Decitabine
drugOn this page
Also known as DACDacogenDecitabinaDecitabine component of astx-727Decitabine component of astx727Decitabine component of inqoviNSC-127716SID47211573SID56324652SID419300SID144205864SID57260149SID170465126DECITABINE_DACOGENZebularine
Summary
Decitabine (CHEMBL1201129) is an approved small molecule (ATC L01BC08); indicated across 67 conditions including myelodysplastic syndrome and neoplasm; with CIViC clinical evidence for 3 variant-indication associations (e.g. DNMT3A Mutation in acute myeloid leukemia).
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: L01BC08
- Indications: 67 conditions
- Clinical trials: 383
- Precision-oncology evidence (CIViC): 3 variant–indication associations
- Chemistry: 228.21 Da · C8H12N4O4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1201129 |
| Name | Decitabine |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 451668 |
| ChEBI | CHEBI:50131 |
| ATC | L01BC08 |
| Molecular formula | C8H12N4O4 |
| Molecular weight | 228.21 |
| InChIKey | XAUDJQYHKZQPEU-KVQBGUIXSA-N |
SMILES: C1[C@@H]([C@H](O[C@H]1N2C=NC(=NC2=O)N)CO)O
IUPAC name: 4-amino-1-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-1,3,5-triazin-2-one
Also known as: DAC, Dacogen, Decitabina, Decitabine, Decitabine component of astx-727, Decitabine component of astx727, Decitabine component of inqovi, NSC-127716, SID47211573, SID56324652, DECITABINE, SID419300
Patent coverage: 14,797 distinct patent families (58,183 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 58,180 (100%) of the total. 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 (cytosine-5)-methyltransferase 1, Beta-lactamase, Beta-1 adrenergic receptor.
Bioactivity
ChEMBL activities: 1 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 |
|---|---|---|---|---|---|
| DNMT1 | 7.52 | IC50 | 30 | nM | CHEMBL_ACT_18337673 |
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
67 indications (7 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 |
|---|---|---|---|
| myelodysplastic syndrome | 4 | MONDO:0018881 | EFO:0000198 |
| neoplasm | 4 | MONDO:0005070 | EFO:0000616 |
| chronic myelomonocytic leukemia | 4 | MONDO:0020311 | EFO:1001779 |
| myelodysplastic syndrome with excess blasts | 4 | MONDO:0019454 | EFO:0003811 |
| anemia | 4 | MONDO:0002280 | MONDO:0002280 |
| myeloid leukemia | 4 | MONDO:0004643 | MONDO:0004643 |
| acute myeloid leukemia | 3 | MONDO:0018874 | EFO:0000222 |
| leukemia | 3 | MONDO:0005059 | EFO:0000565 |
| thrombocytopenia | 3 | MONDO:0002049 | HP:0001873 |
| autoimmune thrombocytopenic purpura | 3 | MONDO:0008558 | EFO:0007160 |
| diffuse large B-cell lymphoma | 3 | MONDO:0018905 | EFO:0000403 |
| peripheral T-cell lymphoma, not otherwise specified | 3 | MONDO:0004964 | EFO:0000211 |
| myeloproliferative neoplasm | 3 | MONDO:0020076 | EFO:0002428 |
| mature T-cell and NK-cell non-Hodgkin lymphoma | 3 | MONDO:0000430 | MONDO:0000430 |
| graft versus host disease | 3 | MONDO:0013730 | MONDO:0013730 |
| hematologic disorder | 3 | MONDO:0005570 | HP:0001871 |
| lymphoma | 2 | MONDO:0005062 | EFO:0000574 |
| Hodgkins lymphoma | 2 | MONDO:0004952 | EFO:0000183 |
| chronic myeloid leukemia | 2 | MONDO:0011996 | EFO:0000339 |
| thyroid gland follicular carcinoma | 2 | MONDO:0005034 | EFO:0000501 |
| thyroid gland papillary carcinoma | 2 | MONDO:0005075 | EFO:0000641 |
| renal cell carcinoma | 2 | MONDO:0005086 | EFO:0000681 |
| melanoma | 2 | MONDO:0005105 | EFO:0000756 |
| metastatic melanoma | 2 | MONDO:0005191 | EFO:0002617 |
| non-small cell lung carcinoma | 2 | MONDO:0005233 | EFO:0003060 |
| breast neoplasm | 2 | MONDO:0021100 | EFO:0003869 |
| thalassemia | 2 | MONDO:0000984 | EFO:1001996 |
| peritoneal neoplasm | 2 | MONDO:0006901 | MONDO:0002087 |
| fallopian tube neoplasm | 2 | MONDO:0021092 | MONDO:0002158 |
| ovarian cancer | 2 | MONDO:0008170 | MONDO:0008170 |
| nasal cavity and paranasal sinus lethal midline granuloma | 2 | MONDO:0006828 | MONDO:0019472 |
| severe acute respiratory syndrome | 2 | MONDO:0005091 | MONDO:0100096 |
| aplastic anemia | 2 | MONDO:0015909 | HP:0001915 |
| esophageal squamous cell carcinoma | 2 | MONDO:0005580 | EFO:0005922 |
| sickle cell disease | 2 | MONDO:0011382 | MONDO:0011382 |
| ovarian carcinoma | 2 | MONDO:0005140 | EFO:0001075 |
| fallopian tube carcinoma | 2 | MONDO:0006206 | EFO:1000251 |
| lung neoplasm | 2 | MONDO:0021117 | MONDO:0008903 |
| primary myelofibrosis | 2 | MONDO:0009692 | MONDO:0044903 |
| acute lymphoblastic leukemia | 1 | MONDO:0004967 | EFO:0000220 |
| lymphoid neoplasm | 1 | MONDO:0005157 | EFO:0001642 |
| acute promyelocytic leukemia | 1 | MONDO:0012883 | EFO:0000224 |
| Burkitt lymphoma | 1 | MONDO:0007243 | EFO:0000309 |
| neuroblastoma | 1 | MONDO:0005072 | EFO:0000621 |
| plasma cell myeloma | 1 | MONDO:0009693 | EFO:0001378 |
| primary cutaneous T-cell non-Hodgkin lymphoma | 1 | MONDO:0000607 | EFO:0002913 |
| medulloblastoma | 1 | MONDO:0007959 | EFO:0002939 |
| non-Hodgkin lymphoma | 1 | MONDO:0018908 | EFO:0005952 |
| mantle cell lymphoma | 1 | MONDO:0018876 | EFO:1001469 |
| precursor T-cell acute lymphoblastic leukemia | 1 | MONDO:0020512 | EFO:1001830 |
| metastatic prostate carcinoma | 1 | MONDO:0004956 | EFO:0000196 |
| head and neck cancer | 1 | MONDO:0005627 | EFO:0006859 |
| HIV infectious disease | 1 | MONDO:0005109 | EFO:0000180 |
| prostate carcinoma | 1 | MONDO:0005159 | EFO:0001663 |
| triple-negative breast carcinoma | 1 | MONDO:0005494 | EFO:0005537 |
| head and neck squamous cell carcinoma | 1 | MONDO:0010150 | EFO:0000181 |
| synovial sarcoma | 1 | MONDO:0010434 | EFO:0001376 |
| paraganglioma | 1 | MONDO:0000448 | EFO:1000453 |
| neoplasm of mature B-cells | 0 | MONDO:0004949 | EFO:0000096 |
8 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 383.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 139 |
| PHASE1 | 109 |
| PHASE1/PHASE2 | 66 |
| PHASE3 | 28 |
| PHASE2/PHASE3 | 18 |
| Not specified | 10 |
| PHASE4 | 8 |
| EARLY_PHASE1 | 5 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT07101588 | PHASE4 | RECRUITING | Ruxolitinib-Decitabine Intensified Conditioning Regimen for AML: A Randomized Trial |
| NCT07555405 | PHASE4 | NOT_YET_RECRUITING | Evaluating the Safety and Efficacy of Decitabine in the Treatment of XMEN Patients |
| NCT01011283 | PHASE4 | TERMINATED | To Demonstrate Superiority of Decitabine Over Azacitidine in Subjects With Intermediate- or High-risk MDS. |
| NCT01806116 | PHASE4 | UNKNOWN | Decitabine for Myelodysplastic Syndromes and Acute Myeloid Leukemia Before Allogeneic Hematopoietic Cell Transplantation |
| NCT02013102 | PHASE4 | UNKNOWN | A Phase Ⅳ Study of Decitabine in Myelodysplastic Syndrome |
| NCT03026842 | PHASE4 | UNKNOWN | Decitabine Versus Conventional Chemotherapy for Maintenance Therapy of Acute Myeloid Leukemia With t(8;21) |
| NCT03558412 | PHASE4 | UNKNOWN | A Clinical Trial of Decitabine in Relapsed or Refractory T-lymphoblastic Lymphoma |
| NCT03579082 | PHASE4 | UNKNOWN | A Clinical Trial of Decitabine in Relapse and Refractory Diffuse Large B Cell Lymphoma |
| NCT03793517 | PHASE2/PHASE3 | RECRUITING | Decitabine Plus mBU/CY for High Risk Acute Leukemia With MRD Pre-HSCT |
| NCT04229979 | PHASE3 | ACTIVE_NOT_RECRUITING | Galinpepimut-S Versus Investigator’s Choice of Best Available Therapy for Maintenance in AML CR2/CRp2 |
| NCT05177731 | PHASE3 | ACTIVE_NOT_RECRUITING | Venetoclax + Decitabine vs. 7+3 Induction Chemotherapy in Young AML |
| NCT05351346 | PHASE3 | RECRUITING | Genotype-guided Treatment in DLBCL |
| NCT05586074 | PHASE3 | RECRUITING | HEC73543 Versus Salvage Chemotherapy in R/R FLT3-ITD AML |
| NCT05669079 | PHASE3 | NOT_YET_RECRUITING | Decitabine and Umbilical Cord Blood for Poor Graft Function Post Allo-HSCT |
| NCT05883956 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia |
| NCT05907499 | PHASE3 | NOT_YET_RECRUITING | Decitabine for Poor Graft Function Post Allo-HSCT |
| NCT06297772 | PHASE3 | NOT_YET_RECRUITING | Compare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR |
| NCT06972641 | PHASE2/PHASE3 | RECRUITING | Molecular Genetics Guide the Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation |
| NCT07052422 | PHASE2/PHASE3 | NOT_YET_RECRUITING | VEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT |
| NCT00043134 | PHASE3 | UNKNOWN | Low-Dose Decitabine Compared With Standard Supportive Care in Treating Older Patients With Myelodysplastic Syndrome |
| NCT00043381 | PHASE3 | COMPLETED | Decitabine Versus Supportive Care in Adults With Advanced-stage MDS |
| NCT00398983 | PHASE2/PHASE3 | COMPLETED | Randomized Study of Decitabine in Maintenance Therapy of Acute Myeloid Leukemia (AML) |
| NCT01303796 | PHASE3 | COMPLETED | A Study of Oral Sapacitabine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT01633099 | PHASE3 | UNKNOWN | Therapeutic Effect and Safety Study of Decitabine in Elderly Acute Myeloid Leukemia Patients |
| NCT01809392 | PHASE2/PHASE3 | UNKNOWN | Decitabine Augments for Post Allogeneic Stem Cell Transplantation in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome |
| NCT02072811 | PHASE3 | UNKNOWN | Role of the Therapy Tailored to Risk Factors in Treating Adult Patients (≤60) With Acute Myeloid Leukemia |
| NCT02085408 | PHASE3 | COMPLETED | Clofarabine or Daunorubicin Hydrochloride and Cytarabine Followed By Decitabine or Observation in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT02172872 | PHASE3 | UNKNOWN | InDACtion vs 3+7 Induction in AML |
| NCT02214407 | PHASE3 | COMPLETED | Randomized Phase III Study of Decitabine +/- Hydroxyurea (HY) Versus HY in Advanced Proliferative CMML |
| NCT02272478 | PHASE2/PHASE3 | UNKNOWN | Trial to Test the Effects of Adding 1 of 2 New Treatment Agents to Commonly Used Chemotherapy Combinations |
| NCT02472145 | PHASE2/PHASE3 | COMPLETED | An Efficacy and Safety Study of Decitabine (DACOGEN) Plus Talacotuzumab (JNJ-56022473; Anti CD123) Versus Decitabine (DACOGEN) Alone in Participants With Acute Myeloid Leukemia (AML) Ineligible for Intensive Chemotherapy |
| NCT02487563 | PHASE3 | COMPLETED | Prospective Study of Patients With Thrombocytopenia Following HSCT |
| NCT02744742 | PHASE2/PHASE3 | COMPLETED | G-CSF+Decitabine+BUCY vs BUCY Conditioning Regimen for RAEB-1, REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT |
| NCT02785900 | PHASE3 | TERMINATED | Vadastuximab Talirine (SGN-CD33A; 33A) Combined With Azacitidine or Decitabine in Older Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT02985372 | PHASE3 | UNKNOWN | Decitabine in Combination With Low-dose Cytarabine in Elderly Patients With Acute Myeloid Leukemia |
| NCT03092674 | PHASE2/PHASE3 | COMPLETED | Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome |
| NCT03252457 | PHASE3 | UNKNOWN | Decitabine Combining Dexamethasone Versus Dexamethasone in Management of ITP |
| NCT03306264 | PHASE3 | COMPLETED | Study of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML |
| NCT03377725 | PHASE3 | WITHDRAWN | Decitabine and Arsenic Trioxide for Myelodysplastic Syndrome(MDS) |
| NCT03530085 | PHASE2/PHASE3 | UNKNOWN | Dec+Flu+Bu Conditioning Regimen for Elderly AML in CR Undergoing Allo-HSCT |
Clinical evidence (CIViC)
Variant × indication × effect (3 predictive associations from 3 curated evidence items):
| Variant | Indication | Effect | Therapy | Level | CIViC |
|---|---|---|---|---|---|
| DNMT3A Mutation | Acute Myeloid Leukemia | Sensitivity/Response | Decitabine | CIViC B | EID1587 |
| DNMT1 EXPRESSION | Ovarian Cancer | Sensitivity/Response | Decitabine | CIViC D | EID819 |
| KRAS Mutation | Ovarian Cancer | Sensitivity/Response | Decitabine | CIViC D | EID820 |
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).
Related Atlas pages
- Diseases: myelodysplastic syndrome, neoplasm, chronic myelomonocytic leukemia, myelodysplastic syndrome with excess blasts, anemia, myeloid leukemia, acute myeloid leukemia, leukemia, thrombocytopenia, autoimmune thrombocytopenic purpura, diffuse large B-cell lymphoma, peripheral T-cell lymphoma, not otherwise specified, myeloproliferative neoplasm, mature T-cell and NK-cell non-Hodgkin lymphoma, graft versus host disease, hematologic disorder, acute myeloid leukemia by FAB classification, ovarian carcinoma
- Biomarker genes: DNMT1, DNMT3A