Recruiting
Phase 1

CD83 CAR T Cells

Sponsor:

Roswell Park Cancer Institute

Code:

NCT06871410

Conditions

Recurrent Acute Myeloid Leukemia

Refractory Acute Myeloid Leukemia

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Autologous Anti-CD83 CAR T-cells

Biospecimen Collection

Chest Radiography

Computed Tomography

Cyclophosphamide

Study Details

Brief summary:

This phase I trial tests the safety, side effects, and best dose of genetically engineered cells (CD83 chimeric antigen receptor \[CAR\] T cells) in treating patients with acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or has not responded to previous treatment (refractory). CD83 is a protein that is found on AML blasts. Blasts are abnormal immature white blood cells that can multiply uncontrollably: filling up the bone marrow and preventing the production of other cells important for survival. CD83 CAR T cells represent a new cell therapy to eliminate AML blasts, while avoiding the risk for graft versus host disease (GVHD) after stem cell transplant to replace bone marrow or, tumor toxicity like myeloid aplasia where the body's own immune system causes damage to the bone marrow stem cells. Therefore, human CD83 CAR T cells are a promising cell-based approach to preventing two critical complications of stem-cell transplant - GVHD and relapse. Giving CD83 CAR T cells may be safe, tolerable, and/or effective in treating patients with relapsed or refractory AML.

Conditions

Recurrent Acute Myeloid Leukemia

Refractory Acute Myeloid Leukemia

Study ID

NCT06871410

Start date

Feb 2, 2026

Status verified date

Aug, 2026

Completion date

Apr 1, 2028

Anticipated

Primary completion date

Apr 1, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 or any treatments other than hydroxyurea, single agent cytarbine,hypomethylating agents with or without ventoclax 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.

Study Design

Enrollment

26 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (fludarabine, cyclophosphamide, CD83 CAR T-cells)

Patients undergo leukapheresis to obtain PBMCs for CD83 CAR T-cell product manufacturing on day -21 and may receive hydroxyurea at the discretion of the treating physician on study. Patients then receive fludarabine IV over 30 minutes and cyclophosphamide IV over 2 hours on days -5 to -3 in the absence of disease progression or unacceptable toxicity. Patients then receive CD83 CAR T-cells IV over 15 minutes on day 0. Patients also undergo ECHO and chest x-ray during screening, blood sample collection throughout the study, and CT and/or PET, as well as lumbar puncture as clinically indicated. In addition, patients may undergo bone marrow aspiration throughout the study.

Interventions

Autologous Anti-CD83 CAR T-cells

Given IV

Biospecimen Collection

Undergo bone marrow aspiration blood sample collection

Chest Radiography

Undergo chest x-ray

Computed Tomography

Undergo CT

Cyclophosphamide

Given IV

Echocardiography

Undergo ECHO

Fludarabine Phosphate

Given IV

Hydroxyurea

Given hydroxyurea

Leukapheresis

Undergo leukapheresis

Lumbar Puncture

Undergo lumbar puncture

Positron Emission Tomography

Undergo PET

Questionnaire Administration

Ancillary studies

Primary outcome measure

  • Incidence of dose-limiting toxicity (DLT) [ Time Frame: Up to 28 days ]

Central Contacts and Locations

Locations

Roswell Park Cancer Institute

Recruiting

Buffalo, New York, United States, 14263

Contacts

Ask RPCI

800-767-9355askrpci@roswellpark.org

Principal Investigator:

Shernan G. Holtan

More Information

Sponsor

Roswell Park Cancer Institute

Last update posted

Aug 17, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Roswell Park Cancer Institute on 2026-08-17.