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CD123 Redirected T Cells for AML in Pediatric Subjects

Clinical Trial: NCT04678336

For more details on this clinical trial, including contact information, please see this trial’s listing on

This is a Phase 1 study to determine the safety, manufacturing feasibility, and efficacy of CART123 cells following lymphodepleting chemotherapy in pediatric subjects with relapsed/refractory AML.

Subjects will receive CART123 cells via a single IV infusion at a dose of 2x10^6 CART123 cells/kg, following lymphodepleting chemotherapy. The total dose administered to each subject will be based on the subject's body weight obtained at the time of apheresis. The minimum acceptable dose for infusion is 1x10^5 CART123 cells/kg.

There will be a 28-day stagger between the first 3 subject infusions, such that the next subject may not receive treatment (lymphodepleting chemotherapy plus CART123 cells) until the previous subject has completed their Day 28 safety follow-up visit and a DLT assessment has been performed. Subsequent infusions will be staggered by a minimum of 14 days.

It is recommended that subjects with marrow aplasia at Day 28+/-5 undergo an allogeneic hematopoietic cell transplantation (alloHCT) as a rescue strategy. If required, this procedure will be performed as part of routine care, outside of the scope of this research study; however, subjects will continue to be followed onstudy. All subjects must, therefore, have a previously identified stem cell donor as part of their eligibility to participate in this study.

All subjects will be followed monthly for up to 6 months after the CART123 cell infusion (Day 0). Thereafter, subjects will be transitioned into LTFU for up to 15 years post infusion.

Not yet recruiting
Study Date: 
Mon, 12/21/2020 to Tue, 01/01/2036
Bone Marrow Disease(s): 
  • acute myeloid leukemia (AML)
  • pediatric
Experimental: Treatment Arm CART123 cells; cyclophosphamide; fludarabine Biological: CART123 cells; cyclophosphamide; fludarabine CART123 cells following lymphodepleting chemotherapy in pediatric patients with relapsed/refractory AML. Subjects will be treated with a single IV dose of CART123 cells on Day 0. Other Name: T Cells Containing Anti-CD123 Signaling Domains
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