Arsenic Trioxide
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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
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL5483015 |
| Name | Arsenic Trioxide |
| Type | Small molecule |
| Max phase | 4 |
| ATC | L01XX27 |
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):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| P28646 | 7.28 | Kd | 52.48 | nM | CHEMBL_ACT_2033146 |
| LMNA | 5.15 | Potency | 7080 | nM | CHEMBL_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).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| acute promyelocytic leukemia | 4 | MONDO:0012883 | EFO:0000224 |
| neoplasm | 4 | MONDO:0005070 | EFO:0000616 |
| leukemia | 3 | MONDO:0005059 | EFO:0000565 |
| acute myeloid leukemia | 3 | MONDO:0018874 | EFO:0000222 |
| myelodysplastic syndrome | 3 | MONDO:0018881 | EFO:0000198 |
| hepatocellular carcinoma | 2 | MONDO:0007256 | EFO:0000182 |
| exocrine pancreatic carcinoma | 2 | MONDO:0005192 | EFO:0002618 |
| small cell lung carcinoma | 2 | MONDO:0008433 | EFO:0000702 |
| B-cell chronic lymphocytic leukemia | 2 | MONDO:0004948 | EFO:0000095 |
| lymphoma | 2 | MONDO:0005062 | EFO:0000574 |
| plasma cell myeloma | 2 | MONDO:0009693 | EFO:0001378 |
| endometrial carcinoma | 2 | MONDO:0002447 | EFO:1001512 |
| neuroblastoma | 2 | MONDO:0005072 | EFO:0000621 |
| kidney cancer | 2 | MONDO:0002367 | MONDO:0002367 |
| breast neoplasm | 2 | MONDO:0021100 | MONDO:0007254 |
| ureter cancer | 2 | MONDO:0008627 | MONDO:0008627 |
| lung neoplasm | 2 | MONDO:0021117 | MONDO:0008903 |
| systemic lupus erythematosus | 2 | MONDO:0007915 | MONDO:0007915 |
| plasma cell neoplasm | 2 | MONDO:0004959 | EFO:0000200 |
| lung carcinoma | 2 | MONDO:0005138 | EFO:0001071 |
| chronic myeloid leukemia | 1 | MONDO:0011996 | EFO:0000339 |
| gliosarcoma | 1 | MONDO:0016681 | EFO:1001465 |
| essential thrombocythemia | 1 | MONDO:0005029 | EFO:0000479 |
| acquired polycythemia vera | 1 | MONDO:0009891 | EFO:0002429 |
| primary myelofibrosis | 1 | MONDO:0009692 | EFO:0002430 |
| metastatic melanoma | 1 | MONDO:0005191 | EFO:0002617 |
| non-small cell lung carcinoma | 1 | MONDO:0005233 | EFO:0003060 |
| AIDS | 1 | MONDO:0012268 | EFO:0000765 |
| central nervous system neoplasm | 1 | MONDO:0006130 | EFO:1000158 |
| brain cancer | 1 | MONDO:0001657 | MONDO:0001657 |
| basal cell carcinoma | 1 | MONDO:0020804 | MONDO:0005341 |
| colorectal neoplasm | 1 | MONDO:0005335 | MONDO:0005575 |
| glioblastoma | 1 | MONDO:0018177 | MONDO: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
| Phase | Trials |
|---|---|
| PHASE2 | 60 |
| PHASE1 | 23 |
| PHASE1/PHASE2 | 11 |
| PHASE3 | 9 |
| Not specified | 3 |
| PHASE4 | 2 |
| EARLY_PHASE1 | 2 |
| PHASE2/PHASE3 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00196768 | PHASE4 | UNKNOWN | Treatment Protocol for Relapsed Acute Promyelocytic Leukemia (APL) With Arsenic |
| NCT00504764 | PHASE4 | COMPLETED | Treatment of Relapsed Promyelocytic Leukemia With Arsenic Trioxide (ATO) |
| NCT02339740 | PHASE3 | ACTIVE_NOT_RECRUITING | Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia |
| NCT00003934 | PHASE3 | COMPLETED | Tretinoin, 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 |
| NCT00378365 | PHASE3 | UNKNOWN | Acute Promyelocytic Leukemia 2006 (APL) |
| NCT00454480 | PHASE2/PHASE3 | COMPLETED | Combination Chemotherapy With or Without Gemtuzumab Ozogamicin or Tipifarnib in Treating Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes |
| NCT00482833 | PHASE3 | COMPLETED | Phase III Trial in Acute Promyelocytic Leukemia Patients |
| NCT00513305 | PHASE3 | TERMINATED | Cytarabine in Combination With Arsenic Trioxide vs. Cytarabine Alone in Elderly Patients With Acute Myeloid Leukemia |
| NCT00866918 | PHASE3 | COMPLETED | Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Promyelocytic Leukemia |
| NCT02688140 | PHASE3 | COMPLETED | Study for Patients With Newly Diagnosed, High-risk Acute Promyelocytic Leukemia |
| NCT02899169 | PHASE3 | UNKNOWN | Treatment of Non-high-risk Acute Promyelocytic Leukemia (APL) With Realgar-Indigo Naturalis Formula (RIF) |
| NCT03377725 | PHASE3 | WITHDRAWN | Decitabine and Arsenic Trioxide for Myelodysplastic Syndrome(MDS) |
| NCT01409161 | PHASE2 | RECRUITING | Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia |
| NCT03031249 | PHASE1/PHASE2 | ENROLLING_BY_INVITATION | Efficacy and Safety of ATO Plus ATRA in Nucleophosmin-1 Mutated Acute Myeloid Leukemia |
| NCT03503864 | PHASE2 | ACTIVE_NOT_RECRUITING | Phase II Study of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma |
| NCT04793919 | PHASE2 | RECRUITING | Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia |
| NCT06088030 | PHASE2 | RECRUITING | Arsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer |
| NCT06778187 | PHASE2 | RECRUITING | Oral-ATO for TP53-mutated Myeloid Malignancies |
| NCT06933394 | PHASE2 | RECRUITING | Arsenic Trioxide With MAPK Inhibitors and Chemotherapy for Stage 4/M Neuroblastoma |
| NCT00003885 | PHASE2 | UNKNOWN | Arsenic Trioxide in Treating Patients With Recurrent or Refractory Acute Leukemia, Chronic Myeloide Leukemia, Myelodysplasia, Lymphoma, or Myeloma |
| NCT00004149 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Stage IV Prostate Cancer That Has Not Responded to Previous Hormone Therapy |
| NCT00005040 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Relapsed or Refractory Non- Hodgkin’s Lymphoma |
| NCT00005069 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Metastatic Kidney Cancer |
| NCT00005595 | PHASE2 | TERMINATED | Arsenic Trioxide in Treating Patients With Relapsed or Refractory Hodgkin’s Disease |
| NCT00005618 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Relapsed or Refractory Chronic Myelogenous Leukemia |
| NCT00005795 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Acute Myeloid Leukemia |
| NCT00005999 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Stage IVB or Recurrent Cervical Cancer |
| NCT00006008 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia |
| NCT00006021 | PHASE1/PHASE2 | COMPLETED | Arsenic Trioxide Plus Vitamin C in Treating Patients With Recurrent or Refractory Multiple Myeloma |
| NCT00006090 | PHASE2 | WITHDRAWN | Arsenic Trioxide in Treating Patients With Refractory or Relapsed Chronic Lymphocytic Leukemia |
| NCT00006091 | PHASE2 | WITHDRAWN | Arsenic Trioxide in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia That Has Not Responded to Previous Treatment |
| NCT00006092 | PHASE2 | TERMINATED | Arsenic Trioxide for Induction Therapy of Adult Patients With Leukemia |
| NCT00006220 | PHASE1/PHASE2 | TERMINATED | Arsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy |
| NCT00008697 | PHASE1/PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Refractory or Recurrent Acute Promyelocytic Leukemia |
| NCT00009867 | PHASE2 | COMPLETED | Arsenic Trioxide in Treating Patients With Urothelial Cancer |
| NCT00016159 | PHASE2 | COMPLETED | Chemotherapy Plus Monoclonal Antibody in Treating Patients With Acute Promyelocytic Leukemia |
| NCT00017069 | PHASE2 | TERMINATED | Arsenic Trioxide and Dexamethasone in Treating Patients With Recurrent or Refractory Stage II or Stage III Multiple Myeloma |
| NCT00017433 | PHASE2 | TERMINATED | Arsenic Trioxide in Treating Patients With Multiple Myeloma |
| NCT00020969 | PHASE2 | TERMINATED | Arsenic Trioxide in Treating Patients With Myelodysplastic Syndromes |
| NCT00024258 | PHASE2 | COMPLETED | Arsenic 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):
| Variant | Indication | Effect | Therapy | Level | CIViC |
|---|---|---|---|---|---|
| PML::RARA Fusion | Acute Promyelocytic Leukemia | Sensitivity/Response | Arsenic Trioxide + Tretinoin | CIViC A | EID1091 |
| PML::RARA Fusion AND PML A216T | Acute Promyelocytic Leukemia | Resistance | Arsenic Trioxide | CIViC B | EID8179 |
| PML::RARA Fusion AND PML A216V | Acute Promyelocytic Leukemia | Resistance | Arsenic Trioxide | CIViC B | EID8177 |
| PML::RARA Fusion AND PML S214L | Acute Promyelocytic Leukemia | Resistance | Arsenic Trioxide | CIViC B | EID8178 |
| MYCN Amplification | Medulloblastoma | Sensitivity/Response | Arsenic Trioxide | CIViC D | EID5327 |
| SMO Mutation | Basal Cell Carcinoma | Sensitivity/Response | Arsenic Trioxide + PSI | CIViC D | EID747 |
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).
Related Atlas pages
- Diseases: acute promyelocytic leukemia, neoplasm, leukemia, acute myeloid leukemia, myelodysplastic syndrome, medulloblastoma, skin basal cell carcinoma
- Biomarker genes: MYCN, PML