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AML (Acute Myeloid Leukemia) Clinical Trials | TrialFinder
HomeResourcesAML (Acute Myeloid Leukemia) Clinical Trials: How Genetic Mutations Shape Your Options

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Published · September 27, 2026

AML (Acute Myeloid Leukemia) Clinical Trials: How Genetic Mutations Shape Your Options

Leukemia is not one disease. The four main types — AML (acute myeloid leukemia), ALL (acute lymphoblastic leukemia), CML (chronic myeloid leukemia), and CLL (chronic lymphocytic leukemia) — are different diseases. They have different treatments and different trial lists. This page is about AML, the most common acute leukemia in adults.

A trial whose title says "leukemia" is usually written for only one of these. ALL, CML, and CLL studies are not interchangeable with AML studies. A report that only says "leukemia" is not enough to judge eligibility.

AML starts in the bone marrow, when myeloid cells grow out of control and crowd out healthy blood cells. Two people with AML can have different gene changes, a different disease setting (newly diagnosed versus relapsed or refractory), and a different fitness for intensive treatment. Those details usually decide which treatments are standard and which clinical trials may fit.

Genetic testing looks for changes such as FLT3 (including ITD and TKD), IDH1, IDH2, and NPM1. Some of these have approved targeted drugs in defined settings — FLT3 inhibitors such as midostaurin and gilteritinib, and IDH inhibitors such as ivosidenib (IDH1) and enasidenib (IDH2). Testing at diagnosis and again at relapse decides which targeted trials may fit. Ask for the mutation results by name.

A clinical trial is one option to raise with the treating hematologist. It is not automatically better than approved treatment. The useful question is whether a given study matches this exact diagnosis, mutation profile, disease setting, and whether intensive chemotherapy or transplant is on the table.

Newly diagnosed and relapsed/refractory AML are not interchangeable

First-line trials enroll people who have not yet been treated for AML, or who are starting initial therapy. Trials for relapse after treatment, and trials for disease that stopped responding (refractory), enroll different populations. The protocols are separate. A listing written for newly diagnosed AML is not the same as one for relapsed or refractory disease.

Cytogenetics and mutation results can also change between diagnosis and relapse. A mutation that was absent at diagnosis can appear later, and the reverse can happen after targeted therapy.

The plain-language clinical trial glossary can help with terms such as eligibility, phase, and standard of care.

Biomarkers that actually separate one trial from another

FLT3. FLT3 changes are common in AML. Two kinds appear on reports: ITD (internal tandem duplication) and TKD (tyrosine kinase domain). They are not interchangeable. Trials of FLT3 inhibitors, including drugs such as midostaurin and gilteritinib, usually name FLT3 and often name the exact kind.

IDH1 and IDH2. These are different genes. Ivosidenib is an IDH1 inhibitor. Enasidenib is an IDH2 inhibitor. A trial written for IDH1 is not an IDH2 trial. Ask which one, if either, is on the report.

NPM1. NPM1 mutations are also common in AML. Some targeted and combination trials require an NPM1 change, or pair it with FLT3 status. An NPM1 result by itself does not say which studies fit.

Genetic testing at diagnosis and at relapse is what decides which targeted trials belong on the shortlist. Ask the hematologist for the mutation results by name — FLT3 (ITD or TKD), IDH1, IDH2, NPM1 — not only whether "a mutation was found."

Some of these changes have approved targeted treatments in defined settings. Those approvals depend on the exact variant, whether disease is newly diagnosed or relapsed/refractory, prior treatment, the test that was used, and the country where care is given.

Fit and unfit AML are not the same trial lists

Many AML trials split by whether a person is considered eligible for intensive chemotherapy and stem cell transplant, or not. "Fit" and "unfit" (sometimes "older" or "ineligible for intensive therapy") are protocol terms. They change which studies apply.

Transplant-eligible, intensive-therapy trials often enroll younger or fitter adults and may include induction chemotherapy, consolidation, and allogeneic transplant pathways.

Lower-intensity trials enroll people for whom intensive chemotherapy or transplant is not the plan — because of age, other illness, or the hematologist's assessment. The drugs, visit burden, and eligibility cutoffs are different.

A report that only says AML does not say which of these lists to use. Ask whether intensive chemotherapy and transplant are being considered.

Recruiting AML trials

Questions to ask a hematologist

A short list is usually enough:

  • What is the exact diagnosis, including cytogenetics?
  • Were FLT3 (ITD and TKD), IDH1, IDH2, and NPM1 tested, and what were the results by name?
  • Is this newly diagnosed AML, or relapsed or refractory disease?
  • Is intensive chemotherapy and stem cell transplant being considered, or is the plan lower-intensity treatment?
  • For this profile, what is approved or considered standard where I live?
  • Are there recruiting trials that match this exact profile, and how do they differ from standard care?

Want to prepare for the appointment? The guide How to Talk to Your Doctor About Clinical Trials explains how to raise the topic and what to bring with you.

Sources

  • NCI: Leukemia
  • NCI PDQ: Adult AML treatment (patient)
  • NCI PDQ: Adult AML treatment (health professional)
  • Recruiting-trial facts on this page come from ClinicalTrials.gov and are shown through TrialFinder listings.

Further reading

How to Talk to Your Doctor About Clinical Trials What questions should you ask your doctor about clinical trials? A step-by-step guide for patients: when to bring it up, what to prepare, and what to do after.

Key Clinical Trial Terms Plain-language explanations of terms patients and families encounter during the clinical trial process.

Start Here: How to Search for Clinical Trials on TrialFinder A step-by-step guide to finding clinical trials on TrialFinder. Learn how to search by condition, use filters, and understand your results.

Before You Join a Clinical Trial: Screening, Informed Consent, and Red Flags What happens between finding a clinical trial and your first visit: screening, the informed consent form, the questions worth asking, common fears answered plainly, and the red flags that should make you walk away.

Clinical Trials in Mental Health: What Patients and Families Should Know Mental health clinical trials can test medications, psychotherapy, TMS devices, digital therapeutics, and psychedelic-assisted treatment. Here is how they differ from cancer trials, and how to start looking.

The Reminder Moderna and Merck's Personalized mRNA Cancer Vaccine Gives Us About Clinical Trials Moderna and Merck's personalized mRNA cancer vaccine just hit its Phase 3 endpoints in melanoma. Here's what it teaches us about the value of joining clinical trials.

Daraxonrasib (RASONQUE) Is Now FDA-Approved for Metastatic Pancreatic Cancer The FDA approved daraxonrasib (RASONQUE) on August 26, 2026 for metastatic pancreatic adenocarcinoma after prior therapy or when multiagent therapy is not an option.

What is a Clinical Trial? (It's More Than You Think) How clinical trials work, why they matter, and what patients should know before considering one.

Not a Perfect Match? Tips for Getting Into a Clinical Trial Anyway A rejection from a clinical trial doesn't have to be the end. Practical tips for patients and caregivers who hit a wall.

How to Talk About Clinical Trials with a Family Member A guide for caregivers on introducing clinical trials to a loved one who is hesitant, tired, or afraid.

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This article was written by a patient advocate based on personal experience, not by a medical professional. It is not medical advice. Consult your doctor before making any medical decisions.

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Trial data from ClinicalTrials.gov. For information only.

This list is limited to recruiting studies whose title or listed conditions name AML or acute myeloid leukemia. A biomarker or setting heading appears only when that term is named in those same fields. A listing that only says leukemia, without naming AML, is not included — those studies are usually written for a different leukemia type.

Intensity

Biomarker

Setting

Loading matching clinical trials

Trials whose title or listed conditions name FLT3

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

    Leukemia, Acute Myeloid (AML)

    ChinaRecruitingPhase 3
    Simple summary·Eligibility check·Doctor guide
    Hanzhou, China

Trials whose title or listed conditions name IDH2

  • Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial)

    Acute Myeloid Leukemia

    2 countriesRecruitingPhase 2
    Simple summary·Eligibility check·Doctor guide
    Tucson, United States · Tucson, United States

Trials whose title or listed conditions name newly diagnosed AML

  • Leukemia Adapted Protocol

    Acute Myeloid Leukaemia

    MalawiRecruiting
    Simple summary·Eligibility check·Doctor guide
    Lilongwe, Malawi

  • Revumenib in Combination With 7+3 + Midostaurin in AML

    Acute Myeloid Leukemia · AML, Adult · AML With Gene Mutations

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    New Haven, United States · Boston, United States

  • Efficacy of Integrated Induction-Consolidation Chemotherapy and Transplantation for Adult Acute Myeloid Leukemia: Multicenter Study

    Acute Myeloid Leukemia

    ChinaRecruitingPhase 2
    Simple summary·Eligibility check·Doctor guide
    Taiyuan, China

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

    AML · Acute Myeloid Leukemia

    ChinaRecruiting
    Simple summary·Eligibility check·Doctor guide
    Tianjin, China

Trials whose title or listed conditions name relapsed or refractory AML

  • Edetate Calcium Disodium or Succimer in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Chemotherapy

    Acute Myeloid Leukemia · Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome · Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Houston, United States

  • Safety and Efficacy of RVU120 Combined With Venetoclax for Treatment of Relapsed/Refractory AML

    Acute Myeloid Leukemia

    4 countriesRecruitingPhase 2
    Simple summary·Eligibility check·Doctor guide
    Grenoble, France · Le Mans, France

  • A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)

    Acute Myeloid Leukemia

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Houston, United States

  • MiRisten for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

    Recurrent Acute Myeloid Leukemia · Refractory Acute Myeloid Leukemia

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Duarte, United States

  • Clinical Trial of CD123-targeted CAR-NK Therapy for Relapse/Refractory AML or BPDCN

    AML (Acute Myeloid Leukemia) · BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm)

    ChinaRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Wuhan, China

  • Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

    Recurrent Childhood Acute Myeloid Leukemia · Recurrent Childhood Acute Lymphoblastic Leukemia · Recurrent Childhood Large Cell Lymphoma

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Miami, United States

  • Assessing PI3K Gamma Inhibition With Azacitidine, Venetoclax and Eganelisib in Patients With Acute Myeloid Leukemia

    Leukemia, Myeloid · Leukemia · Acute Myeloid Leukemia

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Boston, United States · Boston, United States

  • Niclosamide in Pediatric Patients With Relapsed and Refractory AML

    Acute Myeloid Leukemia (AML)

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Palo Alto, United States

  • CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML

    Leukemia, Myeloid, Acute

    United StatesRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Philadelphia, United States

  • Anti-CD33-CLL1 CAR-T Cells (ICG415) for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia

    Acute Myeloid Leukemia (AML)

    ChinaRecruitingPhase 1
    Simple summary·Eligibility check·Doctor guide
    Nanchang, China · Nanchang, China

  • Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    Acute Myeloid Leukemia · Blasts 20 Percent or More of Bone Marrow Nucleated Cells · High Risk Myelodysplastic Syndrome

    United StatesRecruitingPhase 1/2
    Simple summary·Eligibility check·Doctor guide
    Houston, United States

  • Tagraxofusp and Low-Intensity Chemotherapy for CD123-Positive Relapsed or Refractory AML

    Refractory Acute Myeloid Leukemia · Relapsed Acute Myeloid Leukemia

    United StatesRecruitingPhase 1/2
    Simple summary·Eligibility check·Doctor guide
    Palo Alto, United States

  • Safety and Efficacy of GYA01 (CART84) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Acute Lymphoblastic T Leukemia Patients (T-ALL).

    Acute Myeloblastic Leukaemia · Leukemia, T-Cell

    SpainRecruitingPhase 1/2
    Simple summary·Eligibility check·Doctor guide
    Barcelona, Spain · Valencia, Spain

  • Correlation of Venetoclax Plasma Concentrations With Toxicity of Hypometilating Agents and Venetoclax Combination for Acute Myeloid Leukemia Patients in Remission.

    AML (Acute Myelogenous Leukemia

    ItalyRecruiting
    Simple summary·Eligibility check·Doctor guide
    Pavia, Italy

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