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
-

Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome

First Posted Date
2021-03-15
Last Posted Date
2024-11-11
Lead Sponsor
Syros Pharmaceuticals
Target Recruit Count
550
Registration Number
NCT04797780
Locations
🇺🇸

University of Arizona, Tucson, Arizona, United States

🇺🇸

UC Irvine, Irvine, California, United States

🇺🇸

University of Southern California, Los Angeles, California, United States

and more 134 locations

Study of Magrolimab in Combination With Azacitidine Versus Physician's Choice of Venetoclax in Combination With Azacitidine or Intensive Chemotherapy in Patients With TP53 Mutant Acute Myeloid Leukemia That Have Not Been Treated

First Posted Date
2021-03-03
Last Posted Date
2024-04-15
Lead Sponsor
Gilead Sciences
Target Recruit Count
258
Registration Number
NCT04778397
Locations
🇦🇺

Fiona Stanley Hospital, Murdoch, Western Australia, Australia

🇦🇺

Royal Adelaide Hospital, Adelaide, South Australia, Australia

🇺🇸

Massachusetts General Hospital, Boston, Massachusetts, United States

and more 153 locations

Study of Magrolimab Combinations in Participants With Myeloid Malignancies

First Posted Date
2021-03-03
Last Posted Date
2024-10-08
Lead Sponsor
Gilead Sciences
Target Recruit Count
54
Registration Number
NCT04778410
Locations
🇺🇸

Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute, Los Angeles, California, United States

🇺🇸

Swedish Cancer Institute, Seattle, Washington, United States

🇺🇸

USC/Norris Comprehensive Cancer Center, Los Angeles, California, United States

and more 18 locations

A Study of BGB-11417 in Participants With Myeloid Malignancies

First Posted Date
2021-02-25
Last Posted Date
2024-12-06
Lead Sponsor
BeiGene
Target Recruit Count
260
Registration Number
NCT04771130
Locations
🇦🇺

Austin Health, Heidelberg, Victoria, Australia

🇦🇺

The Alfred Hospital, Melbourne, Victoria, Australia

🇦🇺

Fiona Stanley Hospital, Murdoch, Western Australia, Australia

and more 49 locations

A Study of Evorpacept (ALX148) With Venetoclax and Azacitidine for Acute Myeloid Leukemia (ASPEN-05)

First Posted Date
2021-02-16
Last Posted Date
2024-11-27
Lead Sponsor
ALX Oncology Inc.
Target Recruit Count
14
Registration Number
NCT04755244
Locations
🇺🇸

Vanderbilt University Medical Center, Nashville, Tennessee, United States

🇺🇸

Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States

🇺🇸

Roswell Park Comprehensive Cancer Center, Buffalo, New York, United States

and more 2 locations

A Safety, Tolerability and Preliminary Efficacy Study of CC-90011 in Combination With Venetoclax and Azacitidine in R/R Acute Myeloid Leukemia and Treatment-naïve Participants Not Eligible for Intensive Therapy

First Posted Date
2021-02-10
Last Posted Date
2023-03-02
Lead Sponsor
Celgene
Target Recruit Count
1
Registration Number
NCT04748848
Locations
🇺🇸

Local Institution - 121, New York, New York, United States

🇪🇸

Local Institution - 802, Madrid, Spain

🇺🇸

Local Institution - 104, Dallas, Texas, United States

and more 14 locations

Randomized Phase IIB Trial of Oral Azacytidine Plus Romidepsin Versus Investigator's Choice in PTCL

First Posted Date
2021-02-10
Last Posted Date
2024-08-07
Lead Sponsor
University of Virginia
Target Recruit Count
50
Registration Number
NCT04747236
Locations
🇺🇸

Duke University, Durham, North Carolina, United States

🇺🇸

Icahn School of Medicine at Mount Sinai, New York, New York, United States

🇺🇸

University of Virginia, Charlottesville, Virginia, United States

and more 2 locations

A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML

First Posted Date
2021-01-29
Last Posted Date
2024-04-10
Lead Sponsor
Treadwell Therapeutics, Inc
Target Recruit Count
72
Registration Number
NCT04730258
Locations
🇺🇸

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

🇺🇸

City of Hope, Duarte, California, United States

🇺🇸

New York Presbyterian Weill Cornell Medical Center, New York, New York, United States

and more 6 locations
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