Decitabine

drug
On 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

FieldValue
ChEMBL IDCHEMBL1201129
NameDecitabine
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID451668
ChEBICHEBI:50131
ATCL01BC08
Molecular formulaC8H12N4O4
Molecular weight228.21
InChIKeyXAUDJQYHKZQPEU-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):

TargetpChemblTypeValueUnitActivity ID
DNMT17.52IC5030nMCHEMBL_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).

IndicationTrial phaseMONDOEFO
myelodysplastic syndrome4MONDO:0018881EFO:0000198
neoplasm4MONDO:0005070EFO:0000616
chronic myelomonocytic leukemia4MONDO:0020311EFO:1001779
myelodysplastic syndrome with excess blasts4MONDO:0019454EFO:0003811
anemia4MONDO:0002280MONDO:0002280
myeloid leukemia4MONDO:0004643MONDO:0004643
acute myeloid leukemia3MONDO:0018874EFO:0000222
leukemia3MONDO:0005059EFO:0000565
thrombocytopenia3MONDO:0002049HP:0001873
autoimmune thrombocytopenic purpura3MONDO:0008558EFO:0007160
diffuse large B-cell lymphoma3MONDO:0018905EFO:0000403
peripheral T-cell lymphoma, not otherwise specified3MONDO:0004964EFO:0000211
myeloproliferative neoplasm3MONDO:0020076EFO:0002428
mature T-cell and NK-cell non-Hodgkin lymphoma3MONDO:0000430MONDO:0000430
graft versus host disease3MONDO:0013730MONDO:0013730
hematologic disorder3MONDO:0005570HP:0001871
lymphoma2MONDO:0005062EFO:0000574
Hodgkins lymphoma2MONDO:0004952EFO:0000183
chronic myeloid leukemia2MONDO:0011996EFO:0000339
thyroid gland follicular carcinoma2MONDO:0005034EFO:0000501
thyroid gland papillary carcinoma2MONDO:0005075EFO:0000641
renal cell carcinoma2MONDO:0005086EFO:0000681
melanoma2MONDO:0005105EFO:0000756
metastatic melanoma2MONDO:0005191EFO:0002617
non-small cell lung carcinoma2MONDO:0005233EFO:0003060
breast neoplasm2MONDO:0021100EFO:0003869
thalassemia2MONDO:0000984EFO:1001996
peritoneal neoplasm2MONDO:0006901MONDO:0002087
fallopian tube neoplasm2MONDO:0021092MONDO:0002158
ovarian cancer2MONDO:0008170MONDO:0008170
nasal cavity and paranasal sinus lethal midline granuloma2MONDO:0006828MONDO:0019472
severe acute respiratory syndrome2MONDO:0005091MONDO:0100096
aplastic anemia2MONDO:0015909HP:0001915
esophageal squamous cell carcinoma2MONDO:0005580EFO:0005922
sickle cell disease2MONDO:0011382MONDO:0011382
ovarian carcinoma2MONDO:0005140EFO:0001075
fallopian tube carcinoma2MONDO:0006206EFO:1000251
lung neoplasm2MONDO:0021117MONDO:0008903
primary myelofibrosis2MONDO:0009692MONDO:0044903
acute lymphoblastic leukemia1MONDO:0004967EFO:0000220
lymphoid neoplasm1MONDO:0005157EFO:0001642
acute promyelocytic leukemia1MONDO:0012883EFO:0000224
Burkitt lymphoma1MONDO:0007243EFO:0000309
neuroblastoma1MONDO:0005072EFO:0000621
plasma cell myeloma1MONDO:0009693EFO:0001378
primary cutaneous T-cell non-Hodgkin lymphoma1MONDO:0000607EFO:0002913
medulloblastoma1MONDO:0007959EFO:0002939
non-Hodgkin lymphoma1MONDO:0018908EFO:0005952
mantle cell lymphoma1MONDO:0018876EFO:1001469
precursor T-cell acute lymphoblastic leukemia1MONDO:0020512EFO:1001830
metastatic prostate carcinoma1MONDO:0004956EFO:0000196
head and neck cancer1MONDO:0005627EFO:0006859
HIV infectious disease1MONDO:0005109EFO:0000180
prostate carcinoma1MONDO:0005159EFO:0001663
triple-negative breast carcinoma1MONDO:0005494EFO:0005537
head and neck squamous cell carcinoma1MONDO:0010150EFO:0000181
synovial sarcoma1MONDO:0010434EFO:0001376
paraganglioma1MONDO:0000448EFO:1000453
neoplasm of mature B-cells0MONDO:0004949EFO: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

PhaseTrials
PHASE2139
PHASE1109
PHASE1/PHASE266
PHASE328
PHASE2/PHASE318
Not specified10
PHASE48
EARLY_PHASE15

Top trials by phase / activity

NCTPhaseStatusTitle
NCT07101588PHASE4RECRUITINGRuxolitinib-Decitabine Intensified Conditioning Regimen for AML: A Randomized Trial
NCT07555405PHASE4NOT_YET_RECRUITINGEvaluating the Safety and Efficacy of Decitabine in the Treatment of XMEN Patients
NCT01011283PHASE4TERMINATEDTo Demonstrate Superiority of Decitabine Over Azacitidine in Subjects With Intermediate- or High-risk MDS.
NCT01806116PHASE4UNKNOWNDecitabine for Myelodysplastic Syndromes and Acute Myeloid Leukemia Before Allogeneic Hematopoietic Cell Transplantation
NCT02013102PHASE4UNKNOWNA Phase Ⅳ Study of Decitabine in Myelodysplastic Syndrome
NCT03026842PHASE4UNKNOWNDecitabine Versus Conventional Chemotherapy for Maintenance Therapy of Acute Myeloid Leukemia With t(8;21)
NCT03558412PHASE4UNKNOWNA Clinical Trial of Decitabine in Relapsed or Refractory T-lymphoblastic Lymphoma
NCT03579082PHASE4UNKNOWNA Clinical Trial of Decitabine in Relapse and Refractory Diffuse Large B Cell Lymphoma
NCT03793517PHASE2/PHASE3RECRUITINGDecitabine Plus mBU/CY for High Risk Acute Leukemia With MRD Pre-HSCT
NCT04229979PHASE3ACTIVE_NOT_RECRUITINGGalinpepimut-S Versus Investigator’s Choice of Best Available Therapy for Maintenance in AML CR2/CRp2
NCT05177731PHASE3ACTIVE_NOT_RECRUITINGVenetoclax + Decitabine vs. 7+3 Induction Chemotherapy in Young AML
NCT05351346PHASE3RECRUITINGGenotype-guided Treatment in DLBCL
NCT05586074PHASE3RECRUITINGHEC73543 Versus Salvage Chemotherapy in R/R FLT3-ITD AML
NCT05669079PHASE3NOT_YET_RECRUITINGDecitabine and Umbilical Cord Blood for Poor Graft Function Post Allo-HSCT
NCT05883956PHASE3ACTIVE_NOT_RECRUITINGA Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia
NCT05907499PHASE3NOT_YET_RECRUITINGDecitabine for Poor Graft Function Post Allo-HSCT
NCT06297772PHASE3NOT_YET_RECRUITINGCompare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR
NCT06972641PHASE2/PHASE3RECRUITINGMolecular Genetics Guide the Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation
NCT07052422PHASE2/PHASE3NOT_YET_RECRUITINGVEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT
NCT00043134PHASE3UNKNOWNLow-Dose Decitabine Compared With Standard Supportive Care in Treating Older Patients With Myelodysplastic Syndrome
NCT00043381PHASE3COMPLETEDDecitabine Versus Supportive Care in Adults With Advanced-stage MDS
NCT00398983PHASE2/PHASE3COMPLETEDRandomized Study of Decitabine in Maintenance Therapy of Acute Myeloid Leukemia (AML)
NCT01303796PHASE3COMPLETEDA Study of Oral Sapacitabine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT01633099PHASE3UNKNOWNTherapeutic Effect and Safety Study of Decitabine in Elderly Acute Myeloid Leukemia Patients
NCT01809392PHASE2/PHASE3UNKNOWNDecitabine Augments for Post Allogeneic Stem Cell Transplantation in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome
NCT02072811PHASE3UNKNOWNRole of the Therapy Tailored to Risk Factors in Treating Adult Patients (≤60) With Acute Myeloid Leukemia
NCT02085408PHASE3COMPLETEDClofarabine or Daunorubicin Hydrochloride and Cytarabine Followed By Decitabine or Observation in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT02172872PHASE3UNKNOWNInDACtion vs 3+7 Induction in AML
NCT02214407PHASE3COMPLETEDRandomized Phase III Study of Decitabine +/- Hydroxyurea (HY) Versus HY in Advanced Proliferative CMML
NCT02272478PHASE2/PHASE3UNKNOWNTrial to Test the Effects of Adding 1 of 2 New Treatment Agents to Commonly Used Chemotherapy Combinations
NCT02472145PHASE2/PHASE3COMPLETEDAn 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
NCT02487563PHASE3COMPLETEDProspective Study of Patients With Thrombocytopenia Following HSCT
NCT02744742PHASE2/PHASE3COMPLETEDG-CSF+Decitabine+BUCY vs BUCY Conditioning Regimen for RAEB-1, REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT
NCT02785900PHASE3TERMINATEDVadastuximab Talirine (SGN-CD33A; 33A) Combined With Azacitidine or Decitabine in Older Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT02985372PHASE3UNKNOWNDecitabine in Combination With Low-dose Cytarabine in Elderly Patients With Acute Myeloid Leukemia
NCT03092674PHASE2/PHASE3COMPLETEDAzacitidine 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
NCT03252457PHASE3UNKNOWNDecitabine Combining Dexamethasone Versus Dexamethasone in Management of ITP
NCT03306264PHASE3COMPLETEDStudy of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML
NCT03377725PHASE3WITHDRAWNDecitabine and Arsenic Trioxide for Myelodysplastic Syndrome(MDS)
NCT03530085PHASE2/PHASE3UNKNOWNDec+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):

VariantIndicationEffectTherapyLevelCIViC
DNMT3A MutationAcute Myeloid LeukemiaSensitivity/ResponseDecitabineCIViC BEID1587
DNMT1 EXPRESSIONOvarian CancerSensitivity/ResponseDecitabineCIViC DEID819
KRAS MutationOvarian CancerSensitivity/ResponseDecitabineCIViC DEID820

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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