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Acute Myeloid Leukemia (AML) Clinical Trials and Treatments

Also called AML, Acute Myelogenous Leukemia, Acute Nonlymphocytic Leukemia, Acute Non-Lymphocytic Leukemia, ANLL

Bone marrow makes blood cells from stem cells that mature step by step. In AML a single early myeloid cell picks up gene changes, stops maturing and copies itself into billions of blasts.

FDA-approved medicines

19

Cytarabine, Daunorubicin, Daurismo, Idamycin PFS, Idhifa and 14 more

Active trials

708

See every trial

Next FDA decision

None set

FDA calendar

Medicines are those whose current FDA label names Acute Myeloid Leukemia, checked October 14, 2026. Corticosteroids and immune globulins count once each. Download the data

View 708 active trialsMatch me to a trial

About Acute Myeloid Leukemia

Bone marrow makes blood cells from stem cells that mature step by step. In AML a single early myeloid cell picks up gene changes, stops maturing and copies itself into billions of blasts. The blasts do no useful work, block the production of normal cells, and spill into the blood. That is why the first signs are anemia, infections and bleeding, and why the disease is called acute: without treatment it progresses over weeks, not years.

Diagnosis starts with a complete blood count and a blood smear and is confirmed with a bone marrow aspiration and biopsy, usually from the back of the hip. A diagnosis of AML generally needs 20% or more blasts in the marrow or blood, or one of a short list of defining gene changes. Every new diagnosis should also get cytogenetics (a look at the chromosomes), FISH and molecular testing, because the results decide treatment and outlook. The changes that matter most today are FLT3 (ITD or TKD), NPM1, IDH1, IDH2, KMT2A rearrangements, TP53, CEBPA and the core binding factor changes t(8;21) and inv(16). The European LeukemiaNet 2022 system sorts people into favorable, intermediate and adverse risk on the basis of these results. Acute promyelocytic leukemia (APL), which carries the PML::RARA fusion from t(15;17), is treated as a separate disease with its own 2 drugs, all-trans retinoic acid (tretinoin) and arsenic trioxide, and is a medical emergency because of bleeding risk.

Treatment has 2 broad paths. People who are fit enough get intensive induction chemotherapy, usually 7 days of cytarabine with 3 days of an anthracycline (daunorubicin or idarubicin), called 7+3, with a targeted drug added when the leukemia carries the matching change: Rydapt (midostaurin) or Vanflyta (quizartinib) for FLT3, Mylotarg (gemtuzumab ozogamicin) for CD33-positive disease. Vyxeos, a liposomal form of the 2 chemotherapy drugs, is used for therapy-related AML and AML with myelodysplasia-related changes. People who are 75 or older or have health problems that rule out intensive treatment are usually given Venclexta (venetoclax) with azacitidine or decitabine, which in the VIALE-A trial lengthened median survival from 9.6 to 14.7 months, or Tibsovo (ivosidenib) with azacitidine when the leukemia has an IDH1 change. After remission comes consolidation: more cytarabine, an allogeneic stem cell transplant for people at higher risk of relapse who are fit enough, or, for those who cannot complete that path, maintenance with Onureg (oral azacitidine). If the disease comes back or never responds, the options depend on the mutation profile: Xospata (gilteritinib) for FLT3, Idhifa (enasidenib) for IDH2, Tibsovo or Rezlidhia (olutasidenib) for IDH1, and the menin inhibitors Revuforj (revumenib) for KMT2A or NPM1 and Komzifti (ziftomenib) for NPM1, approved in 2024 and 2025. Many of these pills can cause differentiation syndrome, a reaction that needs quick treatment with steroids, which is why starting them means close monitoring.

Common Symptoms of Acute Myeloid Leukemia

Recognizing the signs of Acute Myeloid Leukemia early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Feeling tired or weak, often with shortness of breath, from low red blood cells
  • Fever or infections that keep coming back, from too few working white blood cells
  • Easy bruising, nosebleeds, bleeding gums or tiny red spots under the skin, from low platelets
  • Pale skin
  • Bone or joint pain, or a feeling of fullness under the ribs from a swollen spleen or liver
  • Swollen gums, skin lumps or, rarely, a solid mass of leukemia cells called a myeloid sarcoma
  • Symptoms that feel like the flu and usually build over 4 to 6 weeks before diagnosis

Who Acute Myeloid Leukemia Affects

AML is mostly a disease of older adults. The median age at diagnosis is 70, it is diagnosed most often between 65 and 74, and it is more common in men (5.2 new cases per 100,000 a year) than women (3.7). It can occur at any age, including in children, and childhood AML is treated differently from adult AML.

Most people have no identifiable cause. Known risk factors include older age, male sex, smoking, long-term benzene exposure, earlier chemotherapy or radiation (therapy-related AML), very high radiation doses, and an earlier blood disorder such as myelodysplastic syndromes or a myeloproliferative neoplasm, which can change into AML over time (secondary AML). Some inherited conditions raise the risk, including Down syndrome, Fanconi anemia, Diamond-Blackfan anemia and Shwachman syndrome, and a small share of families carry germline changes in genes such as CEBPA, DDX41, RUNX1 or GATA2 that predispose to AML. AML is not contagious.

Find Your Next Step

Find trials I might fit Answer a few questions and see which open Acute Myeloid Leukemia trials fit your age, location and treatment history.

FDA-Approved Treatments for Acute Myeloid Leukemia

There are currently 19 FDA-approved medications for Acute Myeloid Leukemia. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

cytarabine
Generic manufacturers
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daunorubicin
Generic manufacturers (Hikma and others)
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idarubicin
Pfizer (Idamycin PFS) and generic manufacturers
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enasidenib
Bristol Myers Squibb
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decitabine and cedazuridine
Taiho Oncology
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ziftomenib
Kura Oncology (with Kyowa Kirin)
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gemtuzumab ozogamicin
Pfizer (Wyeth)
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azacitidine (oral)
Bristol Myers Squibb
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revumenib
Syndax Pharmaceuticals
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olutasidenib
Rigel Pharmaceuticals
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midostaurin
Novartis
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ivosidenib
Servier
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tretinoin (oral)
Generic manufacturers (Vesanoid brand discontinued)
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arsenic trioxide
Teva (Trisenox) and generic manufacturers
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quizartinib
Daiichi Sankyo
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venetoclax
AbbVie (co-marketed in the United States with Genentech)
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daunorubicin and cytarabine (liposomal)
Jazz Pharmaceuticals
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gilteritinib
Astellas Pharma
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Acute Myeloid Leukemia Medicines in Clinical Trials

Investigational medicines Trial Friend tracks for Acute Myeloid Leukemia, with trial results, safety findings and where each one stands.

Help Paying for Acute Myeloid Leukemia Treatment

Charity funds and drugmaker programs for Acute Myeloid Leukemia, checked at the source.

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Acute Myeloid Leukemia Resources

Reputable organizations and medical references for learning more about Acute Myeloid Leukemia, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Acute Myeloid Leukemia

Use this Acute Myeloid Leukemia clinical trial finder to see the 9 studies recruiting patients and 1 opening soon in the United States and worldwide, with eligibility criteria in plain English. Of these, 9 test a treatment and 1 is an observational study or registry; each card says which. These studies play a critical role in advancing care for blood & immune conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

New to trials? What Placebos and Randomization Actually Mean for You (In Plain English) · 4 min readWorried about cost? The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them) · 4 min read
TrialsSite mapPipeline timelineMedication checker

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

708 active trials worldwide
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RECRUITINGTreatment trialPhase 1Recently updatedNCT06575296

Revumenib for the Treatment of Acute Leukemia in Patients Post-Allogeneic Stem Cell Transplant

Intervention: Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Echocardiography, Electronic Health Record Review, Quality-of-Life Assessment, Questionnaire Administration, Revumenib

Sponsor: City of Hope Medical Center

This phase I trial tests the safety, side effects, best dose and effectiveness of revumenib in treating patients with acute leukemia after allogeneic stem cell transplant. Revumenib is in a class of medications called menin inhibitors. Revumenib targets and binds to the protein m...

Ages 2 Years+1 location
Started Dec 2024Updated todayEst. Apr 2028 (~1y 6m)
RECRUITINGTreatment trialPhase 1Recently updatedNCT06904066

Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies

Intervention: aldesleukin, cyclophosphamide, fludarabine phosphate, Individual Patient TCR-Transduced PBL, TruSight Oncology (TSO) 500

Sponsor: National Cancer Institute (NCI)

Background:

Ages 18 Years to 120 Years1 location
Expected to start Oct 2026Updated todayEst. Apr 2029 (~2y 6m)
RECRUITINGTreatment trialRecently updatedNCT07094750

Randomization for the Identification of Best Treatment Intensity for Less Fit Adults With Acute Myeloid Leukemia and Myeloid Neoplasms

Intervention: Biospecimen Collection, Bone Marrow Collection, Electronic Health Record Review, Questionnaire Administration

Sponsor: Fred Hutchinson Cancer Center

This clinical trial studies whether less fit adults with acute myeloid leukemia (AML) or myeloid neoplasms are willing to let a computer program decide (randomization) whether they receive lower- or higher-intensity chemotherapy. Historically, treatment decision-making for patien...

Ages 18 Years+1 location
Started Oct 2026Updated todayEst. Jun 2029 (~2y 7m)
RECRUITINGTreatment trialPhase 1Recently updatedNCT07012044

A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia

Intervention: Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Decitabine, Decitabine and Cedazuridine, Filgrastim, Imaging Procedure, Lumbar Puncture

Sponsor: National Cancer Institute (NCI)

This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have ...

Ages up to 21 Years14 locations
Started Jun 2026Updated todayEst. Mar 2027 (~5 months)
NOT YET RECRUITINGTreatment trialPhase 1Recently updatedNCT07511062

Axatilimab Combined With Decitabine/Venetoclax for the Treatment of TP53-mutated AML

Intervention: Decitabine, Venetoclax, Axatilimab

Sponsor: Northside Hospital, Inc. · Incyte Corporation

Axatilimab combined with Decitabine/Venetoclax for the treatment of TP53-mutated/deleted AML patients

Ages 18 Years+1 location
Expected to start Jan 2027Updated 3 days agoEst. Aug 2029 (~2y 9m)
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Find Acute Myeloid Leukemia trials near you, ranked by distance →
Active trial locations275 cities in the US
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Trial Pipeline

Jun 2024 to Oct 2031
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Can I Join an AML Clinical Trial While Taking My Current Medications?

This medication conflict checker helps people with acute myeloid leukemia find out whether the medicines they are taking now could affect clinical trial eligibility. Select one or more medications below to screen active AML trials for potential conflicts.

AML trials are unusual in that many enroll only at a fixed point in treatment: before the first dose, at first relapse, or during remission. The medicines that most often decide eligibility are the ones used to treat the leukemia itself: whether you have had venetoclax, a hypomethylating agent, a FLT3 inhibitor, an IDH inhibitor or a menin inhibitor, and how recently. Supportive medicines such as antifungals and antibiotics rarely exclude you, but a few interact with study drugs and may need to be changed.

Venetoclax and Hypomethylating Agents
Venclexta, Vidaza (azacitidine), decitabine, Inqovi, Onureg — frontline trials of new combinations usually require that you have never received venetoclax or a hypomethylating agent for AML, and relapse trials often sort people by whether the leukemia progressed on venetoclax. Most protocols ask for a washout of 14 days or 5 half-lives from the last dose, and hydroxyurea to control the white count is usually allowed up to the first study dose.
FLT3 Inhibitors
Rydapt (midostaurin), Xospata (gilteritinib), Vanflyta (quizartinib) — trials of newer FLT3 drugs or FLT3 combinations frequently exclude prior gilteritinib or quizartinib, or cap the number of prior FLT3 inhibitors, while prior midostaurin given with 7+3 is often permitted. Because these drugs and many study drugs prolong the QT interval, protocols also set a QTcF limit (commonly 450 to 480 msec) at screening.
IDH and Menin Inhibitors
Tibsovo (ivosidenib), Rezlidhia (olutasidenib), Idhifa (enasidenib), Revuforj (revumenib), Komzifti (ziftomenib) — studies of a drug in the same class usually exclude people who have already taken one, and frontline menin inhibitor trials (KOMET-017, REVEAL-ND, cAMeLot-2) require no prior treatment for AML at all. Protocols typically require that any earlier differentiation syndrome has fully resolved.
Azole Antifungals and Other CYP3A Drugs
posaconazole, voriconazole, fluconazole, isavuconazole (Cresemba), clarithromycin — these are not reasons for exclusion, but most AML study drugs are cleared by CYP3A4 and the protocol will either lower the study drug dose or ask your team to switch antifungals. Tell the study team about every antifungal, antibiotic and antiviral you take, because the dose tables depend on them.
Steroids, Hydroxyurea and Transfusion Support
dexamethasone, prednisone, hydroxyurea, growth factors (filgrastim), transfusions — short steroid courses for differentiation syndrome, hydroxyurea for a high white count, and transfusions are allowed in nearly every AML trial, often right up to the first dose. Growth factors are usually paused around response assessments so that they do not confuse the count recovery that defines remission.
Post-Transplant Medicines
tacrolimus, cyclosporine, sirolimus, Jakafi (ruxolitinib), Rezurock (belumosudil) — trials for relapse after a transplant usually require that you are off immune suppression, or on a stable low dose, and have no active graft-versus-host disease of a set grade. Maintenance trials after transplant have their own windows, often starting 30 to 100 days after the transplant.
Timing matters more than any single medicine
Most AML trials are built around a treatment moment: newly diagnosed and untreated, first relapse, or remission after a set number of cycles. Once you take the first dose of standard therapy, many frontline trials close to you. If a trial interests you, ask your leukemia doctor to check it before treatment starts, even if that means a day or two of delay that your doctor judges safe.
A conflict does not always mean you cannot join
Many conflicts come with a washout period, often 14 days or 5 half-lives for pills and 4 weeks for antibodies, after which you may qualify. Whether a pause is safe depends on how active the leukemia is, so this is always a decision with your hematologist, and hydroxyurea is usually allowed to hold the white count during the gap.
Supportive care is almost always allowed
Antibiotics, antivirals, anti-nausea medicines, transfusions and pain medicines rarely appear in exclusion criteria. When the checker flags an antifungal, the excerpt is usually about dose adjustment of the study drug rather than exclusion; read the text carefully and bring it to the study coordinator.
Do not see your medication listed?
The checker covers the medicines most relevant to AML trials: leukemia treatments, antifungals, post-transplant immune suppression and common supportive medicines. If yours is not in the list, we have not found it commonly named in AML trial criteria. The study coordinator can confirm it against the protocol.
How the medication conflict checker works: This free tool helps people with acute myeloid leukemia learn whether their current medications could affect clinical trial eligibility. It scans the published eligibility criteria of active AML trials on ClinicalTrials.gov and flags which ones may exclude or restrict your treatment. Matches are grouped by confidence: high confidence means the trial names your exact drug, medium means it names the drug class, and low means the wording could apply to you. It is a starting point for a conversation with your leukemia team, not a determination of eligibility.

Across 1,853 open rare disease treatment trials, a third exclude people over a medication they commonly take. See which medications and diseases, in our September 2026 analysis.

Sources: Drug classes and warnings come from the FDA prescribing information for each medicine (openFDA, read October 19, 2026); washout and timing patterns are summarized from the eligibility criteria of AML trials registered on ClinicalTrials.gov and vary by trial.

Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Acute Myeloid Leukemia patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Acute Myeloid Leukemia treatments, clinical trial participation, and day-to-day disease management.

Find the right community
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Blood Cancer United (formerly the Leukemia & Lymphoma Society): AML
Plain-language AML guide, free booklets, information specialists, clinical trial nurse navigators and the Beat AML precision medicine trial
Blood Cancer United Clinical Trial Support Center
Nurse navigators who search for AML trials for you and talk through the options at no cost
Blood Cancer United Patient Community
Online forum for people with blood cancers and their families, with leukemia discussion groups
Blood Cancer United Family Support Groups
Free support groups, 95 in all, that meet monthly in person or on Zoom, led by oncology mental health professionals; find yours through your local Region
Aplastic Anemia and MDS International Foundation: AML
Education, a patient helpline (800-747-2820), virtual support groups, a peer support network and free patient conferences that include AML tracks
AAMDSIF Patient and Family Webinars
Recorded expert webinars with an AML topic filter, including sessions on secondary AML and leukemic transformation
Know AML
Global AML awareness initiative with animated explainers on genetic testing, measurable residual disease, relapse and treatment, plus caregiver and mental health pages
NMDP Patient Support Center
Free navigators for anyone considering a blood or marrow transplant, peer connections with transplant recipients, and grants for travel, food and copays; call or text 1-888-999-6743
NCI PDQ: Acute Myeloid Leukemia Treatment (patient version)
The National Cancer Institute's reviewed overview of diagnosis, staging and treatment, updated May 2025

Events

Free in-person conferences where people with AML and their families hear from leukemia specialists and meet others with the same diagnosis. Blood Cancer United (formerly the Leukemia & Lymphoma Society) runs regional Blood Cancer Conferences with an adult AML session, and the Aplastic Anemia and MDS International Foundation runs Patient and Family Conferences with an MDS, AML and MPN track. Online options are listed under communities.

Atlanta, GA (venue to be announced by the host)
Blood Cancer United
Blood Cancer UnitedFrom bloodcancerunited.org, checked October 19, 2026
Patient Conference

Southern Blood Cancer Conference (Blood Cancer United)

When
Saturday, October 24, 2026 (times not yet posted)
Where
Atlanta, GA
Cost
Free
For
Patients, survivors, caregivers, family members and health professionals

A free education conference for blood cancer patients, survivors, caregivers, family members and healthcare professionals, covering treatment options, emerging therapies, survivorship and Blood Cancer United's own resources. The 2026 national virtual conference in September included a dedicated adult AML session, and the regional conferences follow the same format.

Blood Cancer United's page lists the date, city and registration link; the venue and schedule had not been posted when this was checked, so look for them when you register.

Chicago, IL (venue to be announced by the host)
Blood Cancer United
Blood Cancer UnitedFrom bloodcancerunited.org, checked October 19, 2026
Patient Conference

Upper Midwest Blood Cancer Conference (Blood Cancer United)

When
Saturday, October 31, 2026 (times not yet posted)
Where
Chicago, IL
Cost
Free
For
Patients, survivors, caregivers, family members and health professionals

A free education conference for blood cancer patients, survivors, caregivers, family members and healthcare professionals, with sessions on treatment options, emerging therapies, survivorship and clinical trials.

Blood Cancer United's page lists the date, city and registration link; the venue and schedule had not been posted when this was checked.

UT MD Anderson Cancer Center, Frank E. Anderson Conference Hall
1515 Holcombe BlvdHoustonTX77030US
Aplastic Anemia and MDS International Foundation
Aplastic Anemia and MDS International FoundationFrom aamdsif.salsalabs.org, checked October 19, 2026
Patient Conference

AAMDSIF Houston Patient and Family Conference

When
Saturday, November 7, 2026, 8:00 AM to 2:45 PM Central
Where
MD Anderson Cancer Center, Houston, TX
Cost
Free; parking is complimentary
For
Patients, family members and caregivers; health professionals welcome

A free, in-person conference for patients, family members and caregivers affected by bone marrow failure diseases including AML. The MDS, AML and MPN track runs from 8:00 AM to 2:45 PM Central: an overview of bone marrow failure, a session on treatment and management of lower-risk MDS, higher-risk MDS, AML and MDS/MPN overlap, an Ask the Experts panel for MDS, AML and MPN, a transplant decision-making session, lunch, a Living Well session and a survivorship patient panel. The agenda is subject to change.

Registration is open to patients, families and healthcare professionals. Questions go to help@aamds.org or 800-747-2820, extension 140.

Details come from each host's own page, checked October 19, 2026. Blood Cancer United had not yet posted start times or venues for its fall conferences, so confirm them when you register.

Related Conditions

Conditions that share genes, symptoms or treatments with Acute Myeloid Leukemia, followed by others in the blood & immune category. Patients with Acute Myeloid Leukemia may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Acute Myeloid Leukemia Treatments

8 pharmaceutical companies have Acute Myeloid Leukemia in their rare disease portfolio

Frequently Asked Questions About Acute Myeloid Leukemia