Azacitidine

Generic Name
Azacitidine
Brand Names
Onureg, Vidaza, Azacitidine Accord, Azacitidine betapharm, Azacitidine Mylan, Azacitidine Kabi
Drug Type
Small Molecule
Chemical Formula
C8H12N4O5
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)
Associated Therapies
-

Study of Vidaza (Azacitidine) Versus Support Treatment in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) Without the 5q Deletion and Transfusion Dependent Anaemia

Phase 2
Completed
Conditions
Interventions
First Posted Date
2011-04-19
Last Posted Date
2016-01-26
Lead Sponsor
Asociación Andaluza de Hematología y Hemoterapia
Target Recruit Count
40
Registration Number
NCT01338337
Locations
🇪🇸

Hospital Carlos Haya, Malaga, Spain

🇪🇸

Hospital Universitario San Cecilio, Granada, Spain

🇪🇸

Hospital Costa del Sol, Marbella, Malaga, Spain

and more 6 locations

Azacitidine With or Without Ceplene/Interleukin-2 in Patients With Higher Risk Myelodysplastic Syndromes

First Posted Date
2011-03-29
Last Posted Date
2014-03-20
Lead Sponsor
Groupe Francophone des Myelodysplasies
Registration Number
NCT01324960
Locations
🇫🇷

CHU Cochin, Paris, Ile de France, France

🇫🇷

CHU Grenoble, Grenoble, France

🇫🇷

Hôpital Versailles, Le Chesnay, France

and more 25 locations

Intensified Azacitidine in High Risk Myelodysplastic Syndrome (MDS)

Phase 1
Completed
Conditions
Interventions
First Posted Date
2011-02-28
Last Posted Date
2021-12-28
Lead Sponsor
Groupe Francophone des Myelodysplasies
Target Recruit Count
27
Registration Number
NCT01305460
Locations
🇫🇷

CHU d'Amiens, Amiens, France

🇫🇷

Centre henri Mondor, Creteil, France

🇫🇷

Centre Hospitalier de La Cote Basque, Bayonne, France

and more 18 locations

Elderly Patients With Acute Myeloid Leukemia (AML), Maintenance Phase After Complete Remission (CR)

Phase 2
Completed
Conditions
Interventions
First Posted Date
2011-02-23
Last Posted Date
2016-03-16
Lead Sponsor
French Innovative Leukemia Organisation
Target Recruit Count
120
Registration Number
NCT01301820
Locations
🇫🇷

Mathilde HUNAULT BERGER, Angers, France

Azacitidine in Treating Patients With Previously Treated Advanced Non-Small Cell Lung Cancer

First Posted Date
2011-01-21
Last Posted Date
2019-09-24
Lead Sponsor
National Cancer Institute (NCI)
Target Recruit Count
1
Registration Number
NCT01281124
Locations
🇺🇸

Ohio State University Comprehensive Cancer Center, Columbus, Ohio, United States

Sorafenib and 5-Azacitidine in Acute Leukemia + Myelodysplastic Syndrome

Phase 1
Completed
Conditions
Interventions
First Posted Date
2010-12-07
Last Posted Date
2016-05-06
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
60
Registration Number
NCT01254890
Locations
🇺🇸

UT MD Anderson Cancer Center, Houston, Texas, United States

Study Examining the Combination of Revlimid (Lenalidomide)and Vidaza (Azacitidine) for Relapsed/Refractory CLL and SLL

First Posted Date
2010-11-16
Last Posted Date
2022-07-20
Lead Sponsor
Hackensack Meridian Health
Target Recruit Count
5
Registration Number
NCT01241786
Locations
🇺🇸

Hackensack University Medical Center, Hackensack, New Jersey, United States

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