NCT06317662 · National Cancer Institute (NCI)
Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia
What this study is about
This phase II trial tests the addition of venetoclax and/or blinatumomab to usual chemotherapy for treating infants with newly diagnosed acute lymphoblastic leukemia (ALL) with a KMT2A gene rearrangement (KMT2A-rearranged \[R\]) or without a KMT2A gene rearrangement (KMT2A-germline \[G\]). Venetoclax is in a class of medications called B-cell lymphoma-2 (Bcl-2) inhibitors.
View original scientific description
This phase II trial tests the addition of venetoclax and/or blinatumomab to usual chemotherapy for treating infants with newly diagnosed acute lymphoblastic leukemia (ALL) with a KMT2A gene rearrangement (KMT2A-rearranged \[R\]) or without a KMT2A gene rearrangement (KMT2A-germline \[G\]). Venetoclax is in a class of medications called B-cell lymphoma-2 (Bcl-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival.
Interventions
DRUG
Asparaginase Erwinia chrysanthemi
Given recombinant crisantaspase IM or crisantaspase IM or IV
PROCEDURE
Biospecimen Collection
Undergo collection of blood samples
BIOLOGICAL
Blinatumomab
Given IV
PROCEDURE
Bone Marrow Aspiration
Undergo bone marrow aspiration
DRUG
Calaspargase Pegol
Given IV
PROCEDURE
Computed Tomography
Undergo CT
DRUG
Cyclophosphamide
Given IV
DRUG
Cytarabine
Given IT or IV
DRUG
Daunorubicin
Given IV
DRUG
Dexamethasone
Given PO or NG or IV
DRUG
Doxorubicin
Given IV
PROCEDURE
Echocardiography Test
Undergo ECHO
PROCEDURE
FDG-Positron Emission Tomography
Undergo FDG-PET
DRUG
Leucovorin
Given PO or NG or IV
DRUG
Levoleucovorin
Given IV
PROCEDURE
Lumbar Puncture
Undergo lumbar puncture
PROCEDURE
Magnetic Resonance Imaging
Undergo MRI
DRUG
Mercaptopurine
Given PO or NG
DRUG
Methotrexate
Given IT or IV or PO or NG
DRUG
Methylprednisolone
Given IV
PROCEDURE
Multigated Acquisition Scan
Undergo MUGA
DRUG
Prednisolone
Given PO or NG
DRUG
Prednisone
Given PO or NG
DRUG
Therapeutic Hydrocortisone
Given IT
DRUG
Thioguanine
Given PO or NG
DRUG
Venetoclax
Given PO or NG
DRUG
Vincristine
Given IV
Primary outcome measures
Incidence of dose-limiting toxicities (DLTs) (safety phase)
Time frame: For the duration of the induction + venetoclax cycle
For the safety phase, DLTs of the induction + venetoclax cycle of KMT2A-rearranged (R) patients will be assessed.
Incidence of DLTs (expansion phase)
Time frame: During the induction, consolidation, and MARMA cycles of Arm B
For the expansion phase, DLTs of Arm B will be assessed and monitored for the cycles that contain venetoclax (induction, consolidation, and MARMA cycles of Arm B).
Minimal residual disease (MRD)-negative remission rate
Time frame: At the end of induction
The end of induction MRD-negative remission rate will be compared between Arm A and Arm B. MRD negativity is defined as achievement of complete remission and MRD \< 0.01% by flow cytometry. The MRD-negative remission rate at the end of induction between Arm A and Arm B will be compared using a one-sided Z test of proportions with Type I error of 0.15.
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
- 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
Where
- Birmingham, Alabama
- Anchorage, Alaska
- Mesa, Arizona
- Little Rock, Arkansas
- Loma Linda, California
- Long Beach, California
- Oakland, California
- Orange, California
- Sacramento, California
- San Francisco, California
- Aurora, Colorado
- Hartford, Connecticut
And 88 more locations — see the full list below.
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 Jul 13, 2026 · Source of record for eligibility and locations