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
-

Azacitidine in Patients Undergoing Matched Unrelated Stem Cell Transplantation

First Posted Date
2012-12-11
Last Posted Date
2019-10-16
Lead Sponsor
Washington University School of Medicine
Target Recruit Count
54
Registration Number
NCT01747499
Locations
🇺🇸

Washington University School of Medicine, Saint Louis, Missouri, United States

Azacitidine and Lenalidomide for Relapsed and Refractory Patients With Acute Myeloid Leukemia

First Posted Date
2012-12-06
Last Posted Date
2019-10-08
Lead Sponsor
University of Colorado, Denver
Target Recruit Count
37
Registration Number
NCT01743859
Locations
🇺🇸

University of Colorado Cancer Center, Aurora, Colorado, United States

Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk

Phase 2
Completed
Conditions
Interventions
First Posted Date
2012-11-02
Last Posted Date
2020-12-24
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
113
Registration Number
NCT01720225
Locations
🇺🇸

University of Texas MD Anderson Cancer Center, Houston, Texas, United States

Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS

First Posted Date
2012-10-04
Last Posted Date
2022-10-18
Lead Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Target Recruit Count
25
Registration Number
NCT01700673
Locations
🇺🇸

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, United States

5 Day Versus 7 Day Azacitidine in Lower Risk Myelodysplastic Syndrome

Phase 2
Conditions
Interventions
First Posted Date
2012-07-30
Last Posted Date
2015-11-20
Lead Sponsor
Seoul St. Mary's Hospital
Target Recruit Count
92
Registration Number
NCT01652781
Locations
🇰🇷

Seoul St. Mary's Hospital, Seoul, Korea, Republic of

🇰🇷

Asan Medical Center, Seoul, Korea, Republic of

🇰🇷

Seoul National University Hospital, Seoul, Korea, Republic of

Tosedostat and Cytarabine or Azacitidine in Treating Older Participants With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

First Posted Date
2012-07-10
Last Posted Date
2020-04-13
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
18
Registration Number
NCT01636609
Locations
🇺🇸

M D Anderson Cancer Center, Houston, Texas, United States

Randomised Study of Azacitidine Versus Azacitidine With Vorinostat in Patients With AML or High Risk MDS

Phase 2
Completed
Conditions
Interventions
First Posted Date
2012-06-12
Last Posted Date
2021-05-05
Lead Sponsor
University of Birmingham
Target Recruit Count
260
Registration Number
NCT01617226
Locations
🇬🇧

Barts and the London NHS Trust, London, Greater London, United Kingdom

🇬🇧

Hammersmith Hospital, London, Greater London, United Kingdom

🇬🇧

Queen Elizabeth Hospital, Birmingham, West Midlands, United Kingdom

and more 10 locations

Study of Azacitidine to Evaluate Safety and Effectiveness for Chinese Patients With Higher Risk Myelodysplastic Syndrome

Phase 2
Completed
Conditions
Interventions
First Posted Date
2012-05-16
Last Posted Date
2019-03-19
Lead Sponsor
Celgene
Target Recruit Count
72
Registration Number
NCT01599325
Locations
🇨🇳

Peking University People's Hospital, Beijing, China

🇨🇳

The 301 Hospital- Chinese PLA General Hospital, Beijing, China

🇨🇳

The Third Hospital of Peking University, Beijing, China

and more 8 locations

The Efficacy of Azacitidine +/- Lenalidomide in High-risk Myelodysplastic Syndrome (MDS)and Acute Myeloid Leukemia (AML) With Del(5q).

First Posted Date
2012-03-16
Last Posted Date
2012-03-16
Lead Sponsor
Nordic MDS Group
Target Recruit Count
72
Registration Number
NCT01556477
Locations
🇸🇪

Lars Möllgård, Stockholm, Sweden

Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)

Phase 2
Terminated
Conditions
Interventions
First Posted Date
2012-03-02
Last Posted Date
2014-04-30
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
8
Registration Number
NCT01542684
Locations
🇺🇸

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

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