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 Azacitidine Combined With Homoharringtonie Based Regimens in AML

First Posted Date
2020-01-30
Last Posted Date
2022-09-08
Lead Sponsor
Ge Zheng
Target Recruit Count
100
Registration Number
NCT04248595
Locations
🇨🇳

Department of Hematology, Zhongda Hospital, Medical School of Southeast University, NanJing, China

A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

First Posted Date
2020-01-28
Last Posted Date
2024-03-20
Lead Sponsor
Edgewood Oncology Inc.
Target Recruit Count
80
Registration Number
NCT04243785
Locations
🇺🇸

City of Hope National Medical Center, Duarte, California, United States

🇺🇸

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

🇺🇸

Memorial Sloan-Kettering Cancer Center, New York, New York, United States

A Study of Cusatuzumab Plus Azacitidine in Japanese Participants With Newly Diagnosed Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome Who Are Not Candidates for Intensive Treatment

Phase 1
Completed
Conditions
Interventions
First Posted Date
2020-01-27
Last Posted Date
2023-08-09
Lead Sponsor
OncoVerity, Inc.
Target Recruit Count
6
Registration Number
NCT04241549
Locations
🇯🇵

Fukushima Medical University Hospital, Fukushima, Japan

🇯🇵

Osaka City General Hospital, Osaka, Japan

🇯🇵

University of Fukui Hospital, Yoshida, Japan

and more 2 locations

Acute Myeloid Leukemia Real World Treatment Patterns

First Posted Date
2020-01-18
Last Posted Date
2023-06-07
Lead Sponsor
Pfizer
Registration Number
NCT04230564

Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2

First Posted Date
2020-01-18
Last Posted Date
2024-04-09
Lead Sponsor
Sellas Life Sciences Group
Target Recruit Count
128
Registration Number
NCT04229979
Locations
🇺🇸

Mayo Clinic Jacksonville Florida, Jacksonville, Florida, United States

🇫🇷

CHU de Caen, Caen, France

🇬🇷

General Hospital of Athens "Evaggelismos", Athens, Greece

and more 70 locations

A Safety Study of SEA-CD70 in Patients With Myeloid Malignancies

First Posted Date
2020-01-14
Last Posted Date
2024-10-09
Lead Sponsor
Seagen Inc.
Target Recruit Count
140
Registration Number
NCT04227847
Locations
🇺🇸

University of Alabama at Birmingham, Birmingham, Alabama, United States

🇺🇸

City of Hope, Duarte, California, United States

🇺🇸

UCLA Department of Medicine - Hematology & Oncology, Los Angeles, California, United States

and more 20 locations

APR-246 in Combination With Venetoclax and Azacitidine in TP53-Mutant Myeloid Malignancies

First Posted Date
2020-01-02
Last Posted Date
2022-01-19
Lead Sponsor
Aprea Therapeutics
Target Recruit Count
51
Registration Number
NCT04214860
Locations
🇺🇸

Northwestern Medicine, Chicago, Illinois, United States

🇺🇸

H. Lee Moffitt CC, Tampa, Florida, United States

🇺🇸

Weill Cornell Cancer Center, New York, New York, United States

and more 5 locations

Low-dose AZA Combined With Short Term CAG Derived Regimen as a Bridging Treatment in Patients With Advanced MDS Prior to Allo-HSCT

Phase 2
Conditions
Interventions
First Posted Date
2020-01-02
Last Posted Date
2020-01-03
Lead Sponsor
Fujian Medical University
Target Recruit Count
20
Registration Number
NCT04216355
Locations
🇨🇳

Fujian Medical University Union Hospital, Fuzhou, Fujian, China

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

First Posted Date
2019-12-05
Last Posted Date
2024-11-07
Lead Sponsor
Benjamin Tomlinson
Target Recruit Count
20
Registration Number
NCT04187703
Locations
🇺🇸

Cleveland Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center, Cleveland, Ohio, United States

Feasibility of Allogeneic Stem Cell Transplantation in Higher-risk-MDS (ACROBAT)

First Posted Date
2019-12-03
Last Posted Date
2022-10-13
Lead Sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto
Target Recruit Count
274
Registration Number
NCT04184505
Locations
🇮🇹

Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli" Po E. Morelli - Reggio Calabria - Uoc Ematologia, Reggio Calabria, Italy

🇮🇹

Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia, Bologna, Italy

🇮🇹

Aou Città Della Salute E Della Scienza, Ospedale S. Giovanni Battista Molinette - Torino - Sc Ematologia - Università Degli Studi Di Torino, Torino, Italy

and more 17 locations
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