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Phase I/II Study of CD33 CAR-NK Cells for Relapsed/Refractory AML

This Phase I/II study is investigating a new treatment for acute myeloid leukemia (AML) that has returned or hasn't responded to other treatments. The treatment uses special immune cells called CD33 CAR-NK cells, which are made from healthy donor cells and designed to target and destroy CD33+ AML cells. You would first receive chemotherapy (Fludarabine, Cytarabine, and Venetoclax), followed by one or two infusions of the CD33 CAR-NK cells. The first part of the study (Phase I) will determine the safest and most effective dose. The second part (Phase II) will then evaluate how well the treatment works at that dose. This study is for patients aged 1 to 39 years with relapsed or refractory CD33+ AML. The study aims to measure safety and how well the treatment works.

Study design
This is a Phase I/II interventional study planning to enroll 42 participants. It will first find the right dose and then expand to further study safety and how well the treatment works.
What's involved
You will undergo screening, receive chemotherapy, and then have one or two infusions of CD33 CAR-NK cells. Safety will be measured for 28 days after the last infusion, and how well the treatment works will be measured at Day 35.
Compensation
Not stated in the trial record.
Follow-up
Safety is measured for 28 days after the last CAR-NK cell infusion, and the treatment's effectiveness is measured at Day 35.

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NCT07026942

Phase I/II Clinical Trial of Universal Donor CD33 CAR Natural Killer Cells for AML

Not Yet Recruiting
PHASE1Ages 1–39InterventionalTreatment
Nationwide Children's Hospital
~42 participants
Updated 2025-11-12 on ClinicalTrials.gov
What's tested:Universal donor derived CD33 CAR-NK

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase I : Safety and recommended phase 2 dose
Measured over From the first CD33 CAR NK cell infusion until 28 days after the last CAR NK cell dose.
+1 more outcome measured

Conditions

Relapsed/Refractory AML

Explore related trials

Acute Myeloid LeukemiaCD33 Gene

Where it's being run

1 sites across 1 states
Ohio1

Study leadership

  • Margaret Lamb, MD · PRINCIPAL_INVESTIGATOR · Nationwide Children's Hospital

Who to contact

Clelie Peck
[email protected]
614-722-5634
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients with relapsed AML (patients in second or subsequent relapse, or any relapse after HSCT, are eligible).
Refractory AML defined as failure to achieve a complete response after 2 cycles of induction or reinduction chemotherapy, including persistent MRD positivity.
Patients with isolated CNS or extramedullary disease are eligible. Patients with CNS disease are excluded from the phase I dose escalation portion but are eligible for the phase II portion of the study. 2. 1-39.99 years of age (note: the first three subjects treated AND the first subject on each dose level must be ≥ 16 years of age) 3. Negative serum test to rule out pregnancy within 14 days prior to enrollment in females of childbearing potential
Renal function: Creatinine ≤ 2 times the institutional upper limit of normal for age OR creatinine clearance \> 60 ml/min/1.73m2 (measured by 24 hour- urine specimen or radioisotope GFR)
Liver function: Total bilirubin ≤ 2 mg/dl (unless Gilbert's syndrome), AST and ALT ≤ 5 times the upper limit of normal (unless related to leukemic involvement). Upper limit of normal should be determined by the institutional defined normal laboratory range.
Cardiac function: left ventricular ejection fraction ≥ 40% or shortening fraction ≥20%. May be eligible after cardiology clearance if qualitatively normal function or repeat measures are normal.
CNS: Patients with seizure disorder may be eligible if seizures are well controlled
Pulmonary function: baseline oxygen saturation \>92% on room air at rest 5. Due to the risk of hematopoietic toxicity from CD33 targeting, enrolled subjects must have an allogeneic HCT donor identified and be eligible and willing to undergo a subsequent HSCT in the event of aplasia. 6. All patients or their legal guardian must be able to understand and willing to sign a written informed consent document. 7. All patients must consent to enroll in a separate long term follow up study for cell and gene therapy

Exclusion

AML directed therapies in the 14 days prior to beginning treatment on this protocol (except for hydroxyurea) Note: There is no waiting period required for patients having received intrathecal cytarabine, methotrexate and/or hydrocortisone
Gemtuzumab or other CD33-targeted antibody within 42 days of enrollment
CNS radiation within 28 days of enrollment
DLI or adoptive cell therapy within 30 days of enrollment
Allogeneic SCT within 90 days of enrollment
Patients with CNS disease are excluded from the phase I portion of the study but are eligible for the phase II expansion phase.
Patients on immunosuppressive therapy
Patients must be off of systemic immunosuppressive therapy for at least 14 days prior to enrollment with no evidence of recurrent GVHD
Patients on hydrocortisone for treatment of adrenal insufficiency are permitted on study
Patients on corticosteroids ≤ 0.5mg/kg/day (prednisone equivalent) for any other indication are permitted on study 2. Uncontrolled, symptomatic, intercurrent illness or social situations that would limit compliance with study requirements or in the opinion of the site PI would pose an unacceptable risk to the subject 3. Patients who are breastfeeding 4. Patients with prior solid organ transplantation 5. Performance status: Karnofsky or Lansky Performance Scale (PS) \< 50 6. Uncontrolled infection, defined as an infection which has not resolved or does not show evidence of significant resolution after initiating appropriate therapy

What this trial measures

  • Phase I : Safety and recommended phase 2 doseFrom the first CD33 CAR NK cell infusion until 28 days after the last CAR NK cell dose.

    To determine the safety and recommended phase 2 dose of CD33 CAR-NK cells in patients with relapsed/refractory AML investigators will monitor the incidence and severity of adverse events and the rate of dose limiting toxicities.

  • Phase II: Efficacy of CD33 CAR NK cellsDay 35

    To estimate the efficacy of CD33 CAR-NK cells delivered at the RP2D with FLA-VEN chemotherapy, the investigators will determine the complete response rate.