Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia
A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
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Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome
What is acute myeloid leukemia?
Acute myeloid leukemia, or AML, is a fast growing cancer of the bone marrow in which immature myeloid cells multiply and crowd out normal blood production. That causes anemia and tiredness, infections, and easy bruising or bleeding, often within weeks. It is most often diagnosed between ages 65 and 74 and is slightly more common in men. Prior chemotherapy, some blood disorders such as MDS, and smoking raise the risk, but most cases have no known cause.
Treatment starts quickly and depends on fitness and the leukemia's genetics. People who can tolerate it receive intensive chemotherapy, often followed by a stem cell transplant; older or less fit people usually receive venetoclax with a lower intensity drug. Targeted medicines exist for FLT3, IDH1, IDH2, and, since 2024, NPM1 and KMT2A changes. Overall about 3 in 10 people are alive five years after diagnosis, with much better results in younger patients with favorable genetics. Other leukemias are on our leukemia clinical trials page.
Types of acute myeloid leukemia clinical trials
Precision medicine screening studies. National programs that test leukemia genetics within days and assign patients to matching trials.
Targeted drug trials. Menin inhibitors for NPM1 and KMT2A changes, and FLT3 and IDH inhibitors, combined with standard treatment in Phase 3.
Trials for older and less fit patients. How long to continue venetoclax, how to reduce side effects, and new combinations for people who cannot have intensive chemotherapy.
Transplant trials. Conditioning regimens, donor options, and preventing relapse after transplant; see stem cell therapy clinical trials.
Relapsed and refractory trials. Immunotherapies, antibody drug conjugates, and new agents for leukemia that returns.
Measurable residual disease studies. Sensitive blood and marrow tests to guide when to intensify or stop treatment.
Is acute myeloid leukemia curable?
For some people, yes, mainly younger patients with favorable genetics treated with intensive chemotherapy or a transplant. Overall about 3 in 10 people are alive five years after diagnosis, and results are lower for older adults, which is why trials for them are a priority.
Who can participate in acute myeloid leukemia clinical trials?
Because AML must be treated within days, many trials enroll at diagnosis, before treatment begins, and some first require the leukemia's genetic results, which screening studies return quickly. Trials specify age and fitness, typically 18 to 59 for intensive regimens and 60 or 75 and older for lower intensity ones, and often a mutation such as NPM1, FLT3, or KMT2A. Relapsed trials require details of prior treatment and any transplant. Adequate heart, liver, and kidney function are usual requirements. Eligibility always varies by study.
How AML is classified, and why it matters for trials
Genetic risk. Favorable, intermediate, or adverse, based on mutations and chromosome changes; it decides whether a transplant is recommended.
Fitness. Whether intensive chemotherapy is safe, judged by age and other illnesses; trials are usually built for one group or the other.
Treatment phase. Newly diagnosed, in remission, or relapsed; each has its own trials.
Specific mutations. NPM1, FLT3, IDH1, IDH2, and KMT2A changes each open targeted trials.
How to enroll in an acute myeloid leukemia clinical trial
Identify your trial. Use the filters; because timing is tight, ask your treating center about trials on the day of diagnosis and apply to every study that fits.
Select your preferred location. Enter your city or state; national programs run at hundreds of sites, including community cancer centers.
Explore study details. Click "Learn More" for age and fitness rules, mutation requirements, and whether a hospital stay is involved.
Complete the health profile. Click "Get started" to begin the 5-step application. Have ready your genetic and risk results, any treatment already started, and your other health conditions; a family member can complete it for you.
Submit the application. A clinical trial coordinator reviews it and contacts you. Nothing is decided until informed consent, and participation is voluntary at every step.