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Azacitidine Clinical Trials

77 actively recruiting trials across 26 locations

No change since June 2026

Also known as: 5 AZC, 5-AC, 5-AZC, 5-Azacitidine, 5-Azacytidine +22 more

Pipeline

Early 1: 1Phase 1: 21Phase 2: 31Phase 3: 5Phase 4: 1Phase 1/2: 12Phase 2/3: 2

Top Sponsors

M.D. Anderson Cancer Center8
The First Affiliated Hospital of Soochow University5
Institute of Hematology & Blood Diseases Hospital, China5
National Cancer Institute (NCI)4
OHSU Knight Cancer Institute3

Indications

Cancer77
Acute Myeloid Leukemia38
Myelodysplastic Syndrome9
Chronic Myelomonocytic Leukemia7
Refractory Acute Myeloid Leukemia5

Other25 trials

A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.

Phase 1

A Platform Trial Evaluating New Drugs or Combination in R/R Peripheral T-cell Lymphomas

Phase 1/2

A Study of Selinexor in Combination With Azacitidine and Venetoclax (SAV Regimen) in Treatment Naïve Participants With Acute Myeloid Leukemia

Phase 2

A Trial to Assess Cobicistat Boosted Venetoclax in Combination With Azacitidine in Adult Patients With Newly Diagnosed AML

Phase 2

AZA + Venetoclax as Maintenance Therapy in Younger Adults With AML in First Remission

Phase 2/3

An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.

Phase 3

Chidamide in Combination With Azacitidine, Liposomal Mitoxantrone, and Prednisone (CAMP Regimen) for the Treatment of Previously Untreated Nodal TFH Cell Lymphoma

Phase 2

Chidamide, Venetoclax, and Azacitidine for Newly Diagnosed Acute Myeloid Leukemia

N/A

Clinical Study on Anti-PD-1 Plus Lenalidomide and Azacitidine in Relapsed/Refractory Peripheral T-cell Lymphoma

Phase 2

Comparison of VA and D/IA Induction Regimens in Elderly Fit Acute Myeloid Leukemia Patients

Phase 3

De-Escalation Study Evaluating Venetoclax and Azacitidine Discontinuation in AML Responding Patients

Phase 2

Efficacy of Venetoclax Combined With Intensive Chemotherapy in Different Subgroups of AML

Phase 2

HEC73543 Versus Salvage Chemotherapy in R/R FLT3-ITD AML

Phase 3

IMM01+Azacitidine VS Placebo +Azacitidine in Patients With Newly Diagnosed Chronic Myelomonocytic Leukemia (CMML1-2)

Phase 3

MRD-positive AML Clinical Study

N/A

PD-1 Inhibitor, Azacitidine and Low-dose DLI in AML Relapse After Allo-HSCT

N/A

Randomized Study of ABC-14 Regimen Compared With "3+7" Standard Induction Therapy or AB-14 for ND AML

Phase 2

Single Arm Study of Post-transplant Azacitidine and Chidamide for Prevention of Acute Myelogenous Leukemia Relapse

Phase 1/2

Study of A Venetoclax-based, Anthracycline-free Regimen in Newly Diagnosed CBFβ::MYH11(+) AML

Phase 2

Study of Lisaftoclax (APG-2575) Single Agent and Combination With Therapy in Patients Relapsed/Refractory AML

Phase 1/2

Study of Venetoclax and Azacitidine in Advanced BCR-ABL Negative Myeloproliferative Neoplasms

Phase 2

Study to Assess Adverse Events and Change in Disease Activity of Oral Venetoclax in Combination With Subcutaneous (SC) or Intravenous (IV) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Standard Induction Therapy in India

Phase 4

The Efficacy and Safety of Third-generation TKIs Combined With Azacitidine and Bcl-2 Inhibitor in Patients With CML-MBP

Venetoclax + Azacytidine for Newly Diagnosed ETP-like ALL and T-ALL With Myeloid Mutations

Phase 2

Venetoclax and Azacitidine Combined With Chidamide (VAC) for the Treatment of Newly Diagnosed Acute Monocytic Leukemia

Phase 2

Birmingham, Alabama6 trials

A Safety Study of SEA-CD70 in Patients With Myeloid Malignancies

University of Alabama at Birmingham

Phase 1

Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)

University of Alabama at Birmingham Cancer Center

Phase 2

MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)

University of Alabama at Birmingham Cancer Center

Phase 2

Study of SLS009 (Formerly GFH009) a Potent Highly Selective CDK9 Inhibitor in Patients With Hematologic Malignancies and High-Risk Newly Diagnosed AML

O'Neal Comprehensive Cancer Center, University of Alabama

Phase 1/2

Testing the Effects of Novel Therapeutics for Newly Diagnosed, Untreated Patients With High-Risk Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)

University of Alabama at Birmingham Cancer Center

Phase 2

Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial)

University of Alabama at Birmingham Cancer Center

Phase 2

Houston, Texas5 trials

A Phase 1B/2A Trial of NADUNOLIMAB in Combination With Azacitidine (With/Without Venetoclax) in Patients With Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)

MD Anderson Cancer Center

Phase 1/2

A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

The University of Texas MD Anderson Cancer Center

Phase 1

Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm

M D Anderson Cancer Center

Phase 1/2

Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation

M D Anderson Cancer Center

Phase 2

Venetoclax, Cladribine, Low Dose Cytarabine, and Azacitidine in Treating Patients With Previously Untreated Acute Myeloid Leukemia

M D Anderson Cancer Center

Phase 2

Duarte, California4 trials

A Study of BGB-11417 in Participants With Myeloid Malignancies

City of Hope National Medical Center

Phase 1/2

A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

City of Hope National Medical Center

Phase 1

SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

City of Hope

Phase 1

Tagraxofusp and Azacitidine for Maintenance Treatment in Patients With CD123 Positive AML and MDS Following Donor Hematopoietic Cell Transplant

City of Hope Medical Center

Phase 1

Phoenix, Arizona3 trials

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Phoenix Children's Hospital

Phase 1/2

A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML

Mayo Clinic - Phoenix

Phase 1

Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)

Phoenix Children's Hospital

Phase 3

Los Angeles, California3 trials

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

Keck School of Medicine of USC

Phase 2/3

A Study of Tagraxofusp in Combination With Venetoclax and Azacitidine in Adults With Untreated CD123+ Acute Myeloid Leukemia Who Cannot Undergo Intensive Chemotherapy

University of California, Los Angeles

Phase 2

Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies

Children's Hospital Los Angeles

Phase 1

Aurora, Colorado3 trials

Cladribine Venetoclax in Monocytic AML

Universtiy of Colorado Hospital

Phase 2

IDH Targeted/Non- Targeted vs Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)

University of Colorado

Phase 2

Mitoxantrone for Venetoclax Resistant Acute Myeloid Leukemia

University of Colorado Hospital

Phase 1

Miami, Florida3 trials

Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy

University of Miami

Phase 2

Tagraxofusp and Azacitidine With Venetoclax in Newly Diagnosed Secondary AML After Hypomethylating Agents

University of Miami

Phase 2

Venetoclax in Combination With Cladribine and Cytarabine Alternating With Azacitidine Plus Venetoclax for the Treatment of Newly Diagnosed Monocytic AML and Active Signaling Mutated AML

University of Miami Miller School of Medicine-Sylvester Cancer Center

Phase 2

New Haven, Connecticut2 trials

ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies

Yale University, Yale Cancer Center

Phase 1

Study of BMS-986497 (ORM-6151) as a Monotherapy, in Double and Triple Combination With Azacitidine and Venetoclax in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Yale-New Haven Hospital

Phase 1

Chicago, Illinois2 trials

BRE-04: Window of Opportunity Trial of Preoperative Low Dose Azacitidine in High-Risk Early Stage Breast Cancer

University of Illinois Cancer Center

Phase 2

Selumetinib and Azacitidine in High Risk Chronic Blood Cancers

The University of Chicago

Phase 1

Baltimore, Maryland2 trials

Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

Johns Hopkins University/Sidney Kimmel Cancer Center

Phase 2

Momelotinib in Combination With Hypomethylating Agent for Chronic Phase Myelodysplastic Syndromes/Myeloproliferative Overlap Neoplasms and Chronic Neutrophilic Leukemia

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Early 1

New York, New York2 trials

Imetelstat Combinations in Relapsed AML

Icahn School of Medicine at Mount Sinai

Phase 1

Pacritinib in CMML

The Mount Sinai Hospital

Phase 1/2

Columbus, Ohio2 trials

Axatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia

Ohio State University Comprehensive Cancer Center

Phase 1/2

Ruxolitinib in Combination With Venetoclax With and Without Azacitidine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Ohio State University Comprehensive Cancer Center

Phase 1

Portland, Oregon2 trials

Iadademstat in Combination With Azacitidine and Venetoclax in Treating Newly Diagnosed Acute Myeloid Leukemia

OHSU Knight Cancer Institute

Phase 1

Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation

OHSU Knight Cancer Institute

Phase 1

Clovis, California1 trial

Azacitidine or Decitabine With Venetoclax for Acute Myeloid Leukemia With Prior Hypomethylating Agent Failure

UCSF-Fresno

Phase 2

Sacramento, California1 trial

Olutasidenib With Azacitidine Followed by Olutasidenib Maintenance for the Treatment of IDH1-mutated Acute Myeloid Leukemia in Patients With Prior Treatment With Venetoclax Plus a Hypomethylating Agent

University of California Davis Comprehensive Cancer Center

Phase 2

San Diego, California1 trial

Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia

Rady Children's Hospital

Phase 1

San Francisco, California1 trial

Venetoclax Basket Trial for High Risk Hematologic Malignancies

University of California San Francisco-Benioff Children's Hospital

Phase 1

Denver, Colorado1 trial

APVO436 Phase 1b/2 Study in Patients With Newly Diagnosed AML

Colorado Blood Cancer Institute

Phase 1

Tampa, Florida1 trial

Discontinuation of Hypomethylating Agent and Venetoclax in Patients With AML MRD

Moffitt Cancer Center

Phase 2

Atlanta, Georgia1 trial

Study to Evaluate CCS1477 (Inobrodib) in Haematological Malignancies

Emory Winship Cancer Institute

Phase 1/2

Iowa City, Iowa1 trial

High-dose Ascorbate (HDA) in Combination With Standard of Care Azacitidine and Venetoclax in Acute Myeloid Leukemia (AML)

University of Iowa Health Care

Phase 1

Boston, Massachusetts1 trial

Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies

Dana-Farber Cancer Institute

Phase 1/2

Detroit, Michigan1 trial

Azacitidine and Chimerism in MDS or AML Patients After Allogeneic Stem Cell Transplant

Henry ford hospital

Phase 2

Basking Ridge, New Jersey1 trial

A Study of the Combination of Ivosidenib, Azacitidine, and Venetoclax Followed by Ivosidenib Alone in People With Acute Myeloid Leukemia

Memoral Sloan Kettering at Basking Ridge (Limited Protocol Activities)

Phase 2

Cleveland, Ohio1 trial

A Phase Ib Trial of Azacitidine, Venetoclax and Allogeneic NK Cells for Acute Myeloid Leukemia (ADVENT-AML)

Case Western Reserve University

Phase 1

Seattle, Washington1 trial

Pacritinib With Standard of Care Azacitidine or Decitabine as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant for Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms

Fred Hutch/University of Washington Cancer Consortium

Phase 2

Trial data from ClinicalTrials.gov. Trial status and eligibility can change — verify directly with the study contact or on ClinicalTrials.gov.

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