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OncoMatch/Clinical Trials/NCT04318678

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

Is NCT04318678 recruiting? Yes, currently enrolling (May 2026). This Phase 1 trial studies multiple treatments for aml/mds.

Phase 1RecruitingSt. Jude Children's Research HospitalNCT04318678Data as of May 2026

Treatment: Cyclophosphamide · Fludarabine · Mesna · Rituximab · CD123-CAR TThe 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. * To determine the safety of an intravenous infusion of escalating doses of donor derived, CD123-CAR T cells in patients (≤21 years) with recurrent/refractory CD123+ disease (AML/MDS, B-ALL, T-ALL, BPDCN or MPAL) 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 * To compare in vivo properties of donor-derived versus autologous CD123- CAR T cells

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Extracted eligibility criteria

Cancer type

Acute Myeloid Leukemia

Acute Lymphoblastic Leukemia

Biomarker criteria

Required: IL3RA expression (CD123 positive)

Relapsed/refractory CD123+ disease defined as follows:

Required: CD19 negative/dim (negative/dim)

CD19 negative/dim or patients otherwise ineligible for CD19 directed therapies

Prior therapy

Must have received: induction chemotherapy — AML/MDS refractory: not achieving CR after 2 cycles

Patients not achieving a CR after 2 cycles of induction chemotherapy

Must have received: allogeneic HSCT — B-cell ALL: relapse after allogeneic HSCT

Patients with relapse after allogeneic HSCT

Must have received: front-line therapy — BPDCN: relapsed/refractory disease that has failed front-line therapy

BPDCN • Relapsed/refractory disease that has failed front-line therapy

Cannot have received: donor lymphocyte infusion

Exception: within the 28 days prior to apheresis or planned infusion

have not received a donor lymphocyte infusion (DLI) within the 28 days prior to apheresis/planned infusion

Cannot have received: systemic steroids

Exception: exceeding the equivalent of 0.5 mg/kg/day of methylprednisolone, in the 7 days prior to CD123-CAR T-cell infusion

Receiving systemic steroids therapy exceeding the equivalent of 0.5 mg/kg/day of methylprednisolone, in the 7 days prior to CD123-CAR T-cell infusion

Cannot have received: systemic therapy

Exception: in the 14 days prior to CD123-CAR T-cell infusion, which will interfere with the activity of the CD123-CAR T cells in vivo (in the opinion of the study PI(s))

Receiving systemic therapy in the 14 days prior to CD123-CAR T-cell infusion, which will interfere with the activity of the CD123-CAR T cells in vivo (in the opinion of the study PI(s))

Cannot have received: rituximab (rituximab)

Exception: in the 30 days prior to CD123-CAR T cell infusion

Receiving rituximab therapy in the 30 days prior to CD123-CAR T cell infusion

Cannot have received: intrathecal chemotherapy

Exception: in the 7 days prior to CD123-CAR T cell infusion

Receiving intrathecal chemotherapy in the 7 days prior to CD123-CAR T cell infusion

Lab requirements

Kidney function

creatinine clearance or radioisotope GFR ≥50 ml/min/1.73m2 (GFR ≥40 ml/min/1.73m2 if < 2 years of age)

Liver function

Total Bilirubin ≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome; ALT or AST ≤5 times the upper limit of normal for age

Cardiac function

left ventricular ejection fraction >40%, OR shortening fraction ≥25%; EKG without evidence of clinically significant arrhythmia

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

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • St Jude Children's Research Hospital · Memphis, Tennessee
  • St. Jude Children's Research Hospital · Memphis, Tennessee

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