Azacitidine

Generic Name: Azacitidine

Brand Names: Onureg, Vidaza, Azacitidine Accord, Azacitidine betapharm, Azacitidine Mylan, Azacitidine Kabi

Drug Type: Small Molecule

Chemical Formula: C_₈H_₁₂N_₄O_₅

CAS Number: 320-67-2

Unique Ingredient Identifier: M801H13NRU

Background: Azacitidine is a pyrimidine nucleoside analogue with anti-neoplastic activity. It differs from cytosine by the presence of nitrogen in the C5-position, key in its hypomethylating activity. Two main mechanisms of action have been proposed for azacitidine. One of them is the induction of cytotoxicity. As an analogue of cytidine, it is able to incorporate into RNA and DNA, disrupting RNA metabolism and inhibiting protein and DNA synthesis. The other one is through the inhibition of DNA methyltransferase, impairing DNA methylation. Due to its anti-neoplastic activity and its ability to inhibit methylation in replicating DNA, azacytidine has been used mainly used in the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), two types of cancer characterized by the presence of aberrant DNA methylation.

In May 2004, the FDA approved the use of azacitidine administered subcutaneously for the treatment of MDS of all French-American-British (FAB) subtypes. In January 2007, the FDA approved the intravenous administration of azacitidine. The use of oral azacitidine for the treatment of AML in patients in complete remission was approved by the FDA in September 2020.

Indication: Azacitidine (for subcutaneous or intravenous use) is indicated for the treatment of adult patients with the following French-American-British (FAB) myelodysplastic syndrome (MDS) subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL). Azacitidine is also indicated for the treatment of pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML).

Azacitidine (for oral use) is indicated for continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission or complete remission with incomplete blood count recovery following intensive induction chemotherapy and are not able to complete intensive curative therapy.

Associated Conditions: Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Refractory Anemia, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Excess of Blasts (RAEB), Refractory Anemia With Ringed Sideroblasts, Newly diagnosed Juvenile Myelomonocytic Leukaemias (JMML)

Clinical Trials

Azacytidine With Valproic Acid Versus Ara-C in Acute Myeloid Leukemia (AML)/ Myelodysplastic Syndrome (MDS) Patients

Phase 2

Completed

Conditions: Acute Myelogenous Leukemia
Myelodysplastic Syndrome
Leukemia

Interventions: Drug: 5-Azacytidine
Drug: Ara-C
Drug: Valproic Acid (VPA)

First Posted Date: 2006-09-29

Last Posted Date: 2012-08-07

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 11

Registration Number: NCT00382590

Locations: 🇺🇸
UT MD Anderson Cancer Center, Houston, Texas, United States

Azacitidine in Treating Patients With Myelofibrosis

Phase 2

Terminated

Conditions: Chronic Myeloproliferative Disorders
Secondary Myelofibrosis

First Posted Date: 2006-09-28

Last Posted Date: 2011-04-21

Lead Sponsor: Mayo Clinic

Target Recruit Count: 10

Registration Number: NCT00381693

Locations: 🇺🇸
Mayo Clinic in Arizona, Scottsdale, Arizona, United States
🇺🇸
Mayo Clinic, Rochester, Minnesota, United States

Azacitidine and Erythropoietin Versus Azacitidine Alone for Patients With Low-Risk Myelodysplastic Syndromes

Phase 2

Terminated

Conditions: Myelodysplastic Syndromes

Interventions: Drug: Azacitidine (Monotherapy)
Drug: Azacitidine
Drug: Erythropoietin

First Posted Date: 2006-09-25

Last Posted Date: 2018-02-14

Lead Sponsor: Larry Cripe, MD

Target Recruit Count: 15

Registration Number: NCT00379912

Locations: 🇺🇸
Center for Hematology-Oncology of S Michigan, Jackson, Michigan, United States
🇺🇸
Horizon Oncology Center, Lafayette, Indiana, United States
🇺🇸
Indiana University Cancer Center, Indianapolis, Indiana, United States

and more 6 locations

Belinostat and Azacitidine in Treating Patients With Advanced Hematologic Cancers or Other Diseases

Phase 1

Completed

Conditions: Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable
Accelerated Phase of Disease
Adult Acute Promyelocytic Leukemia With t(15;17)(q22;q12); PML-RARA
de Novo Myelodysplastic Syndrome
Primary Myelofibrosis
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1
Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11
Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL
Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative
Chronic Myelogenous Leukemia, BCR-ABL1 Positive

Interventions: Drug: Belinostat
Drug: Azacitidine
Other: Laboratory Biomarker Analysis

First Posted Date: 2006-07-14

Last Posted Date: 2014-12-23

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 56

Registration Number: NCT00351975

Locations: 🇳🇿
Princess Margaret Hospital, Cashmere, Canterbury, New Zealand
🇨🇦
University Health Network-Princess Margaret Hospital, Toronto, Ontario, Canada
🇺🇸
University of Chicago Comprehensive Cancer Center, Chicago, Illinois, United States

and more 1 locations

Lenalidomide and Azacitidine in Treating Patients With Advanced Myelodysplastic Syndromes

Phase 1

Completed

Conditions: Myelodysplastic Syndromes
Leukemia

Interventions: Drug: azacitidine
Drug: lenalidomide

First Posted Date: 2006-07-14

Last Posted Date: 2018-09-19

Lead Sponsor: Mikkael Sekeres MD

Target Recruit Count: 37

Registration Number: NCT00352001

Locations: 🇺🇸
H. Lee Moffitt Cancer Center and Research Institute at University of South Florida, Tampa, Florida, United States
🇺🇸
University of California at Los Angeles, Los Angeles, California, United States
🇺🇸
Cleveland Clinic Taussig Cancer Instititute, Case Comprehensive Cancer Center, Cleveland, Ohio, United States

Azacitidine Maintenance Therapy After Allogeneic Bone Marrow Transplantation (Allo BMT)

Phase 1

Completed

Conditions: Myelodysplastic Syndrome
Leukemia

Interventions: Drug: Azacitidine

First Posted Date: 2006-07-11

Last Posted Date: 2012-07-30

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 90

Registration Number: NCT00350818

Locations: 🇺🇸
UT MD Anderson Cancer Center, Houston, Texas, United States

5-azacytidine Valproic Acid and ATRA in AML and High Risk MDS

Phase 2

Completed

Conditions: Acute Myelogenous Leukaemia (AML)
Myelodysplastic Syndrome (MDS)

Interventions: Drug: 5 azacytidine - VALPROIC acid- Retinoic acid

First Posted Date: 2006-06-20

Last Posted Date: 2011-05-09

Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Target Recruit Count: 25

Registration Number: NCT00339196

Locations: 🇫🇷
Chu Saint Louis, Paris, France

Vorinostat and Azacitidine in Treating Patients With Locally Recurrent or Metastatic Nasopharyngeal Cancer or Nasal Natural Killer T-Cell Lymphoma

Phase 1

Active, not recruiting

Conditions: Adult Nasal Type Extranodal NK/T-Cell Lymphoma
Recurrent Nasopharyngeal Keratinizing Squamous Cell Carcinoma
Recurrent Nasopharyngeal Undifferentiated Carcinoma
Stage IV Nasopharyngeal Keratinizing Squamous Cell Carcinoma AJCC v7
Stage IV Nasopharyngeal Undifferentiated Carcinoma AJCC v7

Interventions: Drug: Azacitidine
Other: Laboratory Biomarker Analysis
Other: Pharmacological Study
Drug: Vorinostat

First Posted Date: 2006-06-12

Last Posted Date: 2025-03-21

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 18

Registration Number: NCT00336063

Locations: 🇸🇬
National University Hospital Singapore, Singapore, Singapore
🇯🇵
National Cancer Center Hospital, Tokyo, Japan
🇺🇸
Johns Hopkins University/Sidney Kimmel Cancer Center, Baltimore, Maryland, United States

and more 2 locations