Mode
Text Size
Log in / Sign up

Acute Myeloid Leukemia

Part of Leukemia

29 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Acute Myeloid Leukemia

244 trials tracked for Acute Myeloid Leukemia: 29 in phase 3 or 4 and 19 with published results. The most-cited published study has 358 citations.

244Trials tracked
29Phase 3 & 4
0Recruiting
19With published results
Phase distribution
Phase 4 1 Phase 3 28 Phase 2 160 Phase 1 33 Other / NA 22
  1. Phase 3 Quizartinib With Standard of Care Chemotherapy and as Continuation Therapy in Patients With Newly Diagnosed FLT3-ITD (+) Acute Myeloid Leukemia (AML) Completed · 358 cited
  2. Phase 3 A Trial of the FMS-like Tyrosine Kinase 3 (FLT3) Inhibitor Gilteritinib Administered as Maintenance Therapy Following Allogeneic Transplant for Patients With FLT3/Internal Tandem Duplication (ITD) Acute Myeloid Leukemia (AML) Completed · 182 cited
  3. Phase 3 Study of Vosaroxin or Placebo in Combination With Cytarabine in Patients With First Relapsed or Refractory AML Completed · 157 cited
  4. 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
  5. Phase 3 Clofarabine Plus Cytarabine Versus Conventional Induction Therapy And A Study Of NK Cell Transplantation In Newly Diagnosed Acute Myeloid Leukemia Completed · 113 cited
  6. Phase 3 Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant Completed · 111 cited
Show 44 more trials
  1. Phase 3 Caspofungin Versus Fluconazole in Preventing Invasive Fungal Infections (IFI) in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia Completed · 108 cited
  2. Phase 3 Bortezomib and Sorafenib Tosylate in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia Completed · 101 cited
  3. Phase 3 Study of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML Completed · 82 cited
  4. Phase 3 Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission Completed · 63 cited
  5. 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
  6. Phase 3 Volasertib in Combination With Low-dose Cytarabine in Patients Aged 65 Years and Above With Previously Untreated Acute Myeloid Leukaemia, Who Are Ineligible for Intensive Remission Induction Therapy (POLO-AML-2) Completed · 39 cited
  7. Phase 3 Cytarabine and Daunorubicin Hydrochloride or Idarubicin and Cytarabine With or Without Vorinostat in Treating Younger Patients With Previously Untreated Acute Myeloid Leukemia Completed · 32 cited
  8. Phase 3 SGI-110 in Adults With Untreated Acute Myeloid Leukemia (AML), Not Considered Candidates for Intensive Remission Induction Completed · 25 cited
  9. Phase 3 An Efficacy and Safety Study of Decitabine (DACOGEN) Plus Talacotuzumab (JNJ-56022473; Anti CD123) Versus Decitabine (DACOGEN) Alone in Participants With Acute Myeloid Leukemia (AML) Ineligible for Intensive Chemotherapy Completed · 21 cited
  10. Phase 3 Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Combination With Standard Chemotherapy During Induction and Consolidation Followed by 12 Months of Maintenance Monotherapy in Patients With Newly-diagnosed FMS-like Tyrosine 3 (FLT3) Kinase Receptor-mutated Acute Myeloid Leukemia. Completed · 18 cited
  11. Phase 3 Tac, Mini-MTX, MMF Versus Tac, MTX for GVHD Prevention Completed · 10 cited
  12. Phase 3 Phase 3 Randomized, Open-Label Study of Guadecitabine vs Treatment Choice in Previously Treated Acute Myeloid Leukemia Completed · 9 cited
  13. Phase 3 A Study of Oral Sapacitabine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia Completed · 8 cited
  14. Phase 4 Study Assessing Deep Molecular Response in Adult Patients With CML in Chronic Phase Treated With Nilotinib Firstline. Completed
  15. Phase 3 IV Busulfan With Allo-BMT: Study for Patients With Acute Myelogenous Leukemia and Myelodysplastic Syndrome Completed
  16. 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
  17. 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
  18. Phase 3 Safety and Efficacy of Nilotinib vs. Imatinib in the Treatment of Newly Diagnosed Chinese Ph+ CML-CP Patients Completed
  19. Phase 3 Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML) Completed
  20. Phase 3 Compare Bosutinib To Imatinib In Subjects With Newly Diagnosed Chronic Phase Philadelphia Chromosome Positive CML Completed
  21. Phase 3 Trial of Decitabine in Patients With Acute Myeloid Leukemia Completed
  22. Phase 3 Safety and Efficacy of Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia Patients Completed
  23. Phase 3 Study to Investigate the Efficacy, Safety and Tolerability of AZD1152 Alone and in Combination With Low Dose Cytosine Arabinoside (LDAC)in Acute Myeloid Leukaemia (AML) Patients Completed
  24. Phase 2 T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts Completed
  25. Phase 2 Decitabine and Talazoparib in Untreated AML and R/R AML Completed
  26. Phase 2 Salsalate, Venetoclax, and Decitabine or Azacitidine for the Treatment of Acute Myeloid Leukemia or Advanced Myelodysplasia/Myeloproliferative Disease Completed
  27. Phase 2 Standard Idarubicin and Cytarabine for the Treatment of Acute Myeloid Leukemia (AML) Completed
  28. Phase 2 Safety and Efficacy Study of Pracinostat With Azacitadine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) Completed
  29. Phase 2 Dasatinib (BMS-354835) Versus Imatinib Mesylate in Subjects With Chronic Myeloid Leukemia Completed
  30. Phase 2 Azacitidine and Lenalidomide for Relapsed and Refractory Patients With Acute Myeloid Leukemia Completed
  31. Phase 2 Off-the-shelf NK Cells + SCT for Myeloid Malignancies Completed
  32. Phase 2 Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation Completed
  33. Phase 2 Study of Crenolanib Combined With Chemotherapy in FLT3-mutated Acute Myeloid Leukemia Patients Completed
  34. Phase 2 2018-0674 - IL-7 for T-Cell Recovery Post Haplo and CB Transplant - Phase I/II Completed
  35. Phase 2 Multi-Ctr PII Cmb.Modality Tx Ruxolitinib, Decitabine, and DLI for Post HSCT in AML/MDS Completed
  36. Phase 2 Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies Completed
  37. Phase 2 Standard of Care +/- Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients With FLT3-ITD Mutated AML Completed
  38. Phase 2 Trial of PXD101 (Belinostat) in Combination With Idarubicin to Treat AML Not Suitable for Standard Intensive Therapy Completed
  39. Phase 2 Clofarabine and Low-Dose Total-Body Irradiation in Treating Patients With Acute Myeloid Leukemia Undergoing Donor Peripheral Blood Stem Cell Transplant Completed
  40. 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
  41. Phase 2 Study of Voreloxin (Vosaroxin) in Older Patients With Untreated Acute Myeloid Leukemia Completed
  42. Phase 2 High-dose Cytarabine/Mitoxantrone Followed by Autotransplantation for t-MDS/t-AML Completed
  43. Phase 2 A Phase 2 Study of ABT-199 in Subjects With Acute Myelogenous Leukemia (AML) Completed
  44. Phase 2 Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases Completed

Showing the 50 most-cited and recently-updated of 244 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.

Acute Myeloid Leukemia: current evidence & treatments

For older adults with newly diagnosed acute myeloid leukemia (AML) ineligible for conventional chemotherapy, combining azacitidine (AZA) or low-dose cytarabine (LDAC) with venetoclax likely reduces mortality and improves remission and quality of life 1. In younger, fit patients (median age 54 years), a meta-analysis of 429 patients treated with hypomethylating agents (HMA) plus venetoclax reported a pooled complete remission (CR/CRi) rate of 66% (95% CI, 48%-85%) and a 1-year overall survival of 75% 25. For relapsed/refractory AML, a meta-analysis of 2289 patients receiving HMA-venetoclax found an overall response rate of 50% (95% CI, 48%-53%) and a complete remission rate of 26% 5.

In newly diagnosed AML, a 7-day venetoclax induction combined with intensive chemotherapy achieved a composite complete remission rate of 90.3% and an MRD negativity rate of 92.2%, with estimated 24-month overall survival of 72.9% 2. For AML with myelodysplasia-related changes (AML-MR), CPX-351 improved overall survival compared to standard 7+3 therapy (median OS 9.7 vs 6.8 months, P = .037) 11, and a retrospective study reported a 61% overall response rate with CPX-351 as first-line therapy 13. For FLT3-ITD-positive AML, quizartinib has been approved in combination with standard chemotherapy 19, and a network meta-analysis showed that gilteritinib and midostaurin improved complete remission rates, while gilteritinib and quizartinib prolonged overall survival 8.

Novel strategies in allogeneic hematopoietic cell transplantation aim to separate graft-versus-host disease from graft-versus-leukemia effects 3. Antibody-based therapies, including unconjugated monoclonal antibodies, antibody-drug conjugates, and bispecific T cell engagers, offer potential efficacy for relapsed/refractory AML 4. Donor-derived anti-CD33 CAR T-cell therapy showed a 20% overall response rate in 15 adults with relapsed AML/MDS after transplant 15. Additionally, genetic studies have identified new risk loci for AML, including a pan-AML risk locus at 2p23.3 16.

Key drugs in the evidence

venetoclax 6 azacitidine 4 cytarabine 4 quizartinib 3 imatinib 3 dasatinib 2 asciminib 2 CPX-351 2 decitabine 2 gemtuzumab ozogamicin 2

Latest findings

AI-generated synthesis of 12 cited studies, updated Jun 12, 2026. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.

Research across Leukemia

Related studies from across the Leukemia family.

Rate of drug concentration rise alters hemodynamic responses and mortality risk in coronary heart disease patients
Drug Pipeline Meta-analysis
Rate of drug concentration rise alters hemodynamic responses and mortality risk in coronary heart disease patients New analysis shows how fast drug levels change affects patient safety outcomes
This narrative review and theoretical framework synthesizes evidence on rate-dependent pharmacology. It highlights divergent hemodynamic res…
This review explains that how quickly medicine levels rise in the body matters for safety. Fast changes can cause more harm than slow ones, …
Fertility preservation in female leukemia patients carries risks of leukemic cell contamination and disease reintroduction
Diabetes & Endocrinology Sys. Review
Fertility preservation in female leukemia patients carries risks of leukemic cell contamination and disease reintroduction Fertility preservation options carry risks for female leukemia patients
This review examines fertility preservation strategies for female patients with leukemia. The authors note that oocyte or embryo cryopreserv…
Fertility preservation strategies for female leukemia patients face safety concerns like disease reintroduction and contamination risks that…
Xatmep receives FDA approval for pediatric acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis
Hematology FDA Approval
Xatmep receives FDA approval for pediatric acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis FDA approves Xatmep for pediatric leukemia and arthritis treatment options
The FDA has approved Xatmep for pediatric patients with acute lymphoblastic leukemia or active polyarticular juvenile idiopathic arthritis. …
The FDA has approved Xatmep to treat specific forms of childhood leukemia and arthritis, offering new options for young patients.
Reduced-intensity ATRA plus arsenic with GO or low-dose anthracycline improves event-free survival in high-risk APL
Hematology Meta-analysis
Reduced-intensity ATRA plus arsenic with GO or low-dose anthracycline improves event-free survival in high-risk APL New analysis shows reduced-intensity treatment works well for high-risk leukemia patients compared to standard intensive chemotherapy
This systematic review and meta-analysis of 1,323 patients with high-risk acute promyelocytic leukemia found that reduced-intensity inductio…
A large review of over 1,300 patients found that a gentler treatment plan worked just as well as harsher chemotherapy. This approach may hel…
Narrative review suggests idiotype-directed CAAR-T therapy remains hypothetical for B-cell malignancies
Hematology Sys. Review
Narrative review suggests idiotype-directed CAAR-T therapy remains hypothetical for B-cell malignancies Idiotype-directed CAAR-T therapy remains a hypothesis for B-cell malignancies
This narrative review discusses idiotype-directed CAAR-T therapy for B-cell malignancies including chronic lymphocytic leukemia and indolent…
A narrative review suggests idiotype-directed CAAR-T therapy is a theoretical option for B-cell cancers, not a current treatment.
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…
Case report and review of TTMV::RARA-positive APL with spinal cord compression in a child
Orthopedics & Sports Medicine Meta-analysis
Case report and review of TTMV::RARA-positive APL with spinal cord compression in a child Doctors treated a boy with rare leukemia and spinal pressure using special drugs
This is a case report with a systematic review of 11 published cases of TTMV::RARA-positive acute promyelocytic leukemia (APL). The authors …
A nine-year-old boy had rare leukemia and spinal cord pressure. Doctors used specific drugs that helped clear the disease and fix his spine.
Systematic review and meta-analysis finds high prevalence of ocular manifestations in leukemia patients
Hematology Meta-analysis
Systematic review and meta-analysis finds high prevalence of ocular manifestations in leukemia patients Leukemia patients face eye risks that doctors must spot early now
This systematic review and meta-analysis of 1016 patients with leukemia found a 42% prevalence of overall ocular manifestations.
New data shows almost half of leukemia patients have eye issues, so regular eye checks are essential for better care
Meta-analysis finds fluoroquinolone prophylaxis reduces febrile neutropenia in pediatric ALL
Oncology Meta-analysis
Meta-analysis finds fluoroquinolone prophylaxis reduces febrile neutropenia in pediatric ALL Fluoroquinolone antibiotics lower febrile neutropenia risk in children with acute lymphoblastic leukemia during treatment start
This systematic review and meta-analysis of 991 children with acute lymphoblastic leukemia found that fluoroquinolone prophylaxis reduced fe…
A review of children with acute lymphoblastic leukemia shows fluoroquinolone antibiotics reduce febrile neutropenia risk. This finding sugge…
Lyophilized pegaspargase shows similar activity to liquid form in pediatric ALL
Oncology RCT
Lyophilized pegaspargase shows similar activity to liquid form in pediatric ALL Lyophilized pegaspargase shows similar activity and safety to liquid form for pediatric leukemia patients
This phase 2 study in 89 pediatric ALL patients in Russia found that lyophilized pegaspargase had similar maximum plasma asparaginase activi…
A new form of pegaspargase works just like the liquid version for treating acute lymphoblastic leukemia in children in Russia.
Meta-analysis links maternal infection in pregnancy to childhood ALL risk
Hematology Meta-analysis
Meta-analysis links maternal infection in pregnancy to childhood ALL risk Maternal Infections During Pregnancy Linked to Higher Risk of Childhood Leukemia
This systematic review and meta-analysis of 26 studies found a modest association between maternal infection during pregnancy and childhood …
A large review of 26 studies found that maternal infections during pregnancy are associated with an increased risk of acute lymphoblastic le…
Meta-analysis links perinatal factors to childhood acute leukemia risk
Hematology Meta-analysis
Meta-analysis links perinatal factors to childhood acute leukemia risk Perinatal factors linked to higher leukemia risk in children
This systematic review and meta-analysis of 85 studies examined perinatal factors and childhood acute leukemia risk.
A large analysis of 85 studies finds links between birth weight, maternal history, and cesarean sections with childhood leukemia risk.

Questions about Acute Myeloid Leukemia

Can ponatinib help if my chronic myeloid leukemia is resistant to other drugs?

Yes, ponatinib can help if your CML is resistant to other drugs, especially if you have the T315I mutation, but it has serious side effects like heart problems.

Full answer →
Does taking imatinib lower inflammation markers like TNF-alpha in chronic myeloid leukemia?

Yes, imatinib significantly lowers TNF-alpha levels in CML patients, and persistent high levels are linked to poor treatment response.

Full answer →
Is there a new drug approved for adults with newly diagnosed chronic myeloid leukemia?

Yes, asciminib (Scemblix) was approved in 2024 for newly diagnosed Ph+ CML-CP, and nilotinib (Danziten) was also approved for this indication.

Full answer →
Do specific gene changes affect how much imatinib stays in my blood?

Yes, specific gene changes in ABCB1 and ABCG2 can affect how much imatinib stays in your blood, leading to higher drug levels.

Full answer →
Does a case report show myeloid sarcoma relapse appearing in the breast for Acute Myeloid Leukemia?

Yes, a case report describes a 68-year-old woman with myeloid sarcoma relapse in the breast 6 years after initial nasal cavity presentation.

Full answer →
How common are gram-negative bloodstream infections in children with Acute Myeloid Leukemia?

Gram-negative bloodstream infections occur in about 27% of children with AML during induction chemotherapy, with high rates of multidrug resistance and significant mortality.

Full answer →
Are there new genetic risks identified for Acute Myeloid Leukemia in specific gene locations?

Yes, a large meta-analysis identified new genetic risk loci for AML at 2p23.3, 1q23.3, 2q33.3, and 2p21, and confirmed known mutations in FLT3, NPM1, TP53, and other genes.

Full answer →
What is the overall response rate for CPX-351 in therapy-related Acute Myeloid Leukemia?

The overall response rate for CPX-351 in therapy-related AML is approximately 47-61%, with complete response rates around 47-58%.

Full answer →
See all 10 questions about Acute Myeloid Leukemia →

All Acute Myeloid Leukemia Articles