Drugs / 5-Azacytidine
last change Sep 2026 re-read 3 minutes ago

5-Azacytidine

also known as 5-AZA · AZA · azacitidine · Vidaza · Vidaza (5-azacytidine)

Small molecule

Developed for
myelodysplastic syndrome · acute myeloid leukemia · leukemia · central nervous system cancer · ependymoma · acute erythroid leukemia · acute graft versus host disease · B-cell chronic lymphocytic leukemia
+11 more · beta-thalassemia major · brain cancer · cerebral palsy · childhood acute myeloid leukemia · childhood ependymoma · childhood malignant neoplasm · chronic myelomonocytic leukemia · head and neck squamous cell carcinoma · high grade malignant neoplasm · myelodysplastic/myeloproliferative neoplasm · posterior fossa ependymoma
Investigated by
M.D. Anderson Cancer Center · Celgene Corporation · The University of Texas Health Science Center, Houston · Novartis Pharmaceuticals · Seagen Inc. · Tel-Aviv Sourasky Medical Center
+23 more · Alison Walker · Ariad Pharmaceuticals · Assistance Publique - Hôpitaux de Paris · Barbara Burtness · Baylor College of Medicine · Benjamin Tomlinson · Bristol-Myers Squibb · Center for Cell and Gene Therapy, Baylor College of Medicine · GIMEMA Foundation · Jeffrey Treiber · National Institute of Dental and Craniofacial Research (NIDCR) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · New Mexico Cancer Research Alliance · Patrick Stiff · Philippe ROUSSELOT · Rady Children's Hospital, San Diego · Shirley Ryan AbilityLab · The Hospital for Sick Children · The Methodist Hospital Research Institute · Therapeutic Advances in Childhood Leukemia Consortium · University of Pittsburgh · Virginia Commonwealth University · Weill Medical College of Cornell University

Trials 29

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 04 trials
Phase 0 NCT06598332 Aug 2025 → May 2028 acute graft versus host disease Baylor College of Medicine Withdrawn No outcome recorded Stop: Business
Phase 0 NCT04958486 Jul 2021 → Sep 2023 posterior fossa ependymoma The University of Texas Health Science Center, Houston Completed No outcome recorded
Phase 0 NCT04187703 Nov 2020 → Nov 2026 expected myelodysplastic syndrome, myelodysplastic/myeloproliferative neoplasm Benjamin Tomlinson Recruiting No outcome recorded
Phase 0 NCT02940483 Feb 2017 → Nov 2018 brain cancer The University of Texas Health Science Center, Houston Completed No outcome recorded
Phase 15 trials
Phase 1 NCT06377085 Jul 2025 → Dec 2026 expected cerebral palsy Shirley Ryan AbilityLab Enrolling by invitation No outcome recorded
Phase 1 NCT06466798 Jul 2024 → Jul 2027 central nervous system cancer, ependymoma, medulloblastoma Jeffrey Treiber Withdrawn No outcome recorded
Phase 1 NCT03572530 Feb 2019 → Nov 2020 ependymoma The University of Texas Health Science Center, Houston Completed No outcome recorded
Phase 1 NCT03206021 Aug 2017 → Aug 2022 central nervous system cancer, childhood ependymoma, childhood malignant neoplasm The Hospital for Sick Children Completed No outcome recorded
Phase 1 NCT00996515 Jun 2008 → Jul 2011 high grade malignant neoplasm New Mexico Cancer Research Alliance Completed No outcome recorded
Phase 1/26 trials
Phase 1/2 NCT03825367 Nov 2019 → May 2023 childhood acute myeloid leukemia Therapeutic Advances in Childhood Leukemia Consortium Unknown No outcome recorded
Phase 1/2 NCT02829840 Sep 2016 → Sep 2021 acute myeloid leukemia M.D. Anderson Cancer Center Withdrawn No outcome recorded
Phase 1/2 NCT02096042 Apr 2014 → Aug 2016 leukemia M.D. Anderson Cancer Center Terminated No outcome recorded Stop: Enrollment
Phase 1/2 NCT01202877 Mar 2011 → Jul 2015 leukemia M.D. Anderson Cancer Center Completed No outcome recorded
Phase 1/2 NCT00946647 Dec 2009 → Apr 2019 acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome Novartis Pharmaceuticals Completed No outcome recorded
Phase 1/2 NCT01038635 Dec 2009 → Nov 2015 leukemia M.D. Anderson Cancer Center Completed No outcome recorded
Phase 214 trials · 1 missed
Phase 2 NCT07522801 Sep 2026 → Dec 2029 expected — Philippe ROUSSELOT Not yet recruiting No outcome recorded
Phase 2 NCT05317000 Mar 2023 → Jun 2028 expected head and neck squamous cell carcinoma Barbara Burtness Recruiting No outcome recorded
Phase 2 NCT02204020 Apr 2015 acute myeloid leukemia, myelodysplastic syndrome University of Pittsburgh Withdrawn No outcome recorded Stop: Enrollment
Phase 2 NCT02497404 Feb 2015 → Jun 2020 acute erythroid leukemia, myelodysplastic syndrome Weill Medical College of Cornell University Completed No outcome recorded
Phase 2 NCT02124174 Jan 2012 → Jan 2022 acute myeloid leukemia, myelodysplastic syndrome Patrick Stiff Unknown No outcome recorded
Phase 2 NCT01053806 Aug 2011 → Mar 2014 myelodysplastic syndrome Tel-Aviv Sourasky Medical Center Unknown No outcome recorded
Phase 2 NCT00997243 Nov 2009 → Sep 2010 leukemia, myelodysplastic syndrome Alison Walker Terminated No outcome recorded
Phase 2 NCT00704704 Sep 2008 → Sep 2010 myelodysplastic syndrome Tel-Aviv Sourasky Medical Center Unknown No outcome recorded
Phase 2 NCT00721214 Jul 2008 → Jun 2014 myelodysplastic syndrome Virginia Commonwealth University Completed No outcome recorded
Phase 2 NCT00439673 May 2007 → Jul 2010 myelodysplastic syndrome GIMEMA Foundation Completed No outcome recorded
Comparator or background therapy2 trials
Phase 1 NCT03612739 Background Dec 2018 → May 2019 acute myeloid leukemia, myelodysplastic syndrome Celyad Oncology SA Withdrawn No outcome recorded Stop: Business
Phase 2 NCT00382590 Comparator Aug 2005 → Feb 2008 acute myeloid leukemia, myelodysplastic syndrome M.D. Anderson Cancer Center Completed No outcome recorded

Press releases naming this drug 15 releases

DateIssuerRelease
2023-12-10 AbbVie ImmunoGen Presents Findings from Newly Diagnosed Acute Myeloid Leukemia Cohorts in Phase 1b/2 Study of Pivekimab Sunirine in Combination with Azacitidine and Venetoclax at ASH abbvie.com ↗
2022-12-10 AbbVie ImmunoGen Presents Findings from Expansion Cohorts in Phase 1b/2 Study of Pivekimab Sunirine with Vidaza® and Venclexta® in Acute Myeloid Leukemia at ASH abbvie.com ↗
2021-12-12 AbbVie ImmunoGen Presents Initial Findings From the Phase 1b/2 Study of IMGN632 in Combination With Vidaza® and Venclexta® in Relapsed/Refractory Acute Myeloid Leukemia at ASH abbvie.com ↗
2021-11-04 AbbVie ImmunoGen to Present Initial Data Combining IMGN632 with Vidaza® and Venclexta® in Relapsed/Refractory Acute Myeloid Leukemia at ASH abbvie.com ↗
2021-07-21 Hoffmann-La Roche FDA grants Breakthrough Therapy Designation for Venclexta in combination with azacitidine for the treatment of patients with myelodysplastic syndromes roche.com ↗
2021-06-18 Bristol-Myers Squibb Bristol Myers Squibb Receives European Commission Approval for Onureg® (azacitidine tablets) as Frontline Oral Maintenance Therapy for Adults with Acute Myeloid Leukemia bms.com ↗
2021-04-23 Bristol-Myers Squibb Bristol Myers Squibb Receives Positive CHMP Opinion for Onureg® (azacitidine tablets; CC-486) as Frontline Oral Maintenance Therapy for Adults with Acute Myeloid Leukemia in First Remission bms.com ↗
2021-01-05 Samyang Biopharmaceuticals Corporation Samyang Biopharmaceuticals Corporation successfully launched its anti-cancer treatment drug Azacitidine Injection in the EU for the first time as a Korean company samyang.com ↗
2020-12-07 Bristol-Myers Squibb Bristol Myers Squibb Presents Analyses from Pivotal QUAZAR® AML-001 Study of Onureg® (azacitidine tablets; CC-486) in Adults with Acute Myeloid Leukemia in First Remission bms.com ↗
2020-09-01 Bristol-Myers Squibb U.S. Food and Drug Administration Approves Onureg® (azacitidine tablets), a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia bms.com ↗
2019-02-28 Lupin Limited Lupin receives FDA approval for Azacitidine for Injection, 100 mg Single-Dose Vial lupin.com ↗
2018-10-10 BeOne Medicines BeiGene Announces National Reimbursement Inclusion of VIDAZA® (Azacitidine for Injection) by the State Medical Insurance Administration in China beonemedicines.com ↗

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 31 sources stand behind the page.

FieldValueCited text
Known as 5-azacytidine “5-azacytidine can increase fetal hemoglobin levels, but this drug can damage DNA, which in turn can increase the risk of cancer.” NCT00005934 ↗
33

“5-azacytidine (Vidaza®) + Valproic Acid (Depakine ®) Before Administration of Retinoic Acid (Vesanoid®)” NCT00339196 ↗

“5-azacytidine (Vidaza®) + Valproic Acid (Depakine ®) Before Administration of Retinoic Acid (Vesanoid®)” NCT00339196 ↗

“the combination of 5-Aza-Cytidine + Thalidomide in hrMDS patients.” NCT00704704 ↗

“5-azacytidine, 75 mg/m² sub cut QD started from day5 to day10” NCT07522801 ↗

“Days 1-5: 5-Azacytidine 40 mg/m\^2 daily” NCT02124174 ↗

“5-azacytidine (AZA)” NCT03612739 ↗

NCT00382590 ↗

NCT00721214 ↗

NCT02096042 ↗

NCT03572530 ↗

Show 23 more sources

Known as 5-AZA “5-Azacytidine (5-AZA) is a chemotherapy, and nivolumab is an immunotherapy.” NCT05317000 ↗
4

“5-Azacytidine (5-AZA) and Trastuzumab Infusions Into the Fourth Ventricle” NCT04958486 ↗

“5-azacytidine (5-aza) maintenance therapy after transplant” NCT02204020 ↗

“5-Azacytidine (5-AZA) group 1” NCT03572530 ↗

“Drug: 5-Azacytidine (5-aza)” NCT00326170 ↗

Known as AZA “this drug, called 5-Azacytidine (AZA).” NCT06377085 ↗
1

“5-azacytidine (AZA)” NCT03612739 ↗

Known as azacitidine “I. To document the toxicities, and reversibility of toxicities, of this regimen of 5-azacytidine (azacitidine) and erlotinib (erlotinib hydrochloride).” NCT00996515 ↗
Known as Vidaza “5-azacytidine (Vidaza®) + Valproic Acid (Depakine ®) Before Administration of Retinoic Acid (Vesanoid®)” NCT00339196 ↗
Known as Vidaza (5-azacytidine) “Vidaza (5-azacytidine)” NCT00996515 ↗
Action Activate “reactivate gene transcription through DNA demethylation” NCT00339196 ↗
Modality Small molecule “5-azacytidine is a nucleoside analogue with hypomethylating activity” NCT00326170 ↗
Route Intravenous “An intravenous (IV) catheter (flexible tube inserted into a vein) is placed in a large vein of the patient's neck, chest or arm for infusion of 5-azacytidine at a constant rate...” NCT00005934 ↗