CD64 CAR T Cell Therapy in Adults With Relapsed and/or Refractory AML

Recruiting now · Phase 1

Conditions studied: Refractory Acute Myeloid Leukemia (AML), Relapsed Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, AML (Acute Myeloid Leukemia)

In brief

This is a Phase 1, open label, dose-escalation study to evaluate the safety, expansion, persistence, and preliminary clinical activity of lentivirally transduced autologous T cells expressing anti-CD64 chimeric antigen receptors (CAR) expressing tandem CD3ζ and 4-1BB (CD3ζ/4-1BB) costimulatory domains in subjects with refractory or relapsed (R/R) acute myeloid leukemia (AML). This CAR T cell product will be referred to as "CD64 CAR T" which is CD64 directed, autologous, genetically modified CAR T cells. The primary objective of the study is to identify the safety profile and maximum tolerated dose (MTD) of CD64 CAR T in subjects with R/R AML as determined by the defined DLTs using a standard Bayesian Optimal Interval (BOIN) design.

Key facts

Study ID

NCT07347418

Run by

University of Colorado, Denver

People needed

23

Starts

2026-06-01

Expected to finish

2032-06-01

Last updated by the study team

2026-06-29

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• ≥ 18 years of age.
• Subjects must have one of the following diagnoses per the International Consensus Classification (ICC) 2022 criteria:
• a. Acute Myeloid Leukemia (AML).
• Refractory OR relapsed AML:
• a. Refractory disease i. ≥5% blasts in the bone marrow or peripheral blood by morphology, flow cytometry, or immunohistochemistry after a minimum of 1 cycle of a hypomethylating agent (HMA) and venetoclax (Ven) combination (Ven/HMA) b. Relapsed disease i. Recurrence of ≥ 5% blasts in the bone marrow or peripheral blood by morphology, flow cytometry or immunohistochemistry.
• Subjects must have received at least one prior line of therapy, including at least one line of therapy containing Ven.
• Documentation of CD64 expression on ≥70% of myeloid blasts by flow cytometry after the most recent relapse, as determined by standardized and validated multiparameter flow cytometry assay (Hematologics, Inc., Seattle, WA).
• Total white blood cell (WBC) count ≤ 25 x 10(to the 9th)/L prior to apheresis. Hydroxyurea is permitted to achieve this
• Absolute lymphocyte count (ALC) ≥ 200/µL prior to apheresis OR ALC < 200 µL with concurrent lymphocyte subset analysis (CD3, CD4, and CD8 counts) confirming an absolute CD3 count ≥ 150/µL.
• Confirmed availability of cells for a rescue stem cell transplant AND subject must be deemed an appropriate candidate for such therapy per institutional standards.
• Subjects who have undergone prior allogeneic stem cell transplant must be ≥ 6 months out from transplant and be off systemic immunosuppression for at least 1 month at the time of enrollment with no evidence of active graft versus host disease.
• Adequate organ function, defined as:
• Creatinine clearance ≥ 30 mL/min, based on the CKD-EPI Creatinine Equation (2021).
• AST/ALT ≤ 5x upper limit of the normal range, unless considered to be due to leukemic involvement.
• Bilirubin ≤ 3x upper limit of the normal range, unless the subject has Gilbert's Syndrome or considered to be due to leukemic involvement.
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygen > 92% on room air, unless considered to be due to leukemic involvement.
• Left Ventricle Ejection Fraction (LVEF) ≥ 40% confirmed by ECHO/MUGA.
• ECOG performance status 0, 1, or 2.
• Signed informed consent form.
• Subjects of reproductive potential must agree to use acceptable birth control methods, as described in the protocol main text.
• Willing to participate in the long-term follow-up protocol that is required if CAR T cell therapy is administered.

You may not qualify if…

• Subjects with Acute Promyelocytic Leukemia (APL) with t(15;17)
• Receipt of previous chemotherapy for AML, as follows:
• a. Prior to apheresis, the following washout periods apply: i. Hydroxyurea: 1 day ii. Hypomethylating agent and/or venetoclax: 7 days iii. Small molecule targeted therapy (including tyrosine kinase inhibitors): 3 half-lives or 7 days, whichever is shorter.
• iv. Immune checkpoint inhibitors or other immunological agents: 5 half-lives or 28 days, whichever is shorter.
• v. Investigational products: 5 half-lives or 28 days, whichever is shorter. vi. Any other systemic chemotherapy: 14 days vii. Allogeneic stem cell transplantation: 180 days viii. Donor lymphocyte infusion (DLI): 60 days ix. Craniospinal or total body radiation: 42 days b. After apheresis and prior to lymphodepletion, no treatment for AML is permitted, with the exception of bridging hydroxyurea with a washout period of 1 day prior to the start of the lymphodepletion regimen.
• Concurrent use of systemic steroids or immunosuppressant medications. Recent or current use of inhaled steroids or physiologic replacement with hydrocortisone is not exclusionary.
• Previous treatment with investigational gene or cell therapy (including CAR therapy).
• Signs or symptoms indicative of CNS leukemia involvement. A CNS evaluation should be performed if CNS involvement is suspected to rule out CNS leukemia involvement.
• Pregnant or lactating (nursing) women.
• Known HIV infection or active Hepatitis B or Hepatitis C infection.
• Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).
• Class III/IV cardiovascular disability according to the New York Heart Association Classification.
• Subjects with a known history or prior diagnosis of optic neuritis or other immunologic or inflammatory disease affecting the central nervous system, and unrelated to leukemia or previous leukemia treatment.
• Subjects with cardiac arrhythmia, or arrhythmias that are not stable with medical management, within 2 weeks of the Screening/Enrollment visit.
• Any uncontrolled active medical disorder that would preclude participation as outlined.
• Evidence of another uncontrolled malignancy.
• Apheresis Eligibility To proceed with apheresis, enrolled participants must continue to meet all inclusion criteria within no more than 21 days prior to apheresis, unless otherwise specified.
• Note: Disease evaluation (bone marrow aspirate and biopsy) to meet inclusion criteria must be completed within 30 days prior to enrollment.
• Lymphodepleting Chemotherapy Eligibility To proceed with lymphodepleting chemotherapy, enrolled participants must have specific assessments completed and continue to meet all inclusion criteria within 72 hours of initiation of lymphodepletion
• CD64 CAR T Infusion Eligibility
• Participants must meet the following criteria in order for cells to be infused (based on labs obtained within 24 hours of cell infusion):
• CD64 CAR T must have met manufacturing criteria (unless prospectively approved by IND Sponsor, Gates Institute Medical Lead, and FDA)
• Confirmation that the site has Anakinra and Ruxolitinib in stock and available (should IEC-HS treatment be required).
• Performance status determination (ECOG must be 0, 1 or 2).
• Participant remains clinically stable without evidence of vital sign instability including the lack of supportive vasoactive drugs or intensive care support.

Where it is running

• University of Colorado Hospital — Aurora, Colorado, United States (enrolling)

Full record on ClinicalTrials.gov

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