CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)
Recruiting now · Phase 1
Conditions studied: AML/MDS, B-ALL, T-ALL, BPDCN
In brief
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. * 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
Key facts
- Study ID
- NCT04318678
- Run by
- St. Jude Children's Research Hospital
- People needed
- 108
- Starts
- 2020-07-29
- Expected to finish
- 2030-07-29
- Last updated by the study team
- 2026-05-19
Who can join
Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- 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.
- Inclusion Criteria for Treatment:
- 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
Where it is running
- St Jude Children's Research Hospital — Memphis, Tennessee, United States (enrolling)
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (enrolling)
Full record on ClinicalTrials.gov
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