Genetically Engineered Cells (CD83 CAR T Cells) for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

Launched by ROSWELL PARK CANCER INSTITUTE · Mar 10, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for patients with acute myeloid leukemia (AML) that has either come back after treatment or did not respond to previous therapies. Researchers are testing a type of genetically engineered immune cell called CD83 CAR T cells. These cells are designed to target and eliminate the cancerous cells in the blood while minimizing serious complications that can occur after stem cell transplants, such as graft versus host disease. This trial aims to find out how safe the treatment is, what side effects may occur, and the best dose to use.

To be eligible for this trial, participants must be at least 18 years old and have a specific type of AML that meets certain criteria. They should also be in reasonably good health, with a life expectancy of at least 12 weeks. Participants will need to pass some health checks and will have to agree to use birth control if they are capable of becoming pregnant. If you join the trial, you will be closely monitored throughout the treatment process to ensure your safety and well-being. This is an important step in exploring new options for treating AML and could offer hope for patients who have limited choices left.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Age ≥ 18 years old.
• • Karnofsky performance status score ≥ 70%.
• • Relapsed or refractory AML based upon ELN 2022 criteria.
• • Creatinine clearance: ≥ 40 mL/min (Cockroft-Gault).
• • Total bilirubin: ≤ 2mg/dL except for patients with Gilbert's syndrome, hemolysis, or related to disease.
• • Aspartate aminotransferase (AST) and alanine transaminase (ALT) \< 3.0 x upper limit of normal (ULN).
• • Left ventricular (LV) ejection fraction: \> 45% and be free of symptomatic congestive heart failure or uncontrolled arrhythmia.
• • Oxygen (O2) saturation: ≥ 92% on room air without needs for supplemental O2.
• • Absolute lymphocyte count: ≥ 0.2 x 10\^9/L, HCT of ≥ 27% and platelets of ≥ 20 x 10\^9/L. Transfusion support is allowed to meet HCT and platelet parameters prior to apheresis.
• • Life expectancy ≥12 weeks from the time of enrollment, per clinical judgment.
• • Negative serum pregnancy test in females of child-bearing potential (FOCBP). FOCBP is defined as any female who has experienced menarche and who has not undergone successful surgical sterilization or who is not postmenopausal.
• • If history of allogeneic HCT, must have completed transplant at least 3 months prior, be off immunosuppression, including ruxolitinib, at least 2 weeks prior to apheresis, and have no evidence of GVHD requiring treatment at enrollment.
• • Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry and for 12 months following duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
• • Participants must be considered preliminarily eligible for an allogeneic hematopoietic cell transplantation, with potential donors identified per a transplant and cellular therapy consult at Roswell Park Comprehensive Cancer Center.
• • Participant must understand the investigational nature of this study and sign an Independent Ethics Committee/Institutional Review Board approved written informed consent form prior to receiving any study related procedure.
• Exclusion Criteria:
• • Concomitant systemic glucocorticoid use at a dose equivalent to \> 10 mg daily prednisone at the time of apheresis and/or within 4 weeks of CD83 CAR T infusion for any reasons other than GVHD.
• • Diagnosis of acute promyelocytic leukemia (APL; AML M3 by French-American-British \[FAB\] classification).
• • Active central nervous system (CNS) leukemia; patients with history of CNS leukemia in complete response (CR) are eligible.
• • Patients enrolled in another investigational therapy protocol for their disease within 14 days or 5 half-lives prior to leukapheresis, whichever is shorter.
• • Patients requiring agents any treatments other than hydroxyurea, hypomethylating agents, and/or targeted agents (i.e., FLT3, IDH2 or IDH1 inhibitors) to control blast counts within 14 days or 5 half-lives (whichever is shorter) prior to lymphodepletion.
• • Ongoing uncontrolled serious infection, pulmonary disease or psycho/social concerns.
• • HIV seropositivity or active hepatitis B or C infection within (defined by positive polymerase chain reaction \[PCR\]) 4 weeks of enrollment.
• • Other active malignancy within 2 years of study entry, except for basal cell cancer of skin, cervical cancer treated surgically with curative intent or localized prostate cancer managed with observational approach.
• • Active grade II-IV acute GVHD in patients with relapsed AML after HCT requiring treatment.
• • Prior solid organ transplant.
• • Active autoimmune disease requiring immunosuppressive therapy.
• • Pregnant or nursing female participants.
• • Unwilling or unable to follow protocol requirements.
• • Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug.