CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)
This study is testing a new treatment called CD123-CAR T cells for children and young adults (up to 21 years old) with certain types of blood cancers that have come back or haven't responded to previous treatments. These cancers include acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), acute lymphoblastic leukemia (ALL), or blastic plasmacytoid dendritic cell neoplasia (BPDCN), specifically if they are CD123 positive. The main goal is to find the highest safe dose of CD123-CAR T cells. Before receiving the CD123-CAR T cells, you will receive chemotherapy with Cyclophosphamide and Fludarabine, along with Mesna and potentially Rituximab. The study will look at side effects and how the treatment affects your cancer and overall health. The study plans to enroll 108 participants, but its current status is unclear.
- Study design
- This is an interventional study with an unclear phase. It involves two phases: a Collection and Manufacturing Phase and a Treatment Phase.
- What's involved
- Your blood cells will be collected and processed to create the CD123-CAR T cells. You will then receive chemotherapy for several days, followed by an infusion of the CD123-CAR T cells, and be monitored for side effects.
- Compensation
- Not stated in the trial record.
- Follow-up
- The maximum tolerated dose of CD123-CAR T cells will be measured at 4 weeks after the infusion.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT04318678
CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)
Recruiting
PHASE1Up to 21InterventionalTreatmentSt. Jude Children's Research HospitalInvestigator-initiated
~108 participants
Updated 2026-05-19 on ClinicalTrials.gov
What's tested:CD123-CAR TCyclophosphamideFludarabineMesnaRituximab
At a glance
Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Maximum tolerated dose of CD123-CAR T cells (CATCHAML)
Measured over 4 weeks after CD123-CAR T-cell infusion
Conditions
Where it's being run
2 sites across 1 statesTennessee2
Study leadership
- Swati Naik, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
- Paulina Velasquez, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age ≤21 years old
Relapsed/refractory CD123+ disease defined as follows:
Relapsed disease: Patients developing recurrent disease after a first complete remission (CR)
Refractory disease: Patients not achieving a CR after 2 cycles of induction chemotherapy
Relapsed disease that is CD123 positive and CD19 negative/dim or patients otherwise ineligible for CD19 directed therapies including
Patients in 2nd or greater relapse
Patients with relapse after allogeneic HSCT
Refractory disease that is CD123 positive and CD19 negative/dim or patients otherwise ineligible for CD19 directed therapies
Estimated life expectancy of \>12 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 apheresis
Patient must have an identified, suitable HCT donor
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
Meets eligibility criteria to undergo autologous apheresis, or have previously undergone autologous apheresis
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
Exclusion
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.
Known primary immunodeficiency
History of HIV infection
Severe intercurrent uncontrolled bacterial, viral or fungal infection
History of hypersensitivity reactions to murine protein-containing products
History of severe hypersensitivity reactions to cornstarch or hydroxyethyl starch.
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
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))
Receiving rituximab therapy in the 30 days prior to CD123-CAR T cell infusion. (This exclusion criterion is intended to prevent premature exposure of CD123-CAR T cells to rituximab, which would activate the safety switch and promote CAR T-cell apoptosis).
Receiving intrathecal chemotherapy in the 7 days prior to CD123-CAR T cell infusion.
Known contraindication to the protocol defined lymphodepleting chemotherapy regimen of fludarabine/cyclophosphamide.
Active CNS disease
What this trial measures
- Maximum tolerated dose of CD123-CAR T cells (CATCHAML)4 weeks after CD123-CAR T-cell infusion
A phase I design to determine the maximum tolerated dose (MTD) of autologous, CD123- CAR T cells. Four dose levels (3x10\^5/kg, 1x10\^6/kg, 3x10\^6/kg, and 1x10\^7/kg) will be evaluated.