Acute myeloid leukemia (AML) is an aggressive blood cancer where immature white blood cells accumulate rapidly in the bone marrow and crowd out healthy cells. It is one of the more difficult leukemias to treat, though a wave of targeted drugs has transformed outcomes for specific genetic subtypes.
What's actually going on in research
Targeted therapies against IDH1, IDH2, and FLT3 mutations — all common in AML — have moved from trials into standard care, and many patients who carry these mutations now have chemotherapy-free or reduced-intensity options. Venetoclax, a BCL-2 inhibitor, combined with low-intensity chemotherapy has become standard for older adults unfit for intensive treatment. Menin inhibitors represent the newest class, showing remarkable early responses in patients with NPM1 mutations and KMT2A rearrangements.
Menin inhibitors
A new class of drugs blocking the menin–MLL1 interaction is showing striking responses in AML with NPM1 mutations or KMT2A rearrangements, both in relapsed disease and now in earlier-line combinations.
FLT3 and IDH inhibitors
Oral targeted drugs against FLT3 and IDH1/2 mutations are now standard for those subtypes, and trials are refining the best combinations and testing maintenance therapy after transplant.
Venetoclax combinations
The BCL-2 inhibitor venetoclax combined with azacitidine has changed treatment for older adults with AML. Trials are testing it with newer targeted agents and in fit patients receiving intensive chemotherapy.
What to know before you search
Eligibility depends on AML subtype, specific mutations (FLT3, IDH1/2, NPM1), fitness for intensive chemotherapy, and whether the disease is newly diagnosed or relapsed/refractory.
What types of trials are currently open
- Treatment trials — Testing new drug combinations in newly diagnosed or relapsed and refractory AML.
- Targeted therapy trials — Testing mutation-specific drugs against FLT3, IDH1/2, NPM1, and other AML drivers.
- Transplant trials — Comparing conditioning regimens and post-transplant maintenance strategies to prevent relapse.
- Older adult trials — Testing lower-intensity regimens designed for patients who cannot tolerate standard intensive chemotherapy.
- Supportive care trials — Managing infection, transfusion needs, and treatment toxicity during and after AML therapy.
Recently added Acute Myeloid Leukemia trials
Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
The goal of this clinical trial is to test BSB-2002 which is a new type of cellular therapy to treat blood cancer (AML). It will evaluate the safety of BSB-2002 and also determine whether it works to prevent relapse of your cancer.
Cord Blood Transplantation in Children and Young Adults With Blood Cancer
The purpose of this study is to find out whether Cord Blood Transplantation/CBT as the first or second transplant is an effective treatment for children and young adults with blood cancer.
Find Acute Myeloid Leukemia trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.