Venetoclax combined with chemotherapy for children with acute myeloid leukemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 2 study investigates the safety and effectiveness of venetoclax when used alongside standard chemotherapy in pediatric patients diagnosed with acute myeloid leukemia (AML). The study aims to determine how well patients tolerate this combination treatment and to measure the rate of minimal residual disease negativity after one course of therapy. Treatment plans will be tailored based on the genetic characteristics of the patients and their response to therapy, with different chemotherapy courses for low, intermediate, and high-risk patients. The study will also evaluate overall survival and event-free survival rates among participants.

Why it matters

Who can join: the full rules

Patients with preexisting conditions that may affect chemotherapy tolerance (e.g., bone marrow failure syndrome, cardiac disease, pulmonary disease, and history of significant hepatic impairment) should be discussed with study PI in detail to enable review of the medical history prior to enrollment onto AML23 study.

Inclusion Criteria:

* Diagnosis of AML fulfilling the criteria of the WHO classification of myeloid neoplasms or \< 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality or myeloid sarcoma or primary myelodysplastic syndrome (MDS) with ≥ 10% blasts or a complete blood count with the presence of at least 1,000 blasts/μL (e.g., a WBC count ≥ 10,000/μL with ≥ 10% blasts or a WBC count ≥ 5,000/μL with ≥ 20% blasts
* Age \> 28 days and \< 22 years
* No prior therapy for this malignancy except for one dose of intrathecal therapy and hydroxyurea or low-dose cytarabine (≤ 200 mg/m\^2 per day for ≤ 7 days)
* Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior to enrollment
* Male and female participants of reproductive potential must agree to use an effective contraceptive method during the study and for 6 months after study treatment
* Written informed consent from the patient and/or parent/legal guardian
* Direct bilirubin ≤ 1.5 x institutional upper limit of normal

Exclusion Criteria:

* Patients with treatment-related AML, Down syndrome, acute promyelocytic leukemia, chronic myeloid leukemia in blast crisis, juvenile myelomonocytic leukemia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or other bone marrow failure syndromes are not eligible
* Uncontrolled systemic fungal, bacterial, or viral infection or significant concurrent disease that would compromise patient safety or compliance, study participation, follow up, or interpretation of study results
* Prior exposure to any dose of anthracycline or anthracenedione
* Patients may not receive strong or moderate CYP3A inducers, such as rifampin, within 3 days of enrollment
* Patients may not receive moderate or strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, voriconazole, posaconazole) within 3 days of enrollment.

Where it's running

• St. Jude Children's Research Hospital
  Memphis, Tennessee, United States Recruiting
• Valley Children's Hospital
  Madera, California, United States Suspended
• Children's Hospital of Orange County
  Orange, California, United States Suspended
• Rady Children's Hospital-San Diego
  San Diego, California, United States Suspended
• Children's National Medical Center
  Washington D.C., District of Columbia, United States Suspended
• Ann & Robert H. Lurie Children's Hospital
  Chicago, Illinois, United States Active not recruiting

Study contacts

• Principal investigator: Hiroto Inaba, MD, PhD — St. Jude Children's Research Hospital
• Study coordinator: Hiroto Inaba, MD, PhD
• Email: referralinfo@stjude.org
• Phone: 888-226-4343

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.