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 APG2575 Single Agent and Combination With Therapy in Patients Relapsed/Refractory AML

First Posted Date
2020-08-06
Last Posted Date
2024-10-14
Lead Sponsor
Ascentage Pharma Group Inc.
Target Recruit Count
284
Registration Number
NCT04501120
Locations
🇨🇳

Peking University People's Hospital, Beijing, Beijing, China

🇨🇳

Sun Yat-sen University Cancer Center, Guangzhou, Guandong, China

🇨🇳

Henan Tumor Hospital, Zhengzhou, Henan, China

and more 9 locations

Study of Azacytidine Combined With Lenalidomide As Maintenance Therapy Based on MRD Monitoring in AML

First Posted Date
2020-07-29
Last Posted Date
2022-03-24
Lead Sponsor
Ge Zheng
Target Recruit Count
60
Registration Number
NCT04490707
Locations
🇨🇳

Department of Hematology, Zhongda Hospital Southeast University, Institute of Hematology Southeast University, Nanjing, Jiangsu, China

Efficacy and Safety Evaluation of IBI188 in Combination With Demethylating Agents in Treatment of Patients With Acute Myeloid Leukemia

First Posted Date
2020-07-24
Last Posted Date
2023-10-23
Lead Sponsor
Innovent Biologics (Suzhou) Co. Ltd.
Target Recruit Count
222
Registration Number
NCT04485052
Locations
🇨🇳

The First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China

Expanded Access Program With Pevonedistat (Given With Azacitidine) for Adults With Higher-risk Myelodysplastic Syndromes

First Posted Date
2020-07-23
Last Posted Date
2022-09-30
Lead Sponsor
Takeda
Registration Number
NCT04484363

Azacitidine Combined With Chidamide in the Treatment of Newly Diagnosed PTCL Unfit for Conventional Chemotherapy

First Posted Date
2020-07-21
Last Posted Date
2022-04-20
Lead Sponsor
Ruijin Hospital
Target Recruit Count
28
Registration Number
NCT04480125
Locations
🇨🇳

Shanghai Ruijin Hospital, Shanghai, China

Magrolimab, Azacitidine, and Venetoclax for the Treatment of Acute Myeloid Leukemia

First Posted Date
2020-06-17
Last Posted Date
2024-12-05
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
110
Registration Number
NCT04435691
Locations
🇺🇸

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

A Study of Evorpacept (ALX148) With Azacitidine for Higher Risk Myelodysplastic Syndrome (ASPEN-02)

Phase 1
Active, not recruiting
Conditions
Interventions
First Posted Date
2020-06-04
Last Posted Date
2023-08-14
Lead Sponsor
ALX Oncology Inc.
Target Recruit Count
65
Registration Number
NCT04417517
Locations
🇺🇸

University of Southern California, Norris Comprehensive Cancer Center, Los Angeles, California, United States

🇺🇸

Duke University Medical Center, Durham, North Carolina, United States

🇺🇸

IU Simon Cancer Center, Indianapolis, Indiana, United States

and more 16 locations

Daratumumab, Azacitidine, and Dexamethasone for Treatment of Patients With Recurrent or Refractory Multiple Myeloma Previously Treated With Daratumumab

First Posted Date
2020-05-29
Last Posted Date
2024-04-30
Lead Sponsor
University of California, San Francisco
Target Recruit Count
5
Registration Number
NCT04407442
Locations
🇺🇸

University of California, San Francisco, San Francisco, California, United States

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