PHASE2ENROLLING_BY_INVITATIONEfficacy and Safety of ATO Plus ATRA in Nucleophosmin-1 Mutated Acute Myeloid Leukemia
NCT03503864PHASE2ACTIVE_NOT_RECRUITINGPhase II Study of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma
NCT04793919PHASE2RECRUITINGTreatment Study for Children and Adolescents With Acute Promyelocytic Leukemia
NCT06088030PHASE2RECRUITINGArsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer
NCT06778187PHASE2RECRUITINGOral-ATO for TP53-mutated Myeloid Malignancies
NCT06933394PHASE2RECRUITINGArsenic Trioxide With MAPK Inhibitors and Chemotherapy for Stage 4/M Neuroblastoma
NCT00003885PHASE2UNKNOWNArsenic Trioxide in Treating Patients With Recurrent or Refractory Acute Leukemia, Chronic Myeloide Leukemia, Myelodysplasia, Lymphoma, or Myeloma
NCT00004149PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Stage IV Prostate Cancer That Has Not Responded to Previous Hormone Therapy
NCT00005040PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Relapsed or Refractory Non- Hodgkin’s Lymphoma
NCT00005069PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Metastatic Kidney Cancer
NCT00005595PHASE2TERMINATEDArsenic Trioxide in Treating Patients With Relapsed or Refractory Hodgkin’s Disease
NCT00005618PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Relapsed or Refractory Chronic Myelogenous Leukemia
NCT00005795PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Acute Myeloid Leukemia
NCT00005999PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Stage IVB or Recurrent Cervical Cancer
NCT00006008PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia
NCT00006021PHASE1/PHASE2COMPLETEDArsenic Trioxide Plus Vitamin C in Treating Patients With Recurrent or Refractory Multiple Myeloma
NCT00006090PHASE2WITHDRAWNArsenic Trioxide in Treating Patients With Refractory or Relapsed Chronic Lymphocytic Leukemia
NCT00006091PHASE2WITHDRAWNArsenic Trioxide in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia That Has Not Responded to Previous Treatment
NCT00006092PHASE2TERMINATEDArsenic Trioxide for Induction Therapy of Adult Patients With Leukemia
NCT00006220PHASE1/PHASE2TERMINATEDArsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy
NCT00008697PHASE1/PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Refractory or Recurrent Acute Promyelocytic Leukemia
NCT00009867PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Urothelial Cancer
NCT00016159PHASE2COMPLETEDChemotherapy Plus Monoclonal Antibody in Treating Patients With Acute Promyelocytic Leukemia
NCT00017069PHASE2TERMINATEDArsenic Trioxide and Dexamethasone in Treating Patients With Recurrent or Refractory Stage II or Stage III Multiple Myeloma
NCT00017433PHASE2TERMINATEDArsenic Trioxide in Treating Patients With Multiple Myeloma
NCT00020969PHASE2TERMINATEDArsenic Trioxide in Treating Patients With Myelodysplastic Syndromes
NCT00024258PHASE2COMPLETEDArsenic Trioxide in Treating Patients With Advanced Neuroblastoma or Other Childhood Solid Tumors

Clinical evidence (CIViC)

Variant × indication × effect (6 predictive associations from 6 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
PML::RARA FusionAcute Promyelocytic LeukemiaSensitivity/ResponseArsenic Trioxide + TretinoinCIViC AEID1091
PML::RARA Fusion AND PML A216TAcute Promyelocytic LeukemiaResistanceArsenic TrioxideCIViC BEID8179
PML::RARA Fusion AND PML A216VAcute Promyelocytic LeukemiaResistanceArsenic TrioxideCIViC BEID8177
PML::RARA Fusion AND PML S214LAcute Promyelocytic LeukemiaResistanceArsenic TrioxideCIViC BEID8178
MYCN AmplificationMedulloblastomaSensitivity/ResponseArsenic TrioxideCIViC DEID5327
SMO MutationBasal Cell CarcinomaSensitivity/ResponseArsenic Trioxide + PSICIViC DEID747

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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