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
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Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Participants With Acute Myeloid Leukemia and With Azacitidine With or Without Venetoclax in Participants With Myelodysplastic Syndrome

First Posted Date
2021-06-03
Last Posted Date
2024-02-26
Lead Sponsor
AbbVie
Target Recruit Count
40
Registration Number
NCT04912063
Locations
🇮🇱

Hadassah Medical Center-Hebrew University /ID# 227275, Jerusalem, Yerushalayim, Israel

🇦🇺

Austin Health /ID# 227717, Heidelberg, Victoria, Australia

🇮🇱

Rabin Medical Center /ID# 227738, Petakh Tikva, Israel

and more 26 locations

Azacitidine or Decitabine With Venetoclax for Acute Myeloid Leukemia With Prior Hypomethylating Agent Failure

First Posted Date
2021-05-28
Last Posted Date
2024-05-29
Lead Sponsor
Brian Jonas
Target Recruit Count
20
Registration Number
NCT04905810
Locations
🇺🇸

UCLA / Jonsson Comprehensive Cancer Center, Los Angeles, California, United States

🇺🇸

University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States

🇺🇸

UCSF-Fresno, Clovis, California, United States

and more 1 locations

Tamibarotene Plus Venetoclax/Azacitidine in Participants with Newly Diagnosed AML

Phase 2
Active, not recruiting
Conditions
Interventions
First Posted Date
2021-05-27
Last Posted Date
2024-10-14
Lead Sponsor
Syros Pharmaceuticals
Target Recruit Count
95
Registration Number
NCT04905407
Locations
🇺🇸

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

🇫🇷

Hôpital l'Archet- CHU de Nice, Nice, France

🇺🇸

City of Hope, Duarte, California, United States

and more 25 locations

A Trial of AK117 (Anti-CD47) in Patients With Myelodysplastic Syndrome

Phase 1
Recruiting
Conditions
Interventions
First Posted Date
2021-05-25
Last Posted Date
2022-10-12
Lead Sponsor
Akeso
Target Recruit Count
190
Registration Number
NCT04900350
Locations
🇨🇳

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

BRE-04: Window of Opportunity Trial of Preoperative Low Dose Azacitidine in High-Risk Early Stage Breast Cancer

Phase 2
Recruiting
Conditions
Interventions
First Posted Date
2021-05-18
Last Posted Date
2024-05-20
Lead Sponsor
University of Illinois at Chicago
Target Recruit Count
40
Registration Number
NCT04891068
Locations
🇺🇸

University of Illinois Cancer Center, Chicago, Illinois, United States

STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS

First Posted Date
2021-05-07
Last Posted Date
2024-10-18
Lead Sponsor
Novartis Pharmaceuticals
Target Recruit Count
39
Registration Number
NCT04878432
Locations
🇺🇸

Duke Cancer Institute, Durham, North Carolina, United States

🇺🇸

University Hospitals Of Cleveland, Cleveland, Ohio, United States

🇺🇸

Cleveland Clinic, Cleveland, Ohio, United States

and more 12 locations

Jaktinib and Azacitidine In Treating Patients With MDS With MF or MDS/MPN With MF.

First Posted Date
2021-04-29
Last Posted Date
2022-12-01
Lead Sponsor
Suzhou Zelgen Biopharmaceuticals Co.,Ltd
Target Recruit Count
1
Registration Number
NCT04866056
Locations
🇨🇳

79 Qingchun Road, Hangzhou, Zhejiang, China

Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML

First Posted Date
2021-04-13
Last Posted Date
2023-12-18
Lead Sponsor
Pfizer
Target Recruit Count
14
Registration Number
NCT04842604
Locations
🇦🇹

Landeskrankenhaus Salzburg, Universitatsklinik fur Innere Medizin III der PMU, Salzburg, Austria

🇭🇺

Debreceni Egyetem Klinikai Kozpont Belgyogyaszati Klinika, Hematologia Tanszek, Debrecen, Hungary

🇦🇹

Uniklinikum Salzburg, Landeskrankenhaus Salzburg, Salzburg, Austria

and more 15 locations

A Study of Sabatolimab in Combination With Azacitidine and Venetoclax in High or Very High Risk MDS Participants

First Posted Date
2021-03-23
Last Posted Date
2024-10-22
Lead Sponsor
Novartis Pharmaceuticals
Target Recruit Count
20
Registration Number
NCT04812548
Locations
🇪🇸

Novartis Investigative Site, Barcelona, Catalunya, Spain

Venetoclax + Azacitidine vs. Induction Chemotherapy in AML

First Posted Date
2021-03-17
Last Posted Date
2024-03-18
Lead Sponsor
Massachusetts General Hospital
Target Recruit Count
172
Registration Number
NCT04801797
Locations
🇺🇸

City of Hope, Duarte, California, United States

🇺🇸

Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States

🇺🇸

Dana-Farber Cancer Institute, Boston, Massachusetts, United States

and more 6 locations
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