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OncoMatch/Clinical Trials/NCT04318678

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

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

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

Treatments studied

Immunotherapy

Rituximab

Chemotherapy

CyclophosphamideFludarabine

Other

MesnaCD123-CAR T

Cancer type

Acute Myeloid Leukemia

Myelodysplastic Syndrome

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

Demographics

Ages ≤ 21

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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See all sites on ClinicalTrials.gov →

Frequently asked questions

Is NCT04318678 currently recruiting?

Yes, this trial is currently recruiting patients.

Are there prior therapy exclusions?

Yes. Prior donor lymphocyte infusion, systemic steroids, systemic therapy disqualifies patients from enrollment.

Does this trial require IL3RA?

Yes, IL3RA expression is a required biomarker for enrollment.

Does this trial require CD19?

Yes, CD19 negative/dim is a required biomarker for enrollment.

Is there an age limit?

Yes. Patients must be 21 years or younger.

Could you qualify for this trial?

Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.

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