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Combining venetoclax and blinatumomab with standard chemotherapy for infants with acute leukemia

A Phase 2 Study of Blinatumomab in Combination With Chemotherapy for Infants With Newly Diagnosed Acute Lymphoblastic Leukemia With Randomization of KMT2A-Rearranged Patients to Addition of Venetoclax

Phase 2 Interventional National Cancer Institute (NCI) · NCT06317662

This study is testing whether adding two new treatments, venetoclax and blinatumomab, to standard chemotherapy can help infants with acute leukemia do better and stay cancer-free longer.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment153 (estimated)
AgesN/A to 365 Days
SexAll
SponsorNational Cancer Institute (NCI) NIH
Drugs / interventionschemotherapy, methotrexate, PredniSONE, blinatumomab, chimeric antigen receptor, cyclophosphamide, doxorubicin
Locations108 sites (Birmingham, Alabama and 107 other locations)
Trial IDNCT06317662 on ClinicalTrials.gov

What this trial studies

This phase II trial evaluates the safety and effectiveness of adding venetoclax and/or blinatumomab to standard chemotherapy for infants diagnosed with acute lymphoblastic leukemia (ALL) that either has a KMT2A gene rearrangement or does not. Venetoclax is a Bcl-2 inhibitor that may help stop cancer cell growth, while blinatumomab is a monoclonal antibody that targets cancer cells. The study aims to determine if this combination improves remission rates and event-free survival compared to standard chemotherapy alone. It also assesses the pharmacokinetics of venetoclax in infants and explores the feasibility of treating different subtypes of leukemia.

Who should consider this trial

Good fit: Ideal candidates are infants aged 365 days or less with newly diagnosed KMT2A-rearranged or KMT2A-non-rearranged acute lymphoblastic leukemia.

Not a fit: Patients with Down Syndrome or those who have previously received cytotoxic chemotherapy for another malignancy may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could lead to improved treatment outcomes for infants with acute leukemia.

How similar studies have performed: Other studies have shown promise with similar approaches, but this specific combination is being evaluated for the first time in this population.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* All patients must be enrolled on APEC14B1 and consented to eligibility screening (part A) prior to treatment and enrollment on AALL2321
* Infants (aged 365 days or less) on the date of diagnosis are eligible; infants must be \> 36 weeks gestational age at the time of enrollment
* Patients must have newly diagnosed B-acute lymphoblastic leukemia (B-ALL, 2017 World Health Organization \[WHO\] classification), also termed B-precursor ALL, or acute leukemia of ambiguous lineage (ALAL), which includes mixed phenotype acute leukemia. For patients with ALAL, the immunophenotype of the leukemia must comprise at least 50% B lineage

  * Diagnostic immunophenotype: Leukemia cells must express CD19

Exclusion Criteria:

* Patients with Down Syndrome
* Patients with secondary B-ALL that developed after treatment of a prior malignancy with cytotoxic chemotherapy
* Patients must not have received any cytotoxic chemotherapy for either the current diagnosis of infant ALL or for any cancer diagnosis prior to the initiation of protocol therapy, with the exception of:

  * Steroid pretreatment:

    * PredniSONE, prednisoLONE, or methylPREDNISolone for ≤ 72 hours (3 days) in the 7 days prior to enrollment. The dose of predniSONE, prednisoLONE or methylPREDNISolone does not affect eligibility
    * Inhaled and topical steroids are not considered pretreatment
    * Note: Pretreatment with dexamethasone in the 28 days prior to initiation of protocol therapy is not allowed with the exception of a single dose of dexamethasone used during or within 6 hours prior to or after sedation to prevent or treat airway edema. However, prior exposure to ANY steroids that occurred \> 28 days before enrollment does not affect eligibility
  * Intrathecal cytarabine or methotrexate:

    * An intrathecal dose of cytarabine or methotrexate in the 7 days prior to enrollment does not affect eligibility
    * Note: The preference is to defer the diagnostic lumbar puncture with intrathecal chemotherapy to day 1 of induction to allow for cytoreduction of circulating blasts and decrease the potential for central nervous system (CNS) contamination due to a traumatic tap. If done prior to day 1 of induction, these results will be used to determine CNS status
  * Hydroxyurea:

    * Pretreatment with ≤ 72 hours (3 days) of hydroxyurea in the 7 days prior to enrollment does not affect eligibility
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA) and National Cancer Institute (NCI) requirements for human studies must be met

Where this trial is running

Birmingham, Alabama and 107 other locations

+58 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Leukemia of Ambiguous LineageB Acute Lymphoblastic Leukemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.