Identification

Name
Azacitidine
Accession Number
DB00928  (APRD00809)
Type
Small Molecule
Groups
Approved, Investigational
Description

A pyrimidine nucleoside analogue that inhibits DNA methyltransferase, impairing DNA methylation. It is also an antimetabolite of cytidine, incorporated primarily into RNA. Azacytidine has been used as an antineoplastic agent. [PubChem]

Structure
Thumb
Synonyms
  • 4-Amino-1-beta-D-ribofuranosyl-S-triazin-2(1H)-one
  • 5 AZC
  • 5-Azacytidine
  • Azacitidina
  • Azacitidine
  • Azacitidinum
  • Azacytidine
External IDs
NSC-102816 / U-18496
Product Images
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousActavis Pharma Company2016-09-22Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousBluePoint Laboratories2017-07-01Not applicableUs
Azacitidine for InjectionPowder, for suspension100 mgSubcutaneousDr Reddy's Laboratories2017-10-25Not applicableCanada
VidazaPowder, for suspension100 mgSubcutaneousCelgene2010-01-04Not applicableCanada
VidazaInjection, powder, lyophilized, for suspension100 mg/4mLSubcutaneousPharmion Corporation2006-07-25Not applicableUs
VidazaInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousCelgene Corporation2004-07-05Not applicableUs
Generic Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousDr Reddy's Laboratories2015-06-19Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousMylan Institutional LLC2016-05-23Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousNorthstar RxLLC2018-05-04Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/30mLIntravenous; SubcutaneousFresenius Kabi2017-03-16Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousSandoz2013-09-16Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousDr Reddy's Laboratories2013-09-19Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousCipla USA Inc.2018-05-04Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousSandoz2014-03-27Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/50mLSubcutaneousBreckenridge Pharmaceutical, Inc.2017-06-23Not applicableUs
AzacitidineInjection, powder, lyophilized, for solution100 mg/1Intravenous; SubcutaneousShilpa Medicare Limited2016-12-05Not applicableUs
International/Other Brands
Ladakamycin / Mylosar
Categories
UNII
M801H13NRU
CAS number
320-67-2
Weight
Average: 244.2047
Monoisotopic: 244.080769514
Chemical Formula
C8H12N4O5
InChI Key
NMUSYJAQQFHJEW-KVTDHHQDSA-N
InChI
InChI=1S/C8H12N4O5/c9-7-10-2-12(8(16)11-7)6-5(15)4(14)3(1-13)17-6/h2-6,13-15H,1H2,(H2,9,11,16)/t3-,4-,5-,6-/m1/s1
IUPAC Name
4-amino-1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-1,2-dihydro-1,3,5-triazin-2-one
SMILES
NC1=NC(=O)N(C=N1)[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O

Pharmacology

Indication

For treatment of patients with the following French-American-British myelodysplastic syndrome subtypes: refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts, refractory anemia with excess blasts in transformation (now classified as acute myelogenous leukemia with multilineage dysplasia), and chronic myelomonocytic leukemia.

Associated Conditions
Pharmacodynamics

Azacitidine is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of azacitidine required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The cytotoxic effects of azacitidine cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to azacitidine. Upon uptake into cells, azacitidine is phosphorylated to 5-azacytidine monophosphate by uridine-cytidine kinase, then to diphosphate by pyrimidine monophosphate kinases and triphosphate by diphosphate kinases. 5-Azacitidine triphosphate is incorporated into RNA, leading to the disruption of nuclear and cytoplasmic RNA metabolism and inhibition of protein synthesis. 5-Azacytidine diphosphate is reduced to 5-aza-deoxycytidine diphosphate by ribonucleotide reductase. The resultant metabolite is phosphorylated to 5-azadeoxycitidine triphosphate by nucleoside diphosphate kinases. 5-azadeoxycitidine triphosphate is then incoporated into DNA, leading to inhibition of DNA synthesis. Azacitidine is most toxic during the S-phase of the cell cycle.

Mechanism of action

Azacitidine (5-azacytidine) is a chemical analogue of the cytosine nucleoside used in DNA and RNA. Azacitidine is thought to induce antineoplastic activity via two mechanisms; inhibition of DNA methyltransferase at low doses, causing hypomethylation of DNA, and direct cytotoxicity in abnormal hematopoietic cells in the bone marrow through its incorporation into DNA and RNA at high doses, resulting in cell death. As azacitidine is a ribonucleoside, it incoporates into RNA to a larger extent than into DNA. The incorporation into RNA leads to the dissembly of polyribosomes, defective methylation and acceptor function of transfer RNA, and inhibition of the production of protein. Its incorporation into DNA leads to a covalent binding with DNA methyltransferases, which prevents DNA synthesis and subsequent cytotoxicity.

TargetActionsOrganism
ADNA (cytosine-5)-methyltransferase 1
inhibitor
Human
ARNA
other
Human
ADNA
other
Human
Absorption

Azacitidine is rapidly absorbed after subcutaneous administration. The bioavailability of subcutaneous azacitidine relative to IV azacitidine is approximately 89%, based on area under the curve.

Volume of distribution
  • 76 ± 26 L
Protein binding
Not Available
Metabolism

An in vitro study of azacitidine incubation in human liver fractions indicated that azacitidine may be metabolized by the liver. The potential of azacitidine to inhibit cytochrome P450 (CYP) enzymes is not known.

Route of elimination

Following IV administration of radioactive azacitidine to 5 cancer patients, the cumulative urinary excretion was 85% of the radioactive dose. Fecal excretion accounted for <1% of administered radioactivity over three days. Mean excretion of radioactivity in urine following SC administration of 14C-azacitidine was 50%.

Half life

Mean elimination half-life is approximately 4 hours.

Clearance
  • 167 +/- 49 L/h
Toxicity

One case of overdose with azacitidine was reported during clinical trials. A patient experienced diarrhea, nausea, and vomiting after receiving a single IV dose of approximately 290 mg/m2, almost 4 times the recommended starting dose.

Affected organisms
  • Humans and other mammals
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
DrugInteraction
(R)-warfarinThe risk or severity of bleeding can be increased when (R)-warfarin is combined with Azacitidine.
(S)-WarfarinThe risk or severity of bleeding can be increased when (S)-Warfarin is combined with Azacitidine.
2-MethoxyethanolThe risk or severity of adverse effects can be increased when Azacitidine is combined with 2-Methoxyethanol.
4-hydroxycoumarinThe risk or severity of bleeding can be increased when 4-hydroxycoumarin is combined with Azacitidine.
9-(N-methyl-L-isoleucine)-cyclosporin AThe risk or severity of adverse effects can be increased when Azacitidine is combined with 9-(N-methyl-L-isoleucine)-cyclosporin A.
AbacavirAzacitidine may decrease the excretion rate of Abacavir which could result in a higher serum level.
AbataceptThe risk or severity of adverse effects can be increased when Azacitidine is combined with Abatacept.
AbciximabThe risk or severity of bleeding can be increased when Abciximab is combined with Azacitidine.
AbetimusThe risk or severity of adverse effects can be increased when Azacitidine is combined with Abetimus.
AcarboseAcarbose may decrease the excretion rate of Azacitidine which could result in a higher serum level.
Food Interactions
Not Available

References

Synthesis Reference

Lorenzo DE FERRA, Maurizio ZENONI, Stefano TURCHETTA, Mauro ANIBALDI, Ettore AMMIRATI, Paolo BRANDI, Giorgio BERARDI, "PROCESS FOR THE SYNTHESIS OF AZACITIDINE AND DECITABINE." U.S. Patent US20110245485, issued October 06, 2011.

US20110245485
General References
  1. Cihak A: Biological effects of 5-azacytidine in eukaryotes. Oncology. 1974;30(5):405-22. [PubMed:4142650]
  2. Kaminskas E, Farrell AT, Wang YC, Sridhara R, Pazdur R: FDA drug approval summary: azacitidine (5-azacytidine, Vidaza) for injectable suspension. Oncologist. 2005 Mar;10(3):176-82. [PubMed:15793220]
  3. Leone G, Voso MT, Teofili L, Lubbert M: Inhibitors of DNA methylation in the treatment of hematological malignancies and MDS. Clin Immunol. 2003 Oct;109(1):89-102. [PubMed:14585280]
  4. Ghoshal K, Bai S: DNA methyltransferases as targets for cancer therapy. Drugs Today (Barc). 2007 Jun;43(6):395-422. [PubMed:17612710]
  5. Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF: Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002 May 15;20(10):2429-40. [PubMed:12011120]
  6. Silverman LR: Targeting hypomethylation of DNA to achieve cellular differentiation in myelodysplastic syndromes (MDS). Oncologist. 2001;6 Suppl 5:8-14. [PubMed:11700387]
  7. Issa JP, Kantarjian H: Azacitidine. Nat Rev Drug Discov. 2005 May;Suppl:S6-7. [PubMed:15962522]
  8. O'Dwyer K, Maslak P: Azacitidine and the beginnings of therapeutic epigenetic modulation. Expert Opin Pharmacother. 2008 Aug;9(11):1981-6. doi: 10.1517/14656566.9.11.1981 . [PubMed:18627335]
  9. Siddiqui MA, Scott LJ: Azacitidine: in myelodysplastic syndromes. Drugs. 2005;65(13):1781-9; discussion 1790-1. [PubMed:16114977]
  10. Abdulhaq H, Rossetti JM: The role of azacitidine in the treatment of myelodysplastic syndromes. Expert Opin Investig Drugs. 2007 Dec;16(12):1967-75. [PubMed:18042004]
  11. Keating GM: Azacitidine: a review of its use in higher-risk myelodysplastic syndromes/acute myeloid leukaemia. Drugs. 2009;69(17):2501-18. doi: 10.2165/11202840-000000000-00000. [PubMed:19911860]
  12. Sullivan M, Hahn K, Kolesar JM: Azacitidine: a novel agent for myelodysplastic syndromes. Am J Health Syst Pharm. 2005 Aug 1;62(15):1567-73. [PubMed:16030365]
  13. Dapp MJ, Clouser CL, Patterson S, Mansky LM: 5-Azacytidine can induce lethal mutagenesis in human immunodeficiency virus type 1. J Virol. 2009 Nov;83(22):11950-8. doi: 10.1128/JVI.01406-09. Epub 2009 Sep 2. [PubMed:19726509]
External Links
Human Metabolome Database
HMDB0015063
KEGG Drug
D03021
KEGG Compound
C11262
PubChem Compound
9444
PubChem Substance
46509032
ChemSpider
9072
BindingDB
50424715
ChEBI
2038
ChEMBL
CHEMBL1489
Therapeutic Targets Database
DAP000640
PharmGKB
PA451996
HET
5AE
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
Wikipedia
Azacitidine
ATC Codes
L01BC07 — Azacitidine
AHFS Codes
  • 10:00.00 — Antineoplastic Agents
PDB Entries
4qd3
FDA label
Download (194 KB)
MSDS
Download (68.8 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
0RecruitingTreatmentRecurrent Brain Tumors1
1Active Not RecruitingTreatmentAcute Lymphocytic Leukemia (ALL) / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome1
1Active Not RecruitingTreatmentAdult Acute Megakaryoblastic Leukemia (M7) / Adult Acute Monoblastic Leukemia (M5a) / Adult Acute Monocytic Leukemia (M5b) / Adult Acute Myeloblastic Leukemia With Maturation (M2) / Adult Acute Myeloblastic Leukemia Without Maturation (M1) / Adult Acute Myeloid Leukemia in Remission / Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Del(5q) / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Adult Acute Myelomonocytic Leukemia (M4) / Adult Erythroleukemia (M6a) / Adult Pure Erythroid Leukemia (M6b) / Recurrent Adult Acute Myeloid Leukemia / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Untreated Adult Acute Myeloid Leukemia1
1Active Not RecruitingTreatmentAdult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Promyelocytic Leukemia With PML-RARA / Adult Acute Promyelocytic Leukemia With t(15;17)(q22;q12); PML-RARA / Alkylating Agent-Related Acute Myeloid Leukemia / Recurrent Adult Acute Myeloid Leukemia / Refractory Acute Myeloid Leukemia1
1Active Not RecruitingTreatmentAdult Nasal Type Extranodal NK/T-Cell Lymphoma / Recurrent Nasopharyngeal Keratinizing Squamous Cell Carcinoma / Recurrent Nasopharyngeal Undifferentiated Carcinoma / Stage IV Nasopharyngeal Keratinizing Squamous Cell Carcinoma / Stage IV Nasopharyngeal Keratinizing Squamous Cell Carcinoma AJCC v7 / Stage IV Nasopharyngeal Undifferentiated Carcinoma / Stage IV Nasopharyngeal Undifferentiated Carcinoma AJCC v71
1Active Not RecruitingTreatmentCancer, Breast / Genitourinary / Glioblastoma Multiforme (GBM) / Hematological Neoplasms / Leukemia Acute Myeloid Leukemia (AML) / Leukemias / Lung Cancer Non-Small Cell Cancer (NSCLC) / Lung Cancer Small Cell Lung Cancer (SCLC) / Lymphoma, Hodgkins / Malignant Lymphomas / Melanoma / Metastatic Breast Cancer (MBC) / Multiple Myeloma (MM) / Myelodysplastic Syndromes / Neoplasms / Non-Hodgkin's Lymphoma (NHL) / Renal Cell Adenocarcinoma / Sarcoma, Osteogenic / Sarcomas / Thyroid Cancers1
1Active Not RecruitingTreatmentChronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Essential Thrombocythemia (ET) / Myelodysplastic Syndrome / Myelodysplastic/Myeloproliferative Neoplasms / Polycythemia Vera (PV) / Previously Treated Myelodysplastic Syndromes / Primary Myelofibrosis / Recurrent Adult Acute Myeloid Leukemia / Recurrent Childhood Acute Myeloid Leukemia / Untreated Adult Acute Myeloid Leukemia1
1Active Not RecruitingTreatmentChronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Leukemia Acute Myeloid Leukemia (AML) / Leukemias / Myelodysplastic Syndrome / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Secondary Myelodysplastic Syndromes / Untreated Adult Acute Myeloid Leukemia1
1Active Not RecruitingTreatmentChronic Myelomonocytic Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
1Active Not RecruitingTreatmentEsophageal Cancers / Malignant Neoplasm of Cardio-esophageal Junction of Stomach1
1Active Not RecruitingTreatmentFollicular Lymphoma (FL) / Lymphoma, Large B-Cell, Diffuse (DLBCL)1
1Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes / Smith-Magenis Syndrome1
1Active Not RecruitingTreatmentMyelodysplastic Syndrome1
1Active Not RecruitingTreatmentMyelodysplastic Syndromes1
1CompletedNot AvailableHodgkins Disease (HD) / Leukemia Acute Myeloid Leukemia (AML) / Multiple Myeloma (MM) / Non-Hodgkin's Lymphoma (NHL) / Smith-Magenis Syndrome / Tumors, Solid1
1CompletedNot AvailableMyelodysplastic Syndrome1
1CompletedTreatmentAbdominal wall neoplasm / Fallopian Tube Neoplasms / Lung Cancer Non-Small Cell Cancer (NSCLC) / Neoplasms, Ovarian / Pancreatic Ductal Carcinoma / Transitional Cell Carcinoma / Tumor Virus Infections / Urinary Bladder Neoplasms1
1CompletedTreatmentAccelerated Phase of Disease / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Promyelocytic Leukemia With t(15;17)(q22;q12); PML-RARA / Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative / Blastic Phase / Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Chronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable / Philadelphia Chromosome Negative, BCR-ABL1 Positive Chronic Myelogenous Leukemia / Previously Treated Myelodysplastic Syndromes / Primary Myelofibrosis / Recurrent Adult Acute Lymphoblastic Leukemia / Recurrent Adult Acute Myeloid Leukemia / Recurrent Disease / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Secondary Myelodysplastic Syndromes1
1CompletedTreatmentAcute Lymphoblastic Leukaemias (ALL) / Acute Myelogenous Leukaemia (AML) / B-Cell Chronic Lymphocytic Leukemia / Chronic Chronic myelogenous leukemia / Myelodysplasia1
1CompletedTreatmentAcute Lymphoblastic Leukaemias (ALL) / Acute Myelogenous Leukaemia (AML) / Myelodysplastic Syndrome1
1CompletedTreatmentAcute Lymphocytic Leukemia (ALL) / Leukemia Acute Myeloid Leukemia (AML)1
1CompletedTreatmentAcute Myelogenous Leukaemia (AML)1
1CompletedTreatmentAcute Myelogenous Leukaemia (AML) / Chronic Myelomonocytic Leukemia (CMML) / Myelodysplastic Syndromes (MDS)1
1CompletedTreatmentAcute Myelogenous Leukaemia (AML) / Myelodysplastic Syndrome1
1CompletedTreatmentAdult Nasal Type Extranodal NK/T-Cell Lymphoma / Anaplastic Large Cell Lymphoma / Angioimmunoblastic T-Cell Lymphoma / Hepatosplenic T-Cell Lymphoma / Leukemia, Prolymphocytic / Peripheral T-Cell Lymphoma (PTCL) / Post Transplant Lymphoproliferative Disorder / Recurrent Adult T-Cell Leukemia/Lymphoma / Recurrent Cutaneous T-Cell Non-Hodgkin Lymphoma / Recurrent Mycosis Fungoides/Sezary Syndrome / Small Intestine Lymphoma / T-Cell Large Granular Lymphocyte Leukemia1
1CompletedTreatmentAdult Solid Neoplasm / Hematopoietic and Lymphoid Cell Neoplasm1
1CompletedTreatmentAdvanced Cancers1
1CompletedTreatmentAdvanced Solid Tumor Malignancies1
1CompletedTreatmentAgnogenic Myeloid Metaplasia / Chronic Lymphocytic Leukaemia (CLL) / Chronic Myeloid Leukemia (CML) / Myelodysplastic Syndrome1
1CompletedTreatmentHead and Neck Carcinoma1
1CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemia, Myelomonocytic, Chronic / Malignant Lymphomas / Multiple Myeloma (MM) / Myelodysplastic Syndromes1
1CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemia, Myelomonocytic, Chronic / Myelodysplastic Syndromes1
1CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
1CompletedTreatmentLeukemia, Myelomonocytic, Chronic / Myelodysplastic Syndromes1
1CompletedTreatmentLeukemias / Myelodysplastic Syndrome1
1CompletedTreatmentLeukemias / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Diseases1
1CompletedTreatmentLeukemias / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Neoplasms1
1CompletedTreatmentLung Cancer Non-Small Cell Cancer (NSCLC)1
1CompletedTreatmentLymphoblastic Leukemia, Acute, Childhood / Myelogenous Leukemia, Acute, Childhood1
1CompletedTreatmentMalignancies, Hematologic / Myelodysplastic Syndrome / Tumors, Solid1
1CompletedTreatmentMalignant Lymphomas / Small Intestine Cancer / Unspecified Adult Solid Tumor, Protocol Specific1
1CompletedTreatmentMyelodysplastic Syndromes2
1CompletedTreatmentRecurrent Melanoma / Renal Cell Cancer, Recurrent / Stage III Melanoma / Stage IV Melanoma / Stage IV Renal Cell Cancer1
1CompletedTreatmentRelapsed Or Refractory Multiple Myeloma1
1CompletedTreatmentSmith-Magenis Syndrome1
1CompletedTreatmentTumors, Solid1
1CompletedTreatmentTumors, Solid / Virally Mediated Cancers and Liposarcoma1
1Not Yet RecruitingTreatmentAcute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A / Fibroblast Growth Factor Basic Form Measurement / FLT3 Internal Tandem Duplication / Recurrent Adult Acute Myeloid Leukemia / Refractory Acute Myeloid Leukemia1
1Not Yet RecruitingTreatmentAdult Acute Myeloid Leukemia / Myelodysplastic Syndromes1
1Not Yet RecruitingTreatmentLymphoma, Large B-Cell, Diffuse (DLBCL)1
1RecruitingTreatmentAcute Myelogenous Leukaemia (AML) / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome / Non-Hodgkin's Lymphoma (NHL)1
1RecruitingTreatmentChronic Myeloid Leukemia (CML) / Myelofibroses1
1RecruitingTreatmentChronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Previously Treated Myelodysplastic Syndromes / Refractory Anemia With Excess Blasts in Transformation / Secondary Myelodysplastic Syndromes1
1RecruitingTreatmentColorectal Cancers1
1RecruitingTreatmentEpendymoma, Recurrent1
1RecruitingTreatmentHodgkins Disease (HD) / Lymphoma, Hodgkins / Non-Hodgkin's Lymphoma (NHL)1
1RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)3
1RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome2
1RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
1RecruitingTreatmentMyelodysplastic Syndrome1
1RecruitingTreatmentMyelodysplastic Syndromes (MDS)1
1RecruitingTreatmentMyelodysplastic Syndromes (MDS) / Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)1
1RecruitingTreatmentRecurrent Non-Small Cell Lung Carcinoma / Stage IV Non-Small Cell Lung Cancer1
1SuspendedTreatmentMyelodysplastic Syndromes1
1TerminatedTreatmentAcute Myelogenous Leukaemia (AML) / Myelodysplastic Syndromes1
1TerminatedTreatmentAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Adult Acute Basophilic Leukemia / Adult Acute Eosinophilic Leukemia / Adult Acute Megakaryoblastic Leukemia / Adult Acute Monoblastic Leukemia / Adult Acute Monocytic Leukemia / Adult Acute Myeloid Leukemia in Remission / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With Maturation / Adult Acute Myeloid Leukemia With Minimal Differentiation / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Myeloid Leukemia Without Maturation / Adult Acute Myelomonocytic Leukemia / Adult Erythroleukemia / Adult Pure Erythroid Leukemia / Alkylating Agent-Related Acute Myeloid Leukemia / Recurrent Adult Acute Myeloid Leukemia / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Untreated Adult Acute Myeloid Leukemia1
1TerminatedTreatmentAdvanced Cancers1
1TerminatedTreatmentChronic Myelomonocytic Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Lymphoma, Hodgkins / Multiple Myeloma (MM) / Myelodysplastic Syndromes / Non-Hodgkin's Lymphoma (NHL)1
1TerminatedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
1TerminatedTreatmentLeukemia, Myelomonocytic, Chronic / Myelodysplastic Syndromes1
1TerminatedTreatmentLeukemias / Myelodysplastic Syndromes1
1TerminatedTreatmentLung Cancer Non-Small Cell Cancer (NSCLC) / Squamous Cell Carcinoma of the Head and Neck (SCCHN)1
1TerminatedTreatmentMalignant Neoplasm of Pancreas1
1TerminatedTreatmentMyelodysplastic Syndromes1
1TerminatedTreatmentSquamous Cell Carcinoma (SCC)1
1TerminatedTreatmentStage IA Non-Small Cell Lung Carcinoma / Stage IB Non-Small Cell Lung Carcinoma / Stage IIA Non-Small Cell Lung Carcinoma / Stage IIB Non-Small Cell Lung Carcinoma / Stage IIIA Non-Small Cell Lung Cancer1
1Unknown StatusTreatmentMelanoma (Skin)1
1WithdrawnTreatmentCancer of the Ovary1
1, 2Active Not RecruitingTreatmentAcute erythroid leukemia / Acute Megakaryoblastic Leukemia / Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Adult Acute Megakaryoblastic Leukemia / Adult Acute Monoblastic Leukemia / Adult Acute Monocytic Leukemia / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With Maturation / Adult Acute Myeloid Leukemia With Minimal Differentiation / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Myeloid Leukemia Without Maturation / Adult Acute Myelomonocytic Leukemia / Adult Acute Promyelocytic Leukemia With t(15;17)(q22;q12); PML-RARA / Adult Erythroleukemia / Adult Pure Erythroid Leukemia / Chronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts / Myelodysplastic Syndrome With Ring Sideroblasts / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia / Refractory Anemia / Refractory Anemia With Excess Blasts / Refractory Anemia With Excess Blasts in Transformation / Refractory Anemia With Ring Sideroblasts / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Secondary Myelodysplastic Syndromes / Untreated Adult Acute Myeloid Leukemia1
1, 2Active Not RecruitingTreatmentAcute Myelogenous Leukaemia (AML) / Myelogenous Leukemia / Treatment Naive AML1
1, 2Active Not RecruitingTreatmentAdvanced Malignancies / Advanced Solid Tumors / Cancer, Advanced / Metastatic Cancers / Tumors, Solid1
1, 2Active Not RecruitingTreatmentAdvanced Malignancies / Cancer, Advanced / Metastatic Cancers / Tumors, Solid1
1, 2Active Not RecruitingTreatmentCancer, Advanced / Solid Tumors and Hematologic Malignancy / Tumors, Solid1
1, 2Active Not RecruitingTreatmentHigh Risk Myelodysplastic Syndrome / Leukemia Acute Myeloid Leukemia (AML) / Leukemias1
1, 2Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)3
1, 2Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes / Precursor Cell Lymphoblastic Leukemia-Lymphoma1
1, 2Active Not RecruitingTreatmentLeukemias1
1, 2Active Not RecruitingTreatmentMyelodysplastic Syndrome1
1, 2Active Not RecruitingTreatmentRefractory Multiple Myeloma1
1, 2Active Not RecruitingTreatmentSmith-Magenis Syndrome1
1, 2Active Not RecruitingTreatmentUntreated Adult Acute Myeloid Leukemia1
1, 2CompletedTreatmentAcute Myelogenous Leukaemia (AML) / Leukemias / Myelodysplastic Syndrome1
1, 2CompletedTreatmentAdult Acute Megakaryoblastic Leukemia (M7) / Adult Acute Minimally Differentiated Myeloid Leukemia (M0) / Adult Acute Monoblastic Leukemia (M5a) / Adult Acute Monocytic Leukemia (M5b) / Adult Acute Myeloblastic Leukemia With Maturation (M2) / Adult Acute Myeloblastic Leukemia Without Maturation (M1) / Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Adult Acute Myelomonocytic Leukemia (M4) / Adult Erythroleukemia (M6a) / Adult Pure Erythroid Leukemia (M6b) / Recurrent Adult Acute Myeloid Leukemia1
1, 2CompletedTreatmentAdult Acute Myeloblastic Leukemia / Leukemia Acute Myeloid Leukemia (AML)1
1, 2CompletedTreatmentAdvanced Cancers / Malignant Lymphomas1
1, 2CompletedTreatmentAdvanced or Metastatic Breast Cancer / Advanced or Metastatic Solid Tumors1
1, 2CompletedTreatmentColorectal Cancers1
1, 2CompletedTreatmentDe Novo Myelodysplastic Syndromes / Previously Treated Myelodysplastic Syndromes / Secondary Myelodysplastic Syndromes1
1, 2CompletedTreatmentHigh Grade Myelodysplastic Syndrome Lesions1
1, 2CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
1, 2CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
1, 2CompletedTreatmentLeukemias3
1, 2CompletedTreatmentLeukemias / Myelodysplastic Syndromes1
1, 2CompletedTreatmentLymphoma, Large B-Cell, Diffuse (DLBCL)1
1, 2CompletedTreatmentMalignant Lymphomas1
1, 2CompletedTreatmentMyelodysplastic Syndrome1
1, 2CompletedTreatmentRecurrent Non-Small Cell Lung Carcinoma / Stage IIIA Non-Small Cell Lung Cancer / Stage IIIb Non-small Cell Lung Cancer / Stage IV Non-Small Cell Lung Cancer1
1, 2Not Yet RecruitingTreatmentAcute Myeloid Leukemia With t(9;11)(p21.3;q23.3); MLLT3-KMT2A / Acute, recurrent Myeloid Leukemia / Refractory Acute Myeloid Leukemia1
1, 2Not Yet RecruitingTreatmentOsteosarcoma in Children / Recurrent Osteosarcoma / Sarcoma, Osteogenic / Sarcomas1
1, 2RecruitingTreatmentAcute Myelogenous Leukaemia (AML) / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome1
1, 2RecruitingTreatmentAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Acute, recurrent Myeloid Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Refractory Acute Myeloid Leukemia / Relapsed Acute Myeloid Leukemia1
1, 2RecruitingTreatmentAcute Myeloid Leukemia With Gene Mutations / Chronic Myelomonocytic Leukemia / Myelodysplastic Syndrome With Gene Mutation / Myeloproliferative Neoplasms1
1, 2RecruitingTreatmentCancer, Advanced / Solid Tumors and Hematologic Malignancy1
1, 2RecruitingTreatmentCancer, Advanced / Solid Tumors and Hematologic Malignancy / Tumors, Solid1
1, 2RecruitingTreatmentChronic Myelomonocytic Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome1
1, 2RecruitingTreatmentChronic Myelomonocytic Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome / Myeloproliferative Neoplasms1
1, 2RecruitingTreatmentCohort 1a and 1b: Glioblastoma Multiforme / Cohort 1a and 1b: Glioma / Cohort 2a and 2b: Hepatobiliary Tumors (Hepatocellular Carcinoma, Bile Duct Carcinoma, Intrahepatic Cholangiocarcinoma, Other Hepatobiliary Carcinomas) / Cohort 3a and 3b: Chondrosarcoma / Cohort 4a and 4b: Intrahepatic Cholangiocarcinoma / Cohort 5a: Other Solid Tumors With IDH1 Mutations1
1, 2RecruitingTreatmentAcute, recurrent Myeloid Leukemia / FLT3 Gene Mutation / Leukemias / Recurrent Chronic Myelomonocytic Leukemia / Recurrent Myelodysplastic Syndrome / Refractory Acute Myeloid Leukemia / Refractory Chronic Myelomonocytic Leukemia / Refractory Myelodysplastic Syndrome1
1, 2RecruitingTreatmentHead and Neck Carcinoma1
1, 2RecruitingTreatmentHigh Grade Myelodysplastic Syndromes1
1, 2RecruitingTreatmentHigh-risk Myelodysplastic Syndrome / Leukemia Acute Myeloid Leukemia (AML)1
1, 2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Minimal Residual Disease1
1, 2RecruitingTreatmentMyelodysplastic Syndromes1
1, 2RecruitingTreatmentMyelodysplastic Syndrome / Other Diseases of Blood and Blood-Forming Organs1
1, 2RecruitingTreatmentPreviously Untreated Acute Myeloid Leukemia1
1, 2RecruitingTreatmentT-Cell Lymphomas1
1, 2TerminatedTreatmentLeukemias1
1, 2TerminatedTreatmentMyelodysplastic Syndrome1
1, 2TerminatedTreatmentPain NOS / Prostate Cancer1
1, 2Unknown StatusTreatmentRenal Cell Adenocarcinoma1
1, 2WithdrawnTreatmentFLT3-Mutated Acute Myeloid Leukemia / FLT3-Mutated High-Risk Myelodysplastic Syndrome / Leukemias1
1, 2WithdrawnTreatmentLeukemia Acute Myeloid Leukemia (AML)1
1, 2WithdrawnTreatmentMalignant Lymphomas1
1, 2WithdrawnTreatmentMetastatic Colorectal Cancers1
1, 2WithdrawnTreatmentMyelodysplastic Syndromes2
2Active Not RecruitingTreatmentAcute Lymphocytic Leukemia (ALL) / Acute Myelogenous Leukaemia (AML) / Juvenile Myelomonocytic Leukemia / Myelodysplastic Syndrome1
2Active Not RecruitingTreatmentAcute Myelogenous Leukaemia (AML) / Myelodysplastic Syndrome1
2Active Not RecruitingTreatmentAdult Acute Megakaryoblastic Leukemia / Adult Acute Monoblastic Leukemia / Adult Acute Monocytic Leukemia / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With Maturation / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Myeloid Leukemia Without Maturation / Adult Acute Myelomonocytic Leukemia / Adult Erythroleukemia / Adult Pure Erythroid Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Untreated Adult Acute Myeloid Leukemia1
2Active Not RecruitingTreatmentAdult Myelodysplastic Syndrome / Chronic Myelomonocytic Leukemia / Chronic Myelomonocytic Leukemia-1 / Chronic Myelomonocytic Leukemia-2 / Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts / Myelodysplastic Syndrome With Excess Blasts-1 / Myelodysplastic Syndrome With Excess Blasts-2 / Previously Treated Myelodysplastic Syndromes / Refractory Anemia With Excess Blasts1
2Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)2
2Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemia, Myelomonocytic, Chronic / Myelodysplastic Syndromes1
2Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome1
2Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
2Active Not RecruitingTreatmentLeukemia, Myelomonocytic, Juvenile / Myelodysplastic Syndrome1
2Active Not RecruitingTreatmentLeukemias4
2Active Not RecruitingTreatmentLung Cancer Non-Small Cell Cancer (NSCLC)2
2Active Not RecruitingTreatmentMetastatic Colorectal Cancers1
2Active Not RecruitingTreatmentMyelodysplastic Syndromes1
2Active Not RecruitingTreatmentRecurrent Non-Small Cell Lung Carcinoma / Stage IV Non-Small Cell Lung Cancer / Stage IV Non-Small Cell Lung Cancer AJCC v71
2Active Not RecruitingTreatmentSmith-Magenis Syndrome1
2CompletedSupportive CareMyelodysplastic Syndromes / Smith-Magenis Syndrome / Thrombocytopenias1
2CompletedTreatmentAcute Myelogenous Leukaemia (AML) / Leukemia Acute Myeloid Leukemia (AML)1
2CompletedTreatmentAcute Myelogenous Leukaemia (AML) / Leukemias / Myelodysplastic Syndrome1
2CompletedTreatmentAcute Myelogenous Leukaemia (AML) / Myelodysplastic Syndrome1
2CompletedTreatmentAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Adult Acute Myeloid Leukemia in Remission / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Promyelocytic Leukemia With t(15;17)(q22;q12); PML-RARA / Alkylating Agent-Related Acute Myeloid Leukemia / Chronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Secondary Myelodysplastic Syndromes / Untreated Adult Acute Myeloid Leukemia1
2CompletedTreatmentAdult Acute Megakaryoblastic Leukemia / Adult Acute Monoblastic Leukemia / Adult Acute Monocytic Leukemia / Adult Acute Myeloid Leukemia in Remission / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With Maturation / Adult Acute Myeloid Leukemia With Minimal Differentiation / Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Myeloid Leukemia Without Maturation / Adult Acute Myelomonocytic Leukemia / Adult Erythroleukemia / Adult Pure Erythroid Leukemia / Alkylating Agent-Related Acute Myeloid Leukemia / De Novo Myelodysplastic Syndrome / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia / Refractory Anemia With Excess Blasts / Secondary Myelodysplastic Syndromes / Untreated Adult Acute Myeloid Leukemia1
2CompletedTreatmentAdult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Del(5q) / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(15;17)(q22;q12) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Childhood Myelodysplastic Syndromes / Chronic Myelomonocytic Leukemia / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia / Recurrent Childhood Acute Myeloid Leukemia / Secondary Myelodysplastic Syndromes1
2CompletedTreatmentAgnogenic Myeloid Metaplasia1
2CompletedTreatmentBeta Thalassemia1
2CompletedTreatmentChronic Myelomonocytic Leukemia1
2CompletedTreatmentChronic Myelomonocytic Leukemia / Myelodysplastic Syndrome1
2CompletedTreatmentEstrogen Receptor Negative / Estrogen Receptor Positive / HER2/Neu Negative / Male Breast Carcinoma / Progesterone Receptor Negative / Recurrent Breast Carcinoma / Stage IIIC Breast Cancer / Stage IIIC Breast Cancer AJCC v7 / Stage IV Breast Cancer / Stage IV Breast Cancer AJCC v6 and v7 / Triple-Negative Breast Carcinoma1
2CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML)5
2CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome2
2CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes (MDS)1
2CompletedTreatmentLeukemias4
2CompletedTreatmentLeukemias / Lung Cancers / Malignant Lymphomas / Multiple Myeloma and Plasma Cell Neoplasm / Myelodysplastic Syndromes / Prostate Cancer1
2CompletedTreatmentLeukemias / Transplantation, Stem Cell1
2CompletedTreatmentMetastatic Colon Cancer / Recurrent Colon Cancer / Recurrent Rectal Cancer / Stage IV Rectal Cancer1
2CompletedTreatmentMyelodysplastic Syndrome3
2CompletedTreatmentMyelodysplastic Syndromes6
2CompletedTreatmentMyelodysplastic Syndromes (MDS)2
2CompletedTreatmentMyelodysplastic Syndrome / Smith-Magenis Syndrome1
2CompletedTreatmentNasopharyngeal Neoplasms1
2CompletedTreatmentNeoplasms, Breast1
2CompletedTreatmentProstate Cancer1
2Not Yet RecruitingTreatmentAcute Bilineal Leukemia / Acute Biphenotypic Leukemia (ABL) / Acute, recurrent Myeloid Leukemia / Chronic Myelomonocytic Leukemia / IDH2 Gene Mutation / Myelodysplastic Syndrome / Refractory Acute Myeloid Leukemia1
2Not Yet RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)1
2Not Yet RecruitingTreatmentMyelodysplastic Syndromes (MDS)1
2Not Yet RecruitingTreatmentRecurrent IDH1/2 Mutated Glioma1
2RecruitingTreatmentAcute Bilineal Leukemia / Acute Biphenotypic Leukemia (ABL) / Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Acute, recurrent Myeloid Leukemia / Chronic Myelomonocytic Leukemia / Leukemias / Myelodysplastic Syndrome / Refractory Acute Myeloid Leukemia / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Therapy-Related Acute Myeloid Leukemia1
2RecruitingTreatmentAcute Myelocytic Leukemia / Myelodysplastic Syndrome1
2RecruitingTreatmentAcute Myelogenous Leukemia AML / Myelodysplastic Syndrome MDS1
2RecruitingTreatmentAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Chronic Myelomonocytic Leukemia / Previously Treated Myelodysplastic Syndromes / Recurrent Acute Myeloid Leukemia With Myelodysplasia-Related Changes / Recurrent Adult Acute Myeloid Leukemia1
2RecruitingTreatmentAdult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Del(5q) / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(15;17)(q22;q12) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / De Novo Myelodysplastic Syndromes / Myelodysplastic Syndrome With Isolated Del(5q) / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia1
2RecruitingTreatmentChronic Myelomonocytic Leukemia / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
2RecruitingTreatmentChronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndrome / Myelodysplastic Syndrome / Secondary Myelodysplastic Syndromes1
2RecruitingTreatmentEstrogen Receptor Positive and HER2 Negative Breast Cancer / Microsatellite Stable Colorectal Carcinoma / Platinum Resistant Epithelial Ovarian Cancer Type II1
2RecruitingTreatmentHead and Neck Squamous Cell Carcinoma (HNSCC)1
2RecruitingTreatmentHematopoietic/Lymphoid Cancer / Myelodysplastic Syndrome1
2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)8
2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemia, Myelomonocytic, Chronic / Myelodysplastic Syndromes1
2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome2
2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome With Excess Blasts-2 (MDS-EB-2)1
2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes1
2RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes / Myeloproliferative Neoplasms1
2RecruitingTreatmentLeukemia, Erythroblastic, Acute / Myelodysplastic Syndromes1
2RecruitingTreatmentLeukemias1
2RecruitingTreatmentLeukemias / Myelodysplastic Syndrome1
2RecruitingTreatmentLeukemias / Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable / Myelofibrosis Transformation in Essential Thrombocythemia / Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase / Primary Myelofibrosis1
2RecruitingTreatmentMalignant Neoplasm of Pancreas2
2RecruitingTreatmentMelanoma and Other Malignant Neoplasms of Skin / Metastatic Melanoma1
2RecruitingTreatmentMyelodysplastic Syndrome3
2RecruitingTreatmentMyelodysplastic Syndromes1
2RecruitingTreatmentNeoplasms / Neoplasms, Hematologic1
2RecruitingTreatmentNon-Small Lung Cancer, Epigenetic Therapy1
2RecruitingTreatmentOvarian Epithelial Cancer1
2RecruitingTreatmentPreviously Untreated Peripheral T-cell Lymphoma1
2RecruitingTreatmentSmith-Magenis Syndrome / Systemic Autoimmune Diseases1
2SuspendedTreatmentAcute Leukemias of Ambiguous Lineage / Childhood B Acute Lymphoblastic Leukemia / KMT2A Gene Rearrangement / Mixed Phenotype Acute Leukemia (MPAL)1
2TerminatedTreatmentChronic Lymphocytic Leukaemia (CLL) / Leukemias1
2TerminatedTreatmentChronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL)1
2TerminatedTreatmentChronic Myelomonocytic Leukemia (CMML) / Myelodysplastic Syndromes (MDS)1
2TerminatedTreatmentChronic Myeloproliferative Disorders / Secondary Myelofibrosis1
2TerminatedTreatmentFollicular Lymphoma (FL) / Marginal Zone Lymphoma1
2TerminatedTreatmentHigh Risk MDS1
2TerminatedTreatmentLeukemia Acute Myeloid Leukemia (AML)2
2TerminatedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemias / Myelodysplastic Syndromes (MDS)1
2TerminatedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes (MDS)1
2TerminatedTreatmentLeukemia, Myelocytic, Acute / Myelodysplastic Syndromes1
2TerminatedTreatmentLeukemias2
2TerminatedTreatmentLeukemias / Myelodysplastic Syndromes2
2TerminatedTreatmentLymphoma, Hodgkins / Non-Hodgkin Lymphoma (Follicular or Large Diffuse B-cell Lymphoma or Mantle Cell Lymphoma)1
2TerminatedTreatmentMultiple Myeloma (MM)1
2TerminatedTreatmentMyelodysplastic Syndrome2
2TerminatedTreatmentMyelodysplastic Syndromes2
2TerminatedTreatmentRecurrent Non-Small Cell Lung Carcinoma / Stage IIIA Non-Small Cell Lung Cancer / Stage IIIb Non-small Cell Lung Cancer / Stage IV Non-Small Cell Lung Cancer1
2TerminatedTreatmentSmith-Magenis Syndrome1
2TerminatedTreatmentStage IA Non-Small Cell Lung Carcinoma / Stage IB Non-Small Cell Lung Carcinoma1
2TerminatedTreatmentThalassemia Major (TM)1
2Unknown StatusTreatmentAcute Myelogenous Leukaemia (AML) / Myelodysplastic Syndrome1
2Unknown StatusTreatmentElderly / Leukemia Acute Myeloid Leukemia (AML)1
2Unknown StatusTreatmentLeukemia Acute Myeloid Leukemia (AML)1
2Unknown StatusTreatmentMyelodysplastic Syndrome2
2WithdrawnTreatmentLeukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes (MDS)1
2, 3CompletedTreatmentLeukemias / Myelodysplastic Syndromes1
2, 3RecruitingTreatmentAcute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation / Leukemia Acute Myeloid Leukemia (AML)1
2, 3SuspendedTreatmentAdult Myelodysplastic Syndrome / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts-2 / Untreated Adult Acute Myeloid Leukemia1
3Active Not RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)2
3Active Not RecruitingTreatmentMyelodysplastic Syndrome1
3CompletedNot AvailableMyelodysplastic Syndromes1
3CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
3CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemias / Smith-Magenis Syndrome1
3CompletedTreatmentMyelodysplastic Syndromes1
3Not Yet RecruitingTreatmentRefractory Angioimmunoblastic T-cell Lymphoma / Relapsed Angioimmunoblastic T-Cell Lymphoma1
3Not Yet RecruitingTreatmentT-Cell Lymphomas1
3RecruitingTreatmentIsocitrate Dehydrogenase / Myeloid Leukemias1
3RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML)3
3RecruitingTreatmentLeukemia Acute Myeloid Leukemia (AML) / Leukemia, Myelomonocytic, Chronic / Myelodysplastic Syndrome1
3TerminatedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
3TerminatedTreatmentThrombocytopenias1
3WithdrawnTreatmentLeukemia Acute Myeloid Leukemia (AML)1
4CompletedTreatmentMyelodysplastic Syndromes1
4TerminatedTreatmentMyelodysplastic Syndromes1
Not AvailableActive Not RecruitingNot AvailableLeukemia Acute Myeloid Leukemia (AML)1
Not AvailableAvailableNot AvailableMyelodysplastic Syndromes (MDS)1
Not AvailableCompletedNot AvailableMyelodysplastic Syndrome1
Not AvailableCompletedNot AvailableMyelodysplastic Syndromes1
Not AvailableCompletedTreatmentHypermethylation / Lung Cancers / Non Small Cell Lung Carcinoma (NSCLC)1
Not AvailableCompletedTreatmentLeukemias1
Not AvailableCompletedTreatmentMultiple Myeloma (MM)1
Not AvailableNot Yet RecruitingDiagnosticMyelodysplastic Syndromes / Myeloid Leukemias1
Not AvailableWithdrawnTreatmentRecurrent Breast Cancer / Stage IA Breast Cancer / Stage IB Breast Cancer / Stage II Breast Cancer / Stage IIIA Breast Cancer / Stage IIIB Breast Cancer / Stage IIIC Breast Cancer / Stage IV Breast Cancer / Triple-Negative Breast Cancer (TNBC)1

Pharmacoeconomics

Manufacturers
  • Celgene corp
Packagers
  • Baxter International Inc.
  • Ben Venue Laboratories Inc.
  • Celgene
  • Pharmion LLC
Dosage forms
FormRouteStrength
Injection, powder, lyophilized, for solutionIntravenous; Subcutaneous100 mg/30mL
Injection, powder, lyophilized, for solutionIntravenous; Subcutaneous100 mg/1
Injection, powder, lyophilized, for solutionSubcutaneous100 mg/50mL
Injection, powder, lyophilized, for suspensionSubcutaneous100 mg/4mL
Powder, for suspensionSubcutaneous100 mg
Prices
Unit descriptionCostUnit
Vidaza 100 mg vial588.23USD vial
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patents
Not Available

Properties

State
Solid
Experimental Properties
PropertyValueSource
melting point (°C)229 °CPhysProp
water solubility8.9E+004 mg/LNot Available
logP-3.5Not Available
Predicted Properties
PropertyValueSource
Water Solubility12.1 mg/mLALOGPS
logP-2.4ALOGPS
logP-3.1ChemAxon
logS-1.3ALOGPS
pKa (Strongest Acidic)12.55ChemAxon
pKa (Strongest Basic)-0.38ChemAxon
Physiological Charge0ChemAxon
Hydrogen Acceptor Count8ChemAxon
Hydrogen Donor Count4ChemAxon
Polar Surface Area140.97 Å2ChemAxon
Rotatable Bond Count2ChemAxon
Refractivity52.19 m3·mol-1ChemAxon
Polarizability21.5 Å3ChemAxon
Number of Rings2ChemAxon
Bioavailability1ChemAxon
Rule of FiveYesChemAxon
Ghose FilterNoChemAxon
Veber's RuleNoChemAxon
MDDR-like RuleNoChemAxon
Predicted ADMET features
PropertyValueProbability
Human Intestinal Absorption+0.9509
Blood Brain Barrier+0.8753
Caco-2 permeable-0.8715
P-glycoprotein substrateNon-substrate0.7879
P-glycoprotein inhibitor INon-inhibitor0.9591
P-glycoprotein inhibitor IINon-inhibitor0.9671
Renal organic cation transporterNon-inhibitor0.9355
CYP450 2C9 substrateNon-substrate0.8103
CYP450 2D6 substrateNon-substrate0.8572
CYP450 3A4 substrateNon-substrate0.6067
CYP450 1A2 substrateNon-inhibitor0.9045
CYP450 2C9 inhibitorNon-inhibitor0.9436
CYP450 2D6 inhibitorNon-inhibitor0.9438
CYP450 2C19 inhibitorNon-inhibitor0.9329
CYP450 3A4 inhibitorNon-inhibitor0.9617
CYP450 inhibitory promiscuityLow CYP Inhibitory Promiscuity0.9839
Ames testAMES toxic0.8058
CarcinogenicityNon-carcinogens0.9182
BiodegradationNot ready biodegradable0.8432
Rat acute toxicity1.7991 LD50, mol/kg Not applicable
hERG inhibition (predictor I)Weak inhibitor0.9794
hERG inhibition (predictor II)Non-inhibitor0.8996
ADMET data is predicted using admetSAR, a free tool for evaluating chemical ADMET properties. (23092397)

Spectra

Mass Spec (NIST)
Not Available
Spectra
SpectrumSpectrum TypeSplash Key
Predicted GC-MS Spectrum - GC-MSPredicted GC-MSNot Available
Predicted MS/MS Spectrum - 10V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 10V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Negative (Annotated)Predicted LC-MS/MSNot Available
1H NMR Spectrum1D NMRNot Applicable
13C NMR Spectrum1D NMRNot Applicable

Taxonomy

Description
This compound belongs to the class of organic compounds known as glycosylamines. These are compounds consisting of an amine with a beta-N-glycosidic bond to a carbohydrate, thus forming a cyclic hemiaminal ether bond (alpha-amino ether).
Kingdom
Organic compounds
Super Class
Organic oxygen compounds
Class
Organooxygen compounds
Sub Class
Carbohydrates and carbohydrate conjugates
Direct Parent
Glycosylamines
Alternative Parents
Pentoses / Triazinones / Aminotriazines / 1,3,5-triazines / Tetrahydrofurans / Heteroaromatic compounds / Secondary alcohols / Oxacyclic compounds / Azacyclic compounds / Primary amines
show 4 more
Substituents
N-glycosyl compound / Pentose monosaccharide / Amino-1,3,5-triazine / Aminotriazine / Triazinone / Monosaccharide / Triazine / 1,3,5-triazine / Heteroaromatic compound / Tetrahydrofuran
show 14 more
Molecular Framework
Aromatic heteromonocyclic compounds
External Descriptors
nucleoside analogue, N-glycosyl-1,3,5-triazine (CHEBI:2038)

Targets

Kind
Protein
Organism
Human
Pharmacological action
Yes
Actions
Inhibitor
General Function
Zinc ion binding
Specific Function
Methylates CpG residues. Preferentially methylates hemimethylated DNA. Associates with DNA replication sites in S phase maintaining the methylation pattern in the newly synthesized strand, that is ...
Gene Name
DNMT1
Uniprot ID
P26358
Uniprot Name
DNA (cytosine-5)-methyltransferase 1
Molecular Weight
183163.635 Da
References
  1. Chen X, Ji ZL, Chen YZ: TTD: Therapeutic Target Database. Nucleic Acids Res. 2002 Jan 1;30(1):412-5. [PubMed:11752352]
  2. Kaminskas E, Farrell AT, Wang YC, Sridhara R, Pazdur R: FDA drug approval summary: azacitidine (5-azacytidine, Vidaza) for injectable suspension. Oncologist. 2005 Mar;10(3):176-82. [PubMed:15793220]
  3. Cataldo VD, Cortes J, Quintas-Cardama A: Azacitidine for the treatment of myelodysplastic syndrome. Expert Rev Anticancer Ther. 2009 Jul;9(7):875-84. doi: 10.1586/era.09.61. [PubMed:19589026]
  4. Leone G, Voso MT, Teofili L, Lubbert M: Inhibitors of DNA methylation in the treatment of hematological malignancies and MDS. Clin Immunol. 2003 Oct;109(1):89-102. [PubMed:14585280]
  5. Ghoshal K, Bai S: DNA methyltransferases as targets for cancer therapy. Drugs Today (Barc). 2007 Jun;43(6):395-422. [PubMed:17612710]
  6. Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF: Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002 May 15;20(10):2429-40. [PubMed:12011120]
  7. Fenaux P: Inhibitors of DNA methylation: beyond myelodysplastic syndromes. Nat Clin Pract Oncol. 2005 Dec;2 Suppl 1:S36-44. [PubMed:16341239]
  8. Silverman LR: Targeting hypomethylation of DNA to achieve cellular differentiation in myelodysplastic syndromes (MDS). Oncologist. 2001;6 Suppl 5:8-14. [PubMed:11700387]
  9. O'Dwyer K, Maslak P: Azacitidine and the beginnings of therapeutic epigenetic modulation. Expert Opin Pharmacother. 2008 Aug;9(11):1981-6. doi: 10.1517/14656566.9.11.1981 . [PubMed:18627335]
  10. Hollenbach PW, Nguyen AN, Brady H, Williams M, Ning Y, Richard N, Krushel L, Aukerman SL, Heise C, MacBeth KJ: A comparison of azacitidine and decitabine activities in acute myeloid leukemia cell lines. PLoS One. 2010 Feb 2;5(2):e9001. doi: 10.1371/journal.pone.0009001. [PubMed:20126405]
  11. Glover AB, Leyland-Jones B: Biochemistry of azacitidine: a review. Cancer Treat Rep. 1987 Oct;71(10):959-64. [PubMed:2443243]
2. RNA
Kind
Nucleotide
Organism
Human
Pharmacological action
Yes
Actions
Other
References
  1. Muller A, Florek M: 5-Azacytidine/Azacitidine. Recent Results Cancer Res. 2010;184:159-70. doi: 10.1007/978-3-642-01222-8_11. [PubMed:20072837]
  2. Kaminskas E, Farrell AT, Wang YC, Sridhara R, Pazdur R: FDA drug approval summary: azacitidine (5-azacytidine, Vidaza) for injectable suspension. Oncologist. 2005 Mar;10(3):176-82. [PubMed:15793220]
  3. Hollenbach PW, Nguyen AN, Brady H, Williams M, Ning Y, Richard N, Krushel L, Aukerman SL, Heise C, MacBeth KJ: A comparison of azacitidine and decitabine activities in acute myeloid leukemia cell lines. PLoS One. 2010 Feb 2;5(2):e9001. doi: 10.1371/journal.pone.0009001. [PubMed:20126405]
  4. Glover AB, Leyland-Jones B: Biochemistry of azacitidine: a review. Cancer Treat Rep. 1987 Oct;71(10):959-64. [PubMed:2443243]
  5. Cihak A, Vesely J, Skoda J: Azapyrimidine nucleosides: metabolism and inhibitory mechanisms. Adv Enzyme Regul. 1985;24:335-54. [PubMed:2424284]
  6. Dapp MJ, Clouser CL, Patterson S, Mansky LM: 5-Azacytidine can induce lethal mutagenesis in human immunodeficiency virus type 1. J Virol. 2009 Nov;83(22):11950-8. doi: 10.1128/JVI.01406-09. Epub 2009 Sep 2. [PubMed:19726509]
3. DNA
Kind
Nucleotide
Organism
Human
Pharmacological action
Yes
Actions
Other
General Function:
Used for biological information storage.
Specific Function:
DNA contains the instructions needed for an organism to develop, survive and reproduce.
Molecular Weight:
2.15 x 1012 Da
References
  1. Overington JP, Al-Lazikani B, Hopkins AL: How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. [PubMed:17139284]
  2. Imming P, Sinning C, Meyer A: Drugs, their targets and the nature and number of drug targets. Nat Rev Drug Discov. 2006 Oct;5(10):821-34. [PubMed:17016423]
  3. Allen A: Epigenetic alterations and cancer: new targets for therapy. IDrugs. 2007 Oct;10(10):709-12. [PubMed:17899489]
  4. Kaminskas E, Farrell AT, Wang YC, Sridhara R, Pazdur R: FDA drug approval summary: azacitidine (5-azacytidine, Vidaza) for injectable suspension. Oncologist. 2005 Mar;10(3):176-82. [PubMed:15793220]
  5. O'Dwyer K, Maslak P: Azacitidine and the beginnings of therapeutic epigenetic modulation. Expert Opin Pharmacother. 2008 Aug;9(11):1981-6. doi: 10.1517/14656566.9.11.1981 . [PubMed:18627335]
  6. Muller A, Florek M: 5-Azacytidine/Azacitidine. Recent Results Cancer Res. 2010;184:159-70. doi: 10.1007/978-3-642-01222-8_11. [PubMed:20072837]
  7. Hollenbach PW, Nguyen AN, Brady H, Williams M, Ning Y, Richard N, Krushel L, Aukerman SL, Heise C, MacBeth KJ: A comparison of azacitidine and decitabine activities in acute myeloid leukemia cell lines. PLoS One. 2010 Feb 2;5(2):e9001. doi: 10.1371/journal.pone.0009001. [PubMed:20126405]
  8. Glover AB, Leyland-Jones B: Biochemistry of azacitidine: a review. Cancer Treat Rep. 1987 Oct;71(10):959-64. [PubMed:2443243]
  9. Cihak A, Vesely J, Skoda J: Azapyrimidine nucleosides: metabolism and inhibitory mechanisms. Adv Enzyme Regul. 1985;24:335-54. [PubMed:2424284]
  10. Dapp MJ, Clouser CL, Patterson S, Mansky LM: 5-Azacytidine can induce lethal mutagenesis in human immunodeficiency virus type 1. J Virol. 2009 Nov;83(22):11950-8. doi: 10.1128/JVI.01406-09. Epub 2009 Sep 2. [PubMed:19726509]

Enzymes

Kind
Protein
Organism
Human
Pharmacological action
Unknown
Actions
Substrate
General Function
Zinc ion binding
Specific Function
This enzyme scavenges exogenous and endogenous cytidine and 2'-deoxycytidine for UMP synthesis.
Gene Name
CDA
Uniprot ID
P32320
Uniprot Name
Cytidine deaminase
Molecular Weight
16184.545 Da
References
  1. Garcia-Manero G, Stoltz ML, Ward MR, Kantarjian H, Sharma S: A pilot pharmacokinetic study of oral azacitidine. Leukemia. 2008 Sep;22(9):1680-4. doi: 10.1038/leu.2008.145. Epub 2008 Jun 12. [PubMed:18548103]

Drug created on June 13, 2005 07:24 / Updated on November 20, 2018 00:48