Recruiting
Phase 2

Venetoclax with Chemotherapy

Sponsor:

St. Jude Children's Research Hospital

Code:

NCT05955261

Conditions

Acute Myeloid Leukemia

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

Interventions

Venetoclax

Azacitidine

Cytarabine

Gemtuzumab Ozogamicin

Daunorubicin Hydrochloride

Study Details

Brief summary:

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).

Primary Objectives:

  • Establish the tolerability adding venetoclax to standard chemotherapy in pediatric patients with AML
  • Estimate the proportion of patients who become minimal residual disease (MRD) negative by flow cytometry after one course of venetoclax-based induction therapy

Secondary Objectives:

\- Estimate the rates of complete remission (CR), event-free survival (EFS), and overall survival (OS) in pediatric patients who receive venetoclax-based chemotherapy

Conditions

Acute Myeloid Leukemia

Study ID

NCT05955261

Start date

Jul 25, 2023

Status verified date

Sep, 2026

Completion date

Mar, 2034

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

70 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Low Risk

All eligible patients receive intervention according to the Detailed Description section with the following:

Venetoclax, Cytabrine, Danunorubicin Hydrochloride, Gemtuzumab Ozogamicin, Etoposide, Mitoxantrone Hydrochloride, Gilteritinib

experimental: Intermediate Risk

All eligible patients receive intervention according to the Detailed Description section with the following:

Venetoclax, Cytabrine, Danunorubicin Hydrochloride, Fludarabine Phosphate, Gemtuzumab Ozogamicin, Etoposide, Idarubin Hydrochloride, Mitoxantrone Hydrochloride, Gilteritinib

experimental: High Risk

All eligible patients receive intervention according to the Detailed Description section with the following:

Venetoclax, Azacitidine, Cytabrine, Danunorubicin Hydrochloride, Fludarabine Phosphate, Gemtuzumab Ozogamicin, Etoposide, Idarubin Hydrochloride, Gilteritinib

Interventions

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.

Azacitidine

Given IV over 30 minutes on days 1-5

Cytarabine

Given IV over 30 minutes q12 hours on days 1-8 (16 doses)

Gemtuzumab Ozogamicin

Given IV

Daunorubicin Hydrochloride

IV over 1 hour on days 1, 3, and 5

Fludarabine Phosphate

Given IV over 30 minutes on days 1-5

Idarubicin Hydrochloride

Given IV over 15 minutes on days 3-5

Mitoxantrone Hydrochloride

IV over 1 hour on days 2-4

Etoposide

Given IV over 1 hour on days 1-5

Gilteritinib

PO on days 8-28 (21 doses)

Primary outcome measure

  • Minimal residual disease (MRD)-negativity rate [ Time Frame: At day 29 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 ]

Central Contacts and Locations

Central contacts

Hiroto Inaba, MD, PhD

888-226-4343referralinfo@stjude.org

Locations

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Hiroto Inaba, MD, PhD

888-226-4343referralinfo@stjude.org

Principal Investigator:

Hiroto Inaba, MD, PhD

More Information

Sponsor

St. Jude Children's Research Hospital

Last update posted

Sep 17, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-18. This information was provided to ClinicalTrials.gov by St. Jude Children's Research Hospital on 2026-09-17.