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 Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome
- Conditions
- Interventions
- First Posted Date
- 2020-05-26
- Last Posted Date
- 2024-06-20
- Lead Sponsor
- AbbVie
- Target Recruit Count
- 531
- Registration Number
- NCT04401748
- Locations
- 🇺🇸
Texas Oncology- Baylor Charles A. Sammons Cancer Center /ID# 223727, Dallas, Texas, United States
🇺🇸UPMC Hillman Cancer Ctr /ID# 223201, Pittsburgh, Pennsylvania, United States
🇨🇳China Medical University Hospital /ID# 218987, Taichung, Taiwan
A Safety and Efficacy Study of CC-90009 Combinations in Subjects With Acute Myeloid Leukemia
- Conditions
- Interventions
- First Posted Date
- 2020-04-07
- Last Posted Date
- 2024-05-31
- Lead Sponsor
- Celgene
- Target Recruit Count
- 22
- Registration Number
- NCT04336982
- Locations
- 🇺🇸
Local Institution - 104, San Francisco, California, United States
🇺🇸Local Institution - 103, Boston, Massachusetts, United States
🇨🇦Local Institution - 201, Toronto, Ontario, Canada
Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS)
- Conditions
- Interventions
- First Posted Date
- 2020-03-18
- Last Posted Date
- 2024-06-27
- Lead Sponsor
- Gilead Sciences
- Target Recruit Count
- 539
- Registration Number
- NCT04313881
- Locations
- 🇺🇸
Thomas Jefferson University, Sidney Kimmel Cancer Center; Clinical Research Organization, Philadelphia, Pennsylvania, United States
🇺🇸Swedish Cancer Institute, Seattle, Washington, United States
🇺🇸Winship Cancer Institute, Atlanta, Georgia, United States
BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 2- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Azacitidine and Venetoclax as Frontline Therapy in Unfit Patients With Acute Myeloid Leukemia
- Conditions
- Interventions
- First Posted Date
- 2020-02-26
- Last Posted Date
- 2024-12-10
- Lead Sponsor
- National Cancer Institute (NCI)
- Target Recruit Count
- 76
- Registration Number
- NCT04284787
- Locations
- 🇺🇸
Smilow Cancer Hospital Care Center at Saint Francis, Hartford, Connecticut, United States
🇺🇸University of Kansas Hospital-Westwood Cancer Center, Westwood, Kansas, United States
🇺🇸Johns Hopkins University/Sidney Kimmel Cancer Center, Baltimore, Maryland, United States
A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.
- Conditions
- Interventions
- First Posted Date
- 2020-02-19
- Last Posted Date
- 2024-10-11
- Lead Sponsor
- Ascentage Pharma Group Inc.
- Target Recruit Count
- 102
- Registration Number
- NCT04275518
- Locations
- 🇨🇳
Xiangya Hospital Central South University, Changsha, Hunan, China
🇨🇳The First Affilated Hospital of Ganzhou Medical University, Suzhou, Jiangsu, China
🇨🇳Guangzhou panyu central hospital, Guangzhou, Guangdong, China
Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)
- Conditions
- Interventions
- First Posted Date
- 2020-02-12
- Last Posted Date
- 2024-12-12
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 530
- Registration Number
- NCT04266301
- Locations
- 🇺🇸
University Of Miami, Miami, Florida, United States
🇺🇸Northwestern University, Chicago, Illinois, United States
🇺🇸Massachusetts General Hospital, Boston, Massachusetts, United States
A Study of Pevonedistat and Venetoclax Combined With Azacitidine to Treat Acute Myeloid Leukemia (AML) in Adults Unable to Receive Intensive Chemotherapy
- Conditions
- Interventions
- First Posted Date
- 2020-02-12
- Last Posted Date
- 2024-12-17
- Lead Sponsor
- Takeda
- Target Recruit Count
- 164
- Registration Number
- NCT04266795
- Locations
- 🇺🇸
Houston Methodist Cancer Center, Houston, Texas, United States
🇺🇸UC Irvine Medical Center, Orange, California, United States
🇺🇸University Hospitals Cleveland Medical Center, Cleveland, Ohio, United States
A Study of Cusatuzumab in Combination With Azacitidine Compared With Azacitidine Alone in Patients With Higher-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) and Who Are Not Candidates for Hematopoietic Stem Cell Transplantation (HSCT)
- Conditions
- Interventions
- First Posted Date
- 2020-02-11
- Last Posted Date
- 2022-05-20
- Lead Sponsor
- Janssen Research & Development, LLC
- Registration Number
- NCT04264806
- Locations
- 🇸🇦
King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia
🇨🇭UniversitaetsSpital Zuerich, Zürich, Switzerland
🇦🇺St Vincents Hospital Sydney, Darlinghurst, Australia
Safety and Tolerance of Epigenetic and Immunomodulating Drugs Combined With Chemotherapeutics in Patients Suffering From Advanced Pancreatic Cancer
- Conditions
- Interventions
- First Posted Date
- 2020-02-06
- Last Posted Date
- 2023-05-01
- Lead Sponsor
- GWT-TUD GmbH
- Target Recruit Count
- 75
- Registration Number
- NCT04257448
- Locations
- 🇩🇪
Universitätsklinikum Essen, Essen, Germany
🇩🇪Universitätsmedizin Göttingen, Göttingen, Germany
🇩🇪Universitätsklinikum Hamburg-Eppendorf, Hamburg, Germany
A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS, CMML, or AML
- Conditions
- Interventions
- First Posted Date
- 2020-02-05
- Last Posted Date
- 2024-10-31
- Lead Sponsor
- Taiho Oncology, Inc.
- Target Recruit Count
- 317
- Registration Number
- NCT04256317
- Locations
- 🇺🇸
Keck School of Medicine of USC, Los Angeles, California, United States
🇺🇸Yale University, New Haven, Connecticut, United States
🇺🇸MedStar Georgetown University Hospital, Washington, District of Columbia, United States
Drug Development Updates
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