The study drug, Venetoclax, is approved by the FDA to treat AML in adults, but it is not currently approved for use in children. This means it is considered investigational. Venetoclax will be given to you or your child along with standard-of-care chemotherapy. Venetoclax will be taken by mouth. The chemotherapy drugs in this study will be given through a central line. The treatment phase of the study will last between six months to one year. The exact length of the study and the treatment given will depend on how severe you or your child's AML is. There are two types of visits in this study, inpatient visits and outpatient visits. Inpatient visits will take place any time you or your child gets chemotherapy during the study. During these visits you or your child will have to stay at the hospital. Outpatient visits only last a few hours and happen a few days after you or your child get chemotherapy. All study visits, no matter the type, will take place at Children's Hospital Colorado. At study visits we will ask about you or your child's medical history and perform physical exams. We will also take blood samples from you or your child's arm with a needle or through a central line. We will ask you or your child to give urine samples. We will take pictures and do tests to check the function of you or your child's heart. We will also do a lumbar puncture to do tests on the spinal fluid. We will take small samples (biopsies) of you or your child's bone marrow to see how well the treatment works. We will also take a small skin sample to see if there is a genetic cause for AML. This data may help us better treat you or your child. It may also show whether other people in your family could have a higher risk of cancer.

Who can Participate

You or your child may be able to join this study if you are between 28 days and 22 years old and have a diagnosis of acute myeloid leukemia. If you or your child is of reproductive potential, you must be willing to use an effective form of contraception during the study. You or your child cannot join if you have had any prior exposure to anthracycline or anthracenedione. You or your child cannot join if you have received any prior treatment for this leukemia. However, certain short term treatments, such as one dose of chemotherapy into the spinal fluid or brief use of hydroxyurea or low dose cytarabine, are allowed. Before joining, a study doctor will review all criteria to make sure you or your child qualify and that it is safe for you to join.

Study ID

More information available at ClinicalTrials.gov: NCT05955261