Therapy Detail

Therapy Name Azacitidine
Therapy Description


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Drug Name Trade Name Synonyms Drug Classes Drug Description
Azacitidine Vidaza azacytidine|CC-486|5-azacytidine|5-AC|U-18496 DNMT inhibitor (Pan) 5 Vidaza (azacitidine) is a cytidine analog that incorporates into DNA and RNA and binds to DNA methyltransferases (DNMTs), resulting in DNMT degradation and decreased DNA methylation, and leading to increased tumor cell death (PMID: 28159832, PMID: 28067760). Vidaza (azacitidine) is FDA-approved for use in patients with some subtypes of myelodysplastic syndrome (
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
TET2 mutant acute myeloid leukemia predicted - sensitive Azacitidine Phase I Actionable In a retrospective analysis, presence of a TET2 mutation correlated with higher overall response rate (ORR) in patients with myelodysplastic syndrome and acute myeloid leukemia following treatment with Vidaza (azacitidine), with an ORR of 85% (11/3) in TET2-mutated patients, compared to 47% (34/47) in patients with wild-type TET2, but did not correlate with overall survival (PMID: 21494260). 21494260
IDH1 R132H grade III astrocytoma sensitive Azacitidine Preclinical Actionable In a preclinical study, long-term treatment with Vidaza (azacitidine) resulted in increased cellular differentiation, decreased proliferation, and tumor regression in a patient-derived xenograft (PDX) model of anaplastic astrocytoma harboring IDH1 R132H (PMID: 24077805). 24077805
Unknown unknown myelodysplastic syndrome not applicable Azacitidine FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with Vidaza (azacitidine) resulted in an overall response rate of 16.2% (16/99) in patients with myelodysplastic syndromes, compared to an overall response rate of 0% (0/92) in the observation only arm (PMID: 15897554). 15897554
ASXL1 wild-type TET2 mutant myelodysplastic/myeloproliferative neoplasm sensitive Azacitidine Clinical Study - Cohort Actionable In a clinical study, myelodysplastic syndrome patients carrying TET2 mutations and wild-type ASXL1 demonstrated an increased response to treatment with the DNA hypomethylating agents Dacogen (decitabine) or Vidaza (azacitidine) compared to patients with other mutational profiles (65% vs. 44%; OR 2.37, P=0.49) (PMID: 25224413). 25224413
Clinical Trial Phase Therapies Title Recruitment Status
NCT03397173 Azacitidine TET2 Mutations in Myelodysplastic Syndromes and Acute Myeloid Leukemia With Azacitidine + Ascorbic Acid Recruiting
NCT00887068 Phase III Azacitidine Randomized Allogeneic Azacitidine Study Completed
NCT02269943 Phase II Azacitidine Safety and Efficacy of Oral Azacitidine in Previously Treated Subjects With Locally Advanced or Metastatic Nasopharyngeal Carcinoma Completed
NCT02400281 Phase Ib/II Azacitidine Crenolanib Cytarabine + Idarubicin Study of Crenolanib Combined With Chemotherapy in FLT3-mutated Acute Myeloid Leukemia Patients Active, not recruiting
NCT02265510 Phase I Gemcitabine Carfilzomib Dexamethasone Lenalidomide Nab-paclitaxel Azacitidine INCB052793 Bortezomib An Open-Label Study of a Novel JAK-inhibitor, INCB052793, Given to Patients With Advanced Malignancies Active, not recruiting
NCT01814826 Phase I MLN4924 Azacitidine Study of MLN4924 Plus Azacitidine in Treatment-Naïve Patients With Acute Myelogenous Leukemia (AML) Who Are 60 Years or Older Completed
NCT02920008 Phase III Decitabine Azacitidine Cytarabine Guadecitabine Phase 3 Randomized, Open-Label Study of Guadecitabine vs Treatment Choice in Previously Treated Acute Myeloid Leukemia Active, not recruiting
NCT01993641 Phase II Pracinostat Azacitidine Decitabine Phase 2 Study Adding Pracinostat to a Hypomethylating Agent (HMA) in Patients With MDS Who Failed to Respond to Single Agent HMA Completed
NCT02088541 Phase II Cytarabine Azacitidine Decitabine Selinexor Hydroxyurea Selinexor (KPT-330) in Older Patients With Relapsed AML Completed
NCT01928576 Phase II Nivolumab Entinostat Azacitidine Phase II Anti-PD1 Epigenetic Priming Study in NSCLC. Recruiting
NCT02752035 Phase II Gilteritinib Azacitidine + Gilteritinib Azacitidine A Study of ASP2215 (Gilteritinib), Combination of ASP2215 Plus Azacitidine and Azacitidine Alone in the Treatment of Newly Diagnosed Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation in Patients Not Eligible for Intensive Induction Chemotherapy Recruiting
NCT03946670 Phase II Decitabine + MBG453 Azacitidine Azacitidine + MBG453 Decitabine A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS). Recruiting
NCT03268954 Phase III Azacitidine + MLN4924 Azacitidine Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML) (PANTHER) Recruiting
NCT02721875 Phase I Azacitidine Volasertib Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes Terminated
NCT03151408 Phase III Azacitidine Azacitidine + Pracinostat An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia Recruiting
NCT02530463 Phase II Nivolumab Azacitidine Ipilimumab Nivolumab and Ipilimumab With 5-azacitidine in Patients With Myelodysplastic Syndromes (MDS) Recruiting
NCT02421939 Phase III Cytarabine Cytarabine + Fludarabine + Idarubicin Cytarabine + Etoposide + Mitoxantrone Azacitidine Gilteritinib Filgrastim A Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase (FLT3) Mutation Active, not recruiting
NCT02469415 Phase II Decitabine Azacitidine Pacritinib Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes (MDS) Terminated
NCT03092674 Phase II Cytarabine + Decitabine Azacitidine + Midostaurin Azacitidine + Nivolumab Azacitidine Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Suspended
NCT02599649 Phase II Nivolumab Azacitidine Lirilumab Lirilumab and Nivolumab With 5-Azacitidine in Patients With Myelodysplastic Syndromes (MDS) Completed
NCT03164057 Phase II Etoposide Filgrastim Dexrazoxane Daunorubicin Idarubicin Fludarabine Asparaginase Cytarabine Sorafenib Mitoxantrone Decitabine Azacitidine A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia Recruiting
NCT02610777 Phase II Azacitidine MLN4924 An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML) Active, not recruiting
NCT02343536 Phase I Cyclophosphamide + Prednisone + Rituximab + Vincristine Azacitidine A Phase 1, Open-label,Trial of Oral Azacitidine (CC-486) Plus R-CHOP in Subjects With Large B-Cell Lymphoma or Follicular Lymphoma Active, not recruiting
NCT02546986 Phase II Azacitidine Pembrolizumab Safety and Efficacy Study of CC-486 With MK-3475 to Treat Locally Advanced or Metastatic Non-small Cell Lung Cancer Active, not recruiting
NCT02701673 Phase Ib/II Azacitidine Pyridoxine Gemcitabine Belinostat Busulfan Melphalan Belinostat Combined With Azacitidine/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Refractory or Relapsed Lymphoma Withdrawn
NCT03151304 Phase II Azacitidine + Pracinostat Azacitidine A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes Active, not recruiting
NCT02348489 Phase III Guadecitabine Cytarabine Azacitidine Decitabine SGI-110 in Adults With Untreated Acute Myeloid Leukemia (AML), Not Considered Candidates for Intensive Remission Induction Completed
NCT02508870 Phase I Azacitidine Atezolizumab A Safety and Pharmacology Study of Atezolizumab (MPDL3280A, Anti-PD-L1 Antibody) Administered Alone or in Combination With Azacitidine in Patients With Myelodysplastic Syndromes Suspended
NCT02775903 Phase II Azacitidine Azacitidine + Durvalumab An Efficacy and Safety Study of Azacitidine Subcutaneous in Combination With Durvalumab (MEDI4736) in Previously Untreated Subjects With Higher-Risk Myelodysplastic Syndromes (MDS) or in Elderly Subjects With Acute Myeloid Leukemia (AML) Active, not recruiting
NCT02129101 Phase I Azacitidine Sonidegib Azacitidine and Sonidegib in Treating Patients With Myeloid Malignancies Active, not recruiting
NCT02788201 Phase II Erlotinib Thiotepa Imatinib Dacarbazine Arsenic trioxide Idarubicin Mitomycin C Thioguanine Mercaptopurine Methotrexate Cladribine Epirubicin Gemcitabine Doxorubicin Bleomycin Etoposide Gefitinib Daunorubicin Lomustine Sorafenib Sunitinib Ifosfamide Asparaginase Ixabepilone Abiraterone Azacitidine Ruxolitinib Decitabine Axitinib Estramustine Floxuridine Lapatinib Carmustine Fludarabine Nilotinib Cisplatin Vismodegib Vandetanib Melphalan Busulfan Carboplatin Toremifene Crizotinib Dactinomycin Temsirolimus Vorinostat Romidepsin Fluorouracil Irinotecan Bortezomib Tamoxifen Topotecan Chlorambucil Pentostatin Eribulin Carfilzomib Vemurafenib Hydroxyurea Exemestane Vincristine Dasatinib Mitoxantrone Vinblastine Cytarabine Tretinoin Clofarabine Teniposide Docetaxel Pazopanib Oxaliplatin Streptozocin Paclitaxel Bendamustine Mechlorethamine Mitotane Genomic Based Assignment of Therapy in Advanced Urothelial Carcinoma Recruiting
NCT02178072 Phase II Azacitidine Window Trial 5-aza in HNSCC, T-tare Recruiting
NCT02942290 Phase II Azacitidine Azacitidine + Venetoclax A Study Evaluating Venetoclax in Combination With Azacitidine Compared With Azacitidine Alone in Participants With Previously Untreated Higher-Risk Myelodysplastic Syndromes (MDS) Recruiting
NCT03573024 Phase II Venetoclax Azacitidine Venetoclax and Azacitidine for Non-Elderly Adult Patients With Acute Myeloid Leukemia Recruiting
NCT01787487 Phase III Azacitidine Ruxolitinib Evaluation of RUX and AZA Combination as a Therapy For Patients With Myelofibrosis And Myelodysplastic Syndrome/ Myeloproliferative Neoplasm Recruiting
NCT02577406 Phase III Azacitidine Cytarabine Enasidenib An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation (IDHENTIFY) Recruiting
NCT02551718 Phase I Bosutinib Irinotecan Romidepsin Busulfan Melphalan Nilotinib Crizotinib Cytarabine Mitoxantrone Dasatinib Pazopanib Paclitaxel Clofarabine Hydroxyurea Tretinoin Carfilzomib Nelarabine Bexarotene Pentostatin Everolimus Cabozantinib Mercaptopurine Methotrexate Cladribine Thioguanine Daunorubicin Ponatinib Etoposide Afatinib Gefitinib Gemcitabine Regorafenib Arsenic trioxide Trametinib Imatinib Erlotinib Dabrafenib Decitabine Axitinib Azacitidine Ruxolitinib Fludarabine Lapatinib Ceritinib Sirolimus Sorafenib Lomustine Sunitinib Cabazitaxel Temsirolimus Topotecan Bortezomib Pralatrexate High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia Recruiting
NCT02374099 Phase II Azacitidine Fulvestrant Study to Assess the Efficacy and Safety of the Epigenetic Modifying Effects of CC-486 (Oral Azacitidine) in Combination With Fulvestrant Terminated
NCT03416179 Phase III Cytarabine Cytarabine + Daunorubicin + Glasdegib Azacitidine + Glasdegib Azacitidine Cytarabine + Daunorubicin A Study Evaluating Intensive Chemotherapy With or Without Glasdegib or Azacitidine With or Without Glasdegib In Patients With Previously Untreated Acute Myeloid Leukemia (BRIGHT AML1019) Recruiting
NCT00946647 Phase II Panobinostat Azacitidine A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML). Active, not recruiting
NCT01720225 Phase II Decitabine Azacitidine Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk Active, not recruiting
NCT02677922 Phase Ib/II Azacitidine + Ivosidenib Azacitidine Azacitidine + Enasidenib A Safety and Efficacy Study of Oral AG-120 Plus Subcutaneous Azacitidine and Oral AG-221 Plus Subcutaneous Azacitidine in Subjects With Newly Diagnosed Acute Myeloid Leukemia (AML) Active, not recruiting
NCT02993523 Phase III Azacitidine + Venetoclax Azacitidine A Study of Venetoclax in Combination With Azacitidine Versus Azacitidine in Treatment Naïve Elderly Subjects With Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Therapy Recruiting
NCT02204020 Phase II Azacitidine Phase II Study of 5-azacytidine Maintenance After Transplant for AML or MDS Withdrawn
NCT02281084 Phase II Azacitidine Azacitidine + Durvalumab Safety and Efficacy Study of CC-486 and Durvalumab in Subjects With Myelodysplastic Syndromes Active, not recruiting
NCT03745716 Phase III APR-246 + Azacitidine Azacitidine APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS) Recruiting