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Home › Medications › azacitidine

azacitidine

6 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials of azacitidine

82 trials tracked of azacitidine: 16 in phase 3 or 4 and 9 with published results. The most-cited published study has 1233 citations.

82Trials tracked
16Phase 3 & 4
0Recruiting
9With published results
Phase distribution
Phase 4 1 Phase 3 15 Phase 2 55 Phase 1 10 Other / NA 1
  1. Phase 3 A Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase (FLT3) Mutation Completed · 1,233 cited
  2. Phase 3 Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129) Completed · 211 cited
  3. Phase 3 A Study of Venetoclax in Combination With Azacitidine Versus Azacitidine in Treatment Naïve Participants With Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Therapy Completed · 143 cited
  4. Phase 3 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 Completed · 124 cited
  5. Phase 3 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) Completed · 120 cited
  6. Phase 3 A Study Evaluating Intensive Chemotherapy With or Without Glasdegib or Azacitidine With or Without Glasdegib In Patients With Previously Untreated Acute Myeloid Leukemia Completed · 48 cited
Show 44 more trials
  1. Phase 3 A Study of Oral Venetoclax Tablets and Oral Azacitidine as Maintenance Therapy in Adult Participants With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy Completed · 15 cited
  2. Phase 3 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 Completed · 12 cited
  3. Phase 3 A Study of the Effectiveness of Venetoclax in Combination With Azacitidine or Decitabine in an Outpatient Setting in Patients With Acute Myeloid Leukemia (AML) Ineligible for Intensive Chemotherapy Completed · 10 cited
  4. Phase 4 Study of Azacitidine in Adult Taiwanese Subjects With Higher-Risk Myelodysplastic Syndromes (MDS) Completed
  5. Phase 3 APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS) Completed
  6. Phase 3 Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML Completed
  7. Phase 3 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 Completed
  8. Phase 3 Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML) Completed
  9. Phase 3 A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care Completed
  10. Phase 3 An Extension to Study AZA PH GL 2003 CL 001 Allowing for Continuation of Azacitidine Treatment in Patients With Myelodysplastic Syndromes (MDS) Completed
  11. Phase 2 Azacitidine and Pembrolizumab in Pancreatic Cancer Completed
  12. Phase 2 A Study of FT-2102 in Patients With Advanced Solid Tumors and Gliomas With an IDH1 Mutation Completed
  13. Phase 2 Salsalate, Venetoclax, and Decitabine or Azacitidine for the Treatment of Acute Myeloid Leukemia or Advanced Myelodysplasia/Myeloproliferative Disease Completed
  14. Phase 2 Safety and Efficacy Study of Pracinostat With Azacitadine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) Completed
  15. Phase 2 Azacitidine and Lenalidomide for Relapsed and Refractory Patients With Acute Myeloid Leukemia Completed
  16. Phase 2 Phase 1-2 of Azacitidine + Lenalidomide for Previously Untreated Elderly Patients With Acute Myeloid Leukemia (AML) Completed
  17. Phase 2 Study of Crenolanib Combined With Chemotherapy in FLT3-mutated Acute Myeloid Leukemia Patients Completed
  18. Phase 2 Azacitidine With or Without Entinostat in Treating Patients With Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Completed
  19. Phase 2 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
  20. Phase 2 Pinometostat and Azacitidine in Treating Patients With Relapsed, Refractory, or Newly Diagnosed Acute Myeloid Leukemia With 11q23 Rearrangement Completed
  21. Phase 2 OX40, Venetoclax, Avelumab, Glasdegib, Gemtuzumab Ozogamicin, and Azacitidine in Relapsed or Refractory Acute Myeloid Leukemia Completed
  22. Phase 2 Alternative Dosing Regimens of Subcutaneous Azacitidine for Myelodysplastic Syndromes Completed
  23. Phase 2 Azacitidine and Etanercept in Treating Patients With Myelodysplastic Syndromes Completed
  24. Phase 2 Azacitidine and Entinostat in Treating Patients With Advanced Breast Cancer Completed
  25. Phase 2 Azacitidine and Lenalidomide for Acute Myeloid Leukemia Completed
  26. Phase 2 Azacitidine and Entinostat in Treating Patients With Metastatic Colorectal Cancer Completed
  27. Phase 2 Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy Completed
  28. Phase 2 Phase 1b/2 Safety and Efficacy of APR-246 w/Azacitidine for tx of TP53 Mutant Myeloid Neoplasms Completed
  29. Phase 2 Aza-SAHA-GBM With AutoSCT for Refractory Lymphoma Completed
  30. Phase 2 Azacitidine After Allo Blood And Marrow Transplantation (BMT) for Chronic Myelogenous Leukemia (CML) Completed
  31. Phase 2 Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating Agents Completed
  32. Phase 2 A Study Being Conducted at Multiple Locations to Compare Safety and Efficacy of Three Different Regimens; (1) High-Dose Lenalidomide; (2) Lenalidomide + Azacitidine; or (3) Azacitidine in Subjects ≥ 65 Years With Newly-Diagnosed Acute Myeloid Leukemia Completed
  33. Phase 2 Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia Completed
  34. Phase 2 Geriatric Assessment & Genetic Profiling to Personalize Therapy in Older Adults With Acute Myeloid Leukemia Completed
  35. Phase 2 Azacitidine and CAPOX in Metastatic Colorectal Cancer Completed
  36. Phase 2 Phase I/II Trial of R-CHOP + Azacytidine in Diffuse Large B Cell Lymphoma Completed
  37. Phase 2 Phase I/II Study of 5-Azacytidine With Ara-C in Patients With Relapsed/Refractory Acute Myelogenous Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS) Completed
  38. Phase 2 Sorafenib and 5-Azacitidine in Acute Leukemia + Myelodysplastic Syndrome Completed
  39. Phase 2 Azacitidine, Venetoclax, and Trametinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndrome Completed
  40. Phase 2 Glutaminase Inhibitor CB-839 and Azacitidine in Treating Patients With Advanced Myelodysplastic Syndrome Completed
  41. Phase 2 A Study of Pevonedistat and Venetoclax Combined With Azacitidine to Treat Acute Myeloid Leukemia (AML) in Adults Unable to Receive Intensive Chemotherapy Completed
  42. Phase 2 Lenalidomide and Azacitidine in Treating Patients With Advanced Myelodysplastic Syndromes Completed
  43. Phase 2 Busulfan, Fludarabine Phosphate, and Anti-Thymocyte Globulin Followed By Donor Stem Cell Transplant and Azacitidine in Treating Patients With High-Risk Myelodysplastic Syndrome and Older Patients With Acute Myeloid Leukemia Completed
  44. Phase 2 Donor Lymphocyte Infusion With Azacitidine to Prevent Hematologic Malignancy Relapse After Stem Cell Transplantation Completed

Showing the 50 most-cited and recently-updated of 82 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
AZA or LDAC combined with venetoclax likely improves mortality, remission, and quality of life
Oncology Meta-analysis
AZA or LDAC combined with venetoclax likely improves mortality, remission, and quality of life New combinations show promise for older adults with myeloid leukemia
This systematic review and meta-analysis of 47 studies evaluated treatments for older adults with AML ineligible for conventional chemothera…
Combining specific drugs like venetoclax with other treatments may improve survival and quality of life for older adults with acute myeloid …
Jun 12, 2026
Case report and review: azacytidine plus ruxolitinib in connective tissue disease with CMML and myelofibrosis
Allergy & Immunology Sys. Review
Case report and review: azacytidine plus ruxolitinib in connective tissue disease with CMML and myelofibrosis A single patient found relief from blood cancer with a new drug combo
This case report and literature review describes a 52-year-old male with connective tissue disease, chronic myelomonocytic leukemia, and mye…
One man with blood cancer saw his fever and anemia improve after two years on a specific drug mix, offering a glimpse into potential new tre…
Frontiers May 14, 2026
Case report and literature review on concurrent t-AML and LNTB treated with chemoimmunotherapy
Oncology Sys. Review
Case report and literature review on concurrent t-AML and LNTB treated with chemoimmunotherapy Two Rare Diseases Strike After Lung Cancer Treatment
This case report and literature review describes the management of a 54-year-old male with locally advanced lung squamous cell carcinoma, th…
A lung cancer patient treated with chemo and immunotherapy developed two rare diseases simultaneously: a new blood cancer and tuberculosis.
Frontiers Apr 16, 2026
Case report of ATRA and VAH regimen in refractory EVI1-positive AML
Oncology Sys. Review
Case report of ATRA and VAH regimen in refractory EVI1-positive AML New hope for tough AML cases
This case report with a brief literature review describes a 42-year-old patient with refractory EVI1-positive AML.
A patient with high-risk AML and the tough EVI1 marker achieved complete remission after failing standard treatments, offering new hope for …
Frontiers Apr 16, 2026
Meta-analysis finds HMA plus venetoclax yields 66% CR/CRi rate in younger, fit AML patients
Oncology Meta-analysis
Meta-analysis finds HMA plus venetoclax yields 66% CR/CRi rate in younger, fit AML patients Analysis finds promising results for leukemia treatment in younger patients
A meta-analysis of 429 younger, fit AML patients found HMA plus venetoclax yielded a 66% CR/CRi rate and 75% 1-year OS.
Younger acute myeloid leukemia patients treated with a specific drug combination achieved 75% one-year survival and 66% complete remission r…
Apr 5, 2026
Case report: Low-intensity chemotherapy achieves remission in PMF transformed to MPAL
Oncology Cohort
Case report: Low-intensity chemotherapy achieves remission in PMF transformed to MPAL Single case report describes treatment for rare leukemia transformation in elderly patient
A case report describes a 75-year-old female with JAK2V617F-positive primary myelofibrosis that transformed to mixed phenotype acute leukemi…
A 75-year-old woman with rare leukemia transformation from blood disorder achieved remission after two cycles of low-intensity chemotherapy.
Frontiers Apr 3, 2026