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Therapy Name Azacitidine
Synonyms
Therapy Description

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 and Onureg (azacitidine) is FDA approved for use in continued treatment of acute myeloid leukemia(FDA.gov).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Azacitidine Vidaza azacytidine|CC-486|5-azacytidine|5-AC|U-18496|Onureg 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 and Onureg (azacitidine) is FDA approved for use in continued treatment of acute myeloid leukemia(FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
IDH1 R132H anaplastic 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
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 mutant acute myeloid leukemia sensitive Azacitidine Guideline Actionable Vidaza (azacitidine) is included in guidelines for adult patients with acute myeloid leukemia harboring an IDH1 mutation (NCCN.org). detail...
IDH2 mutant acute myeloid leukemia sensitive Azacitidine Guideline Actionable Vidaza (azacitidine) is included in guidelines for adult patients with acute myeloid leukemia harboring an IDH2 mutation (NCCN.org). detail...

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
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 USA | ITA | FRA | ESP | DEU | BEL 0
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 USA | TUR | POL | ITA | ISR | GBR | FRA | ESP | DEU | CAN | BEL 3
NCT03573024 Phase II Venetoclax Azacitidine Venetoclax and Azacitidine for Non-Elderly Adult Patients With Acute Myeloid Leukemia Active, not recruiting USA 0
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 USA 0
NCT03151408 Phase III Azacitidine Azacitidine + Pracinostat An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia Terminated USA | ROU | POL | ITA | HUN | GBR | FRA | ESP | DEU | CZE | BRA | AUT | AUS | ARG 2
NCT02129101 Phase I Azacitidine Sonidegib Azacitidine and Sonidegib in Treating Patients With Myeloid Malignancies Completed USA 0
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 Completed USA 0
NCT04712942 Phase II Azacitidine + MLN4924 Azacitidine Treatment of MDS/AML Patients With an Impending Hematological Relapse With AZA or ATA and Pevonedistat Completed DEU 0
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 0
NCT05317000 Phase I Azacitidine Azacitidine + Nivolumab Nivolumab 5-Azacytidine and/or Nivolumab in Resectable HPV-Associated HNSCC Recruiting USA 0
NCT02530463 Phase II Nivolumab Azacitidine Ipilimumab Nivolumab and Ipilimumab With 5-azacitidine in Patients With Myelodysplastic Syndromes (MDS) Active, not recruiting USA 0
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 Completed USA 0
NCT02281084 Phase II Azacitidine Azacitidine + Durvalumab Safety and Efficacy Study of CC-486 and Durvalumab in Subjects With Myelodysplastic Syndromes Completed USA | POL | ITA | GBR | FRA | ESP | DEU | CAN | BEL | AUS 0
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 Active, not recruiting USA 0
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 USA 0
NCT02494258 Phase II Azacitidine A Study to Evaluate Long-term Safety in Subjects With Solid Tumors and Hematological Disorders. Active, not recruiting USA | GBR 0
NCT02178072 Phase II Azacitidine Window Trial 5-aza in HNSCC, T-tare Completed USA 0
NCT03151304 Phase II Azacitidine + Pracinostat Azacitidine A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes Terminated USA 0
NCT03926624 Phase III Decitabine + Venetoclax Azacitidine + Venetoclax Cytarabine + Filgrastim + Fludarabine + Idarubicin Cytarabine + Venetoclax Cytarabine + Filgrastim + Fludarabine Decitabine Azacitidine Cladribine + Cytarabine + Filgrastim + Mitoxantrone DFP-10917 Cladribine + Cytarabine + Filgrastim + Idarubicin Cytarabine Cytarabine + Etoposide + Mitoxantrone Trial of DFP-10917 vs Non-Intensive or Intensive Reinduction for AML Patients in 2nd/3rd/4th Salvage Unknown status USA 0
NCT03745352 Phase II Azacitidine Azacitidine + MLN4924 Pevonedistat With Azacitidine Versus Azacitidine Alone in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia Withdrawn 0
NCT03092674 Phase II Cytarabine + Decitabine Azacitidine + Midostaurin Decitabine Azacitidine + Nivolumab Azacitidine Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Active, not recruiting USA 0
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) Completed USA | POL | NLD | ITA | GBR | FRA | ESP | DEU | CAN | BEL | AUT 1
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) Completed USA | NLD | ITA | ISR | IRL | FRA | ESP | DEU | CZE | CAN | BGR | BEL 0
NCT02599649 Phase II Nivolumab Azacitidine Lirilumab Lirilumab and Nivolumab With 5-Azacitidine in Patients With Myelodysplastic Syndromes (MDS) Terminated USA 0
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 Sulfate Dasatinib Mitoxantrone Vinblastine Cytarabine Tretinoin Clofarabine Teniposide Docetaxel Pazopanib Oxaliplatin Streptozocin Paclitaxel Bendamustine Mechlorethamine Mitotane Genomic Based Assignment of Therapy in Advanced Urothelial Carcinoma Completed USA 0
NCT02677922 Phase Ib/II Azacitidine + Ivosidenib Azacitidine Azacitidine + Enasidenib A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy Active, not recruiting USA | SWE | NLD | ITA | GBR | FRA | ESP | DEU | CHE | CAN | BEL | AUS 2
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 Terminated USA 0
NCT02204020 Phase II Azacitidine Phase II Study of 5-azacytidine Maintenance After Transplant for AML or MDS Withdrawn USA 0
NCT01928576 Phase II Nivolumab Entinostat Azacitidine Phase II Anti-PD1 Epigenetic Priming Study in NSCLC. Completed USA 0
NCT04313881 Phase III Azacitidine Azacitidine + Hu5F9-G4 Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS) (ENHANCE) Terminated USA | TUR | POL | NZL | NOR | NLD | ITA | HUN | GBR | FRA | FIN | ESP | DEU | CZE | CHE | CAN | BEL | AUT | AUS 2
NCT06196203 Phase II Azacitidine AK117 + Azacitidine A Study of AK117 in Combination With Azacitidine in Patients With Myelodysplastic Syndromes Recruiting USA 1
NCT02469415 Phase II Decitabine Azacitidine Pacritinib Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes (MDS) Terminated USA 0
NCT02088541 Phase II Cytarabine Azacitidine Decitabine Selinexor Hydroxyurea Selinexor (KPT-330) in Older Patients With Relapsed AML Completed USA | POL | NLD | ITA | ISR | GBR | FRA | ESP | DNK | DEU | CAN 0
NCT02343536 Phase I Cyclophosphamide + Prednisone + Rituximab + Vincristine Sulfate 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 Completed USA 0
NCT04090736 Phase III Azacitidine Azacitidine + MLN4924 Study to Compare Azacitidine Plus Pevonedistat Versus Azacitidine in Patients With Acute Myeloid Leukemia Not Eligible for Standard Chemotherapy (PEVOLAM) Unknown status ESP 1
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) Active, not recruiting USA | ITA | GBR | FRA | DEU | CAN | AUS 0
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) Completed USA | SWE | ROU | POL | ITA | ISR | HUN | GBR | FRA | ESP | DEU | CZE | CAN | BEL | AUT | AUS 6
NCT01720225 Phase II Decitabine Azacitidine Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk Completed USA 0
NCT03946670 Phase II Decitabine + Sabatolimab Azacitidine Azacitidine + Sabatolimab Decitabine A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS). Completed USA | TUR | ITA | HUN | GRC | GBR | FRA | ESP | DEU | CZE | CAN | BEL | AUT 4
NCT03173248 Phase III Azacitidine Azacitidine + Ivosidenib Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation (AGILE) Active, not recruiting USA | POL | NLD | ITA | ISR | GBR | FRA | ESP | DEU | CZE | CAN | BRA | AUT | AUS 6
NCT04266301 Phase III Azacitidine Azacitidine + Sabatolimab 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) (STIMULUS-MDS2) Completed USA | TUR | NLD | LTU | LBN | ITA | ISR | GRC | GBR | FRA | FIN | ESP | DEU | CZE | CHE | CAN | BRA | BEL | AUT | AUS | ARG 15
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) Completed USA | TUR | POL | ITA | ISR | GRC | GBR | FRA | ESP | DEU | CZE | CAN | BRA | BEL | AUS 5
NCT02920008 Phase III Decitabine Azacitidine Cytarabine Guadecitabine Phase 3 Randomized, Open-Label Study of Guadecitabine vs Treatment Choice in Previously Treated Acute Myeloid Leukemia Completed USA | SWE | POL | ITA | HUN | GBR | FRA | ESP | DNK | DEU | CAN | BEL 3
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 USA | SWE | ROU | POL | NLD | ITA | HUN | GBR | FRA | FIN | ESP | DNK | DEU | CZE | CAN | BGR | BEL | AUT | AUS 5
NCT06641414 Phase III Azacitidine APG-2575 + Azacitidine Lisaftoclax (APG-2575) Combined With Azacytidine (AZA) in the Treatment of Patients With Higher-risk Myelodysplastic Syndrome (GLORA-4). Not yet recruiting USA 1
NCT02400281 Phase Ib/II Azacitidine Crenolanib Cytarabine + Idarubicin Study of Crenolanib Combined With Chemotherapy in FLT3-mutated Acute Myeloid Leukemia Patients Completed USA 0
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 Active, not recruiting USA | TUR | SWE | POL | NOR | ITA | ISR | HUN | HRV | FRA | FIN | ESP | DNK | DEU | CZE | CAN | BRA | BEL | AUT | AUS 9
NCT00887068 Phase III Azacitidine Randomized Allogeneic Azacitidine Study Completed USA 0
NCT02269943 Phase II Azacitidine Safety and Efficacy of Oral Azacitidine in Previously Treated Subjects With Locally Advanced or Metastatic Nasopharyngeal Carcinoma Completed USA | ITA | GRC | FRA | ESP | CAN 3
NCT02828358 Phase II Cytarabine + Hydrocortisone + Leucovorin + Mercaptopurine + Pegaspargase Cyclophosphamide + Cytarabine + Hydrocortisone + Mercaptopurine + Methotrexate Cytarabine + Hydrocortisone + Mercaptopurine + Methotrexate Azacitidine Cytarabine + Daunorubicin + Dexamethasone + Hydrocortisone + Pegaspargase + Thioguanine + Vincristine sulfate liposome Cytarabine + Daunorubicin + Dexamethasone + Hydrocortisone + Methotrexate + Pegaspargase + Prednisolone + Vincristine Sulfate Mercaptopurine + Methotrexate Cyclophosphamide + Cytarabine + Thioguanine Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement Active, not recruiting USA | CAN | AUS 1
NCT04797780 Phase III Azacitidine Azacitidine + Tamibarotene Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome Recruiting USA | POL | ITA | ISR | HUN | GBR | FRA | ESP | DEU | CZE | CAN | BEL | AUT 0
NCT03745716 Phase III APR-246 + Azacitidine Azacitidine APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS) Completed USA | FRA 0
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) Completed USA | TUR | ITA | GBR | FRA | ESP | DNK | DEU | CZE | CAN | BRA | BEL | AUT | AUS 4
NCT04264806 Phase II ARGX-110 + Azacitidine Azacitidine 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) Withdrawn TUR | ITA | GBR | FRA | ESP | DEU | CHE | BRA | AUS 2
NCT04229979 Phase III Galinpepimut-S Cytarabine Decitabine Venetoclax Azacitidine Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2 (REGAL) Active, not recruiting USA | POL | HUN | GRC | FRA | ESP | DEU 3
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). Completed USA | SWE | ITA | HUN | GBR | FRA | ESP | DEU | CHE | CAN | BEL | AUT 2
NCT02752035 Phase II Gilteritinib Azacitidine + Gilteritinib Azacitidine A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy Active, not recruiting USA | POL | ITA | GBR | FRA | ESP | DEU | CAN | BEL | AUS 3
NCT02721875 Phase I Azacitidine Volasertib Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes Terminated 1
NCT04401748 Phase III Azacitidine + Venetoclax Azacitidine Safety And Efficacy Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Complete Remission and Overall Survival In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome (Verona) Active, not recruiting USA | TUR | POL | NLD | ITA | ISR | HUN | GBR | FRA | ESP | DEU | CZE | CAN | BRA | BEL | AUT | AUS 6


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