CD123-Directed Autologous T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)

Description

The CD123-CAR T-cell therapy is a new treatment that is being investigated for treatment of AML/myelodysplastic syndrome (MDS), T- or B- acute lymphoblastic leukemia (ALL) or blastic plasmacytoid dendritic cell neoplasia (BPDCN). The purpose of this study is to find the maximum (highest) dose of CD123-CAR T cells that is safe to give to these patients. This would include studying the side effects of the chemotherapy, as well as the CD123-CAR T-cell product on the recipient's body, disease and overall survival. Primary Objective To determine the safety of one intravenous infusion of escalating doses of autologous, CD123-CAR T cells in patients (≤21 years) with recurrent/refractory CD123+ disease (AML/MDS, B-ALL, T-ALL or BPDCN) after lymphodepleting chemotherapy. Secondary Objectives To evaluate the antileukemia activity of CD123-CAR T cells. Exploratory Objectives * To assess the immunophenotype, clonal structure and endogenous repertoire of CD123-CAR T cells and unmodified T cells * To characterize the cytokine profile in the peripheral blood and CSF after treatment with CD123-CAR T cells * To characterize tumor cells post CD123-CAR T-cell therapy

Conditions

AML/MDS, B-ALL, T-ALL, BPDCN

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Study Overview

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Study Details

Study overview

The CD123-CAR T-cell therapy is a new treatment that is being investigated for treatment of AML/myelodysplastic syndrome (MDS), T- or B- acute lymphoblastic leukemia (ALL) or blastic plasmacytoid dendritic cell neoplasia (BPDCN). The purpose of this study is to find the maximum (highest) dose of CD123-CAR T cells that is safe to give to these patients. This would include studying the side effects of the chemotherapy, as well as the CD123-CAR T-cell product on the recipient's body, disease and overall survival. Primary Objective To determine the safety of one intravenous infusion of escalating doses of autologous, CD123-CAR T cells in patients (≤21 years) with recurrent/refractory CD123+ disease (AML/MDS, B-ALL, T-ALL or BPDCN) after lymphodepleting chemotherapy. Secondary Objectives To evaluate the antileukemia activity of CD123-CAR T cells. Exploratory Objectives * To assess the immunophenotype, clonal structure and endogenous repertoire of CD123-CAR T cells and unmodified T cells * To characterize the cytokine profile in the peripheral blood and CSF after treatment with CD123-CAR T cells * To characterize tumor cells post CD123-CAR T-cell therapy

CD123-Directed Autologous T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)

CD123-Directed Autologous T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)

Condition
AML/MDS
Intervention / Treatment

-

Contacts and Locations

Memphis

St Jude Children's Research Hospital, Memphis, Tennessee, United States, 38105

Memphis

St. Jude Children's Research Hospital, Memphis, Tennessee, United States, 38105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Known primary immunodeficiency
  • * History of HIV infection
  • * Severe intercurrent uncontrolled bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection)
  • * History of hypersensitivity reactions to murine protein-containing products
  • * Patients with acute promyelocytic leukemia (APL, t (15;17))
  • * Known contraindication to the protocol defined lymphodepleting chemotherapy regimen of fludarabine/cyclophosphamide.
  • * Age≤21 years old
  • * Detectable disease that is CD123+ (at least MRD+ disease)
  • * Estimated life expectancy of \>8 weeks
  • * Karnofsky or Lansky (age-dependent) performance score≥50
  • * Patients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to planned infusion
  • * Patient must have an identified, suitable HCT donor
  • * Adequate cardiac function defined as left ventricular ejection fraction \>40%, OR shortening fraction ≥25%
  • * EKG without evidence of clinically significant arrhythmia
  • * Adequate renal function defined as creatinine clearance or radioisotope GFR ≥50 ml/min/1.73m2 (GFR ≥40 ml/min/1.73m2 if \< 2 years of age)
  • * Adequate pulmonary function defined as forced vital capacity (FVC)≥50% of predicted value; or pulse oximetry≥92% on room air if patient is unable to perform pulmonary function testing
  • * Total Bilirubin≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome
  • * Alanine aminotransferase (ALT) OR aspartate aminotransferase (AST) ≤5 times the upper limit of normal for age
  • * Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy
  • * For females of child-bearing age
  • * Not lactating with intent to breastfeed
  • * Not pregnant with negative serum pregnancy test within 7 days prior to enrollment
  • * If sexually active, agreement to use birth control until 3 months after T- cell infusion. Male partners should use a condom.
  • * Available autologous transduced T-cell product that has met GMP release criteria

Ages Eligible for Study

to 21 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

St. Jude Children's Research Hospital,

Swati Naik, MD, PRINCIPAL_INVESTIGATOR, St. Jude Children's Research Hospital

Paulina Velasquez, MD, PRINCIPAL_INVESTIGATOR, St. Jude Children's Research Hospital

Study Record Dates

2026-05-01