Leukemia — Acute Myeloid (AML) Clinical Trials

About Leukemia — Acute Myeloid (AML) trials

AML trial design begins with mutation profile because most therapy decisions and trial eligibility depend on which genetic alterations are present. FLT3 mutations (FLT3-ITD and FLT3-TKD, present in roughly 30% of AML) define eligibility for FLT3 inhibitor trials. IDH1 and IDH2 mutations (about 25% combined) gate IDH inhibitor trials and combination strategies. NPM1 mutations (roughly 30%) drive trials of menin inhibitors and inform risk stratification. TP53 mutations define a high-risk subgroup with its own trial set, often combination-based. KMT2A (also called MLL) rearrangements drive menin inhibitor trials. RUNX1, CEBPA, ASXL1, DNMT3A, and TET2 are common in AML and inform risk stratification by ELN criteria. KIT mutations matter most in core-binding factor AML (t(8;21) or inv(16)). NRAS and KRAS mutations contribute to RAS-pathway trial design and often co-occur with other drivers.

Trials split first by treatment intent: newly diagnosed fit, newly diagnosed unfit, and relapsed or refractory (R/R). Newly diagnosed fit trials test additions to the 7+3 induction backbone, with mutation-specific targeted agents added based on the patient's profile. Newly diagnosed unfit trials center on hypomethylating agent (HMA) plus venetoclax as the standard backbone, with novel agents added for specific genotypes. R/R trials are heavily mutation-driven: FLT3-mutated patients have separate trials, as do IDH1, IDH2, NPM1, and KMT2A-rearranged patients, each with dedicated targeted strategies. Pre-allogeneic transplant trials test strategies for clearing measurable residual disease (MRD) before stem cell transplant. Maintenance trials test post-induction or post-transplant strategies. Acute promyelocytic leukemia (APL) trials are rare but distinct, focused on optimizing the curative ATRA + arsenic trioxide backbone.

AML trial eligibility centers on mutation status, treatment fitness, and disease stage. Mutation testing is foundational: most R/R trials require confirmation of a specific mutation (FLT3, IDH1, IDH2, KMT2A, or NPM1) and many newly diagnosed trials are mutation-stratified or mutation-restricted. Treatment intent classification (newly diagnosed fit, newly diagnosed unfit, R/R) determines which trials apply. ELN risk category (favorable, intermediate, adverse) appears in many trial inclusion or stratification criteria. Prior therapy exposure: HMA, intensive induction chemotherapy (typically 7+3), and which targeted agents the patient has received. Prior allogeneic stem cell transplant status and time since transplant matter for post-transplant trials. ECOG performance status, with most trials accepting 0 to 2. Cardiac function (especially LVEF) is important for trials using or planning anthracyclines. White blood cell count and blast percentage at screening may need to be within trial-specific bounds.

Biomarkers tested in Leukemia — Acute Myeloid (AML) trials

These are the molecular markers most commonly required or evaluated in Leukemia — Acute Myeloid (AML) eligibility criteria. OncoMatch extracts them from each trial's protocol and matches them against your test results.