NCT05955261 · St. Jude Children's Research Hospital
A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia
What this study is about
This is a phase 2 study to test the hypothesis that venetoclax in combination with standard chemotherapy will be tolerable and active in pediatric patients with newly diagnosed acute myeloid leukemia (AML).
View original scientific description
This is a phase 2 study to test the hypothesis that venetoclax in combination with standard chemotherapy will be tolerable and active in pediatric patients with newly diagnosed acute myeloid leukemia (AML).
Interventions
DRUG
Venetoclax
Venetoclax will be given with each course of therapy. Patients with low-risk AML will receive four courses of therapy, intermediate-risk patients will receive five courses of therapy, and high-risk patients will receive two or three courses of therapy followed by hematopoietic stem cell transplantation.
DRUG
Azacitidine
Given IV over 30 minutes on days 1-5
DRUG
Cytarabine
Given IV over 30 minutes q12 hours on days 1-8 (16 doses)
DRUG
Gemtuzumab Ozogamicin
Given IV
DRUG
Daunorubicin Hydrochloride
IV over 1 hour on days 1, 3, and 5
DRUG
Fludarabine Phosphate
Given IV over 30 minutes on days 1-5
DRUG
Idarubicin Hydrochloride
Given IV over 15 minutes on days 3-5
DRUG
Mitoxantrone Hydrochloride
IV over 1 hour on days 2-4
DRUG
Etoposide
Given IV over 1 hour on days 1-5
DRUG
Gilteritinib
PO on days 8-28 (21 doses)
Primary outcome measures
Minimal residual disease (MRD)-negativity rate
Time frame: At day 29 after induction 1
Will compute a binomial confidence interval for the proportion of MRD-evaluable patients who become MRD-negative (defined as MRD \< 0.1%) after induction 1.
Incidence of death or unacceptable adverse event
Time frame: From initiation to completion of each course of therapy, an average of 6 weeks
A patient is deemed to have tolerated a course of therapy if they complete that course without death or an unacceptable adverse event. Will monitor the tolerability or each course using multi-stage binomial stopping rules. Will use the method of Jung and Kim to compute 95% confidence intervals that adjust for the multi-stage monitoring.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 biliru
Where
- Madera, California
- Orange, California
- San Diego, California
- Washington D.C., District of Columbia
- Chicago, Illinois
- Boston, Massachusetts
- Charlotte, North Carolina
- Cincinnati, Ohio
- Memphis, Tennessee
- Fort Worth, Texas
Collaborators
AbbVie
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 17, 2026 · Source of record for eligibility and locations