AML Clinical Trial Tracker — Monitor Acute Myeloid Leukemia Research in 2026

Data from ClinicalTrials.gov via DataLookout pipeline. Updated daily. As of March 2026.

Why AML trial monitoring is uniquely challenging

Acute myeloid leukemia has one of the most rapidly evolving treatment landscapes in oncology. With 347 active studies and 174 currently recruiting, the molecular complexity of AML — spanning FLT3, IDH1/2, NPM1, KMT2A, TP53, and dozens of other mutations — means that new targeted agents enter clinical development continuously. No other leukemia has seen this many simultaneous Phase 3 readouts in a two-year window.

Key signals to monitor in the AML landscape:

• Menin inhibitor trials (revumenib, ziftomenib) — validated in r/r disease, now expanding into newly diagnosed AML in Phase 3
• Venetoclax backbone combinations with novel targeted agents in frontline and relapsed/refractory settings
• Post-transplant maintenance trials — MRD-guided and FLT3-directed maintenance post-alloHCT
• TP53-mutant AML — historically treatment-resistant, with emerging MDM2 inhibitor and p53 reactivator programs
• ADC and bispecific T-cell engager programs targeting CD33, CD123, and CLL-1
• Pediatric AML — a separate pipeline from adult disease, with dedicated cooperative group trials

Phase 3 AML trials actively recruiting — 2026

These are the pivotal trials that will define the next generation of AML treatment. Competitive intelligence teams tracking these registrations have earlier signal on competitor development priorities than any other channel.

NCT ID	Trial / Agent	Sponsor	Phase
NCT07211958	Revumenib + intensive induction chemotherapy in newly diagnosed AML (KMT2A/NPM1)	Syndax Pharmaceuticals	Phase 3
NCT07007312	Ziftomenib + ven/aza or 7+3 induction in AML patients with KMT2A or NPM1 alterations	Kura Oncology	Phase 3
NCT07463651	MRD-guided maintenance post-alloHCT: gilteritinib vs sorafenib in FLT3-mutant AML	Soochow University	Phase 3
NCT05183035	Venetoclax in pediatric patients with relapsed or refractory AML	PedAL BCU / Children's Oncology	Phase 3
NCT04628026	Venetoclax + induction and consolidation chemotherapy in AML (frontline, fit patients)	University of Ulm	Phase 3
NCT05429632	Mocravimod (S1P receptor modulator) as adjunctive and maintenance therapy in post-HCT AML	Priothera SAS	Phase 3
NCT04708054	Venetoclax to improve outcomes of fractionated busulfan conditioning in AML (pre-transplant)	MD Anderson Cancer Center	Phase 3
NCT04293562	CPX-351 and/or targeted agents vs standard chemotherapy in pediatric newly diagnosed AML	Children's Oncology Group	Phase 3
NCT04256317	ASTX030 (oral azacitidine/cedazuridine) in MDS/AML — Phase 2/3	Taiho Oncology	Phase 2/3

Source: ClinicalTrials.gov, DataLookout pipeline. March 2026. Recruiting status verified at last pipeline run.

The AML treatment landscape in 2026

Menin inhibitors — validated mechanism, now expanding into frontline

The approval of revumenib (Revuforj, Syndax Pharmaceuticals) in December 2024 for relapsed/refractory AML with KMT2A rearrangement or NPM1 mutation was the most significant mechanism validation in AML since venetoclax. Menin inhibitors disrupt the menin-KMT2A interaction critical for oncogenic gene expression in these leukemia subtypes. Two Phase 3 trials are now enrolling to expand this class into frontline therapy:

• Revumenib + intensive induction (NCT07211958, Syndax): Testing whether adding a menin inhibitor to 7+3 or similar induction improves CR/CRi rates in newly diagnosed KMT2A-r and NPM1-mutant AML. This is the pivotal trial that would shift revumenib from r/r to frontline use.
• Ziftomenib + ven/aza or 7+3 (NCT07007312, Kura Oncology): Ziftomenib, a next-generation menin inhibitor with a different structural scaffold from revumenib, is testing two combination backbones — the ven/aza standard for unfit patients and 7+3 for fit patients. The split design allows Kura to simultaneously address both frontline populations.

Beyond these two, several earlier-stage menin inhibitor programs (BMF-219 from Biomea, DS-1594 from Daiichi Sankyo) are in Phase 1/2. Monitoring Phase 1 trial initiations is the earliest signal of new menin inhibitor entrants — these registrations typically precede public announcement of Phase 1 data by 12-18 months.

Venetoclax combinations beyond ven + azacitidine

Venetoclax + azacitidine (Ven/Aza) established a new standard for older/unfit patients and is now the backbone for a wave of triplet combination trials. The key question in the current trial landscape is which triplet partners add value without unacceptable toxicity:

• Ven + FLAG-IDA: Academic groups (MD Anderson, European centers) are testing whether adding venetoclax to the salvage backbone FLAG-IDA (fludarabine + high-dose cytarabine + idarubicin + G-CSF) improves outcomes in relapsed/refractory fit patients.
• Ven + cladribine combinations: Multiple Phase 2 trials testing cladribine-based triplets, particularly in older patients or those with secondary AML from MDS.
• Ven + FLT3 inhibitors: Gilteritinib + ven + aza triplets are being tested across multiple Phase 1/2 trials; the frontline unfit population is the primary target.
• Ven + IDH inhibitors: Enasidenib + ven + aza and ivosidenib + ven + aza triplets are enrolling in IDH1/IDH2-mutant AML — an approach that may create a targeted backbone for this molecular subtype.

FLT3 inhibitors — post-transplant and combination strategies

The frontline role of FLT3 inhibitors (midostaurin in fit patients, gilteritinib in r/r) is established. The active clinical question in 2026 is post-transplant maintenance. The Phase 3 trial NCT07463651 directly addresses this: MRD-guided selection between gilteritinib and sorafenib for post-alloHCT FLT3-mutant AML, testing whether MRD status at transplant should drive maintenance agent choice. This design represents a meaningful refinement of the QuANTUM-First maintenance paradigm.

Next-generation FLT3 inhibitors — particularly those with dual FLT3/KIT or FLT3/CDK inhibitory activity — are entering Phase 1. These are early-stage signals that Phase 2 data will arrive in 2027-2028.

TP53-mutant AML — the hardest problem in the disease

TP53-mutant AML (approximately 10-15% of AML cases) has historically shown poor response to all available therapies, including ven/aza. Two distinct mechanisms are in active clinical development:

• MDM2 inhibitors (navtemadlin, others): MDM2 inhibitors restore TP53 function in tumors with wild-type TP53, but their role in TP53-mutant AML is being tested in combination with hypomethylating agents on the hypothesis that MDM2 inhibition has TP53-independent effects (TRAIL pathway, etc.).
• p53 reactivators (eprenetapopt, APR-246): These small molecules refold mutant p53 protein toward functional conformation. Phase 2 combination data with azacitidine have been published; Phase 3 development stalled after a failed registration trial. Second-generation p53 reactivators are entering Phase 1.
• Magrolimab (anti-CD47): The CD47 "don't eat me" signal pathway — despite the Gilead program pause for safety — remains an active research direction in TP53-mutant MDS/AML through academic programs and competing anti-CD47 molecules.

Antibody-drug conjugates and bispecifics — CD33, CD123, CLL-1

AML presents multiple surface antigens amenable to ADC and bispecific T-cell engager (BiTE) approaches. The target landscape:

• CD33: The original AML target (gemtuzumab ozogamicin). Next-generation CD33 ADCs with improved linker-payload combinations, and CD33-directed bispecifics, are in Phase 1/2.
• CD123: Expressed on AML blasts and leukemic stem cells. Multiple programs from Pfizer, AbbVie, and mid-cap biotechs testing ADCs and bispecifics; talacotuzumab data established proof of concept despite modest single-agent activity.
• CLL-1 (CLEC12A): Relatively AML-specific antigen, with lower expression on normal hematopoietic progenitors than CD33 or CD123. Phase 1/2 bispecific programs from multiple sponsors, primarily in r/r disease.
• FLT3-directed bispecifics: Emerging FLT3 × CD3 bispecific T-cell engagers are entering Phase 1 — a distinct strategy from FLT3 kinase inhibition, using the immune system to target FLT3-expressing blasts.

Transplant conditioning and post-transplant maintenance

Allogeneic stem cell transplant (alloHCT) remains central to AML management in eligible patients. The clinical trial landscape in this space includes novel conditioning regimens, GvHD prevention strategies, and post-transplant maintenance. Venetoclax-based conditioning intensification (NCT04708054, MD Anderson) tests whether adding venetoclax to busulfan-based myeloablative conditioning improves disease control without excess toxicity. Post-transplant trials like NCT05429632 (mocravimod) explore immune modulation approaches to reduce relapse risk after transplant.

What we monitor for AML

Our pipeline pulls directly from the ClinicalTrials.gov API every morning. For an AML watch profile, configure:

• Condition keywords: "acute myeloid leukemia", "AML", "FLT3-mutant AML", "IDH1 AML", "IDH2 AML", "NPM1-mutant AML", "KMT2A-rearranged AML", "secondary AML"
• Phase filter: Phase 1 only (early pipeline), Phase 2/3 (late-stage), or all phases
• Sponsor filter: Industry-sponsored only (competitive intelligence) or all sponsors including NCI, ECOG-ACRIN, and academic centers
• Status filter: Recruiting only, or all active trials including those not yet recruiting

Who uses AML trial monitoring

Hematology-oncology pharma and biotech teams

BD and competitive intelligence teams at companies with AML programs systematically track competitor trial registrations. A new IND filing or Phase 2 trial initiation reveals competitor development strategy months before any public presentation. For menin inhibitors especially — where the frontline expansion race is unfolding across multiple sponsors — early signal on Phase 3 trial design (which backbone, which patient population) is directly actionable for portfolio decisions.

Academic hematologists and cooperative group investigators

Physician-scientists at academic medical centers and cooperative group members monitor trial registrations to identify collaboration opportunities, understand overlapping trial designs, and ensure investigator-initiated studies differentiate from commercial programs. With 79 Phase 1 AML trials recruiting — many at academic centers — there is a genuine need to track the field systematically.

Biotech and biopharma investors

AML is a high-value target indication. Investors monitoring Phase 1 trial initiations gain earlier visibility into program value than those relying on press releases or conference presentations. The menin inhibitor race is the clearest current example: Phase 3 registrations for revumenib and ziftomenib are now public signals that can be tracked weeks before analyst commentary catches up.

Frequently asked questions

How many AML trials are currently recruiting?

As of March 2026, 174 AML clinical trials are actively recruiting on ClinicalTrials.gov, out of 347 total active AML studies. DataLookout checks for new and updated trials every morning and sends a digest the same day.

What are the key Phase 3 AML trials in 2026?

The most significant Phase 3 trials currently recruiting are: NCT07211958 (revumenib + intensive induction, Syndax), NCT07007312 (ziftomenib + ven/aza or 7+3, Kura Oncology), and NCT07463651 (MRD-guided gilteritinib vs sorafenib post-transplant). These three trials together will define whether menin inhibitors become frontline standards and how FLT3-directed post-HCT maintenance is optimized.

Can I track specific AML molecular subtypes separately?

Yes. On the Starter plan ($29/month), you can create up to 3 watchlists. For example: one profile for FLT3-mutant AML, another for IDH1/2-mutant trials, and a third for all Phase 3 recruiting trials regardless of molecular subtype. The free plan gives you one watchlist permanently at no cost.

Does this cover international AML trials?

ClinicalTrials.gov includes trials with international sites, so major industry-sponsored global programs from European (HOVON, German AML Study Group) and Asian sponsors are captured — particularly those with US enrollment sites or US IND status.

How is this different from ClinicalTrials.gov alerts?

ClinicalTrials.gov offers basic RSS alerts without phase filtering, sponsor type filtering, or clean digest formatting. DataLookout provides the intelligence layer: filtered, labeled, and organized daily alerts for AML specifically — with sponsor classification, status change detection, and the ability to set multiple watchlists for different molecular subtypes.