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

Part of paid clinical trials in Memphis, Tennessee.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT04318678
Phase
PHASE1
Status
Recruiting
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Conditions

  • AML/MDS
  • B-ALL
  • BPDCN
  • T-ALL

Eligibility Criteria

Sex
ALL
Age
N/A - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • CD123-CAR T — DRUG
    To treat relapsed/refractory CD123+ AML/MDS, B-ALL, T-ALL or BPDCN patient population that needs new cancer-directed therapies.
  • Cyclophosphamide — DRUG
    Cyclophosphamide is a nitrogen mustard derivative. It acts as an alkylating agent that causes cross-linking of DNA strands by binding with nucleic acids and other intracellular structures, thus interfering with the normal function of DNA.
  • Fludarabine — DRUG
    Fludarabine phosphate is a synthetic purine nucleoside analog. It acts by inhibiting DNA polymerase, ribonucleotide reductase and DNA primase by competing with the physiologic substrate, deoxyadenosine triphosphate, resulting in inhibition of DNA synthesis
  • Mesna — DRUG
    Mesna is a synthetic sulfhydryl (thiol) compound. Mesna contains free sulfhydryl groups that interact chemically with urotoxic metabolites of oxaza-phosphorine derivatives such as cyclophosphamide and ifosfamide
  • Rituximab — DRUG
    Rituximab is a monoclonal antibody directed against the CD20 antigen on the surface of B-lymphocytes

Study Details

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 Dates

Start date
Jul 29, 2020
Status verified
May 2026
Primary completion
Jul 29, 2029
Completion
Jul 29, 2030

Study Design

Enrollment
108 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: ARM A CD123-CAR T cell therapy
    For patients who have not received an allogeneic transplant or for patients who have received allogeneic transplant and do not have a transplant donor available CD123-CAR T-cell dose and infusion Up to 4 Dose levels will be evaluated with a maximum dose of 2.5 x 10\^8 CAR+ T cells. If dose limiting toxicities (DLTs) are observed on Dose level 1 then the cell dose is de- escalated.
  • Experimental: ARM B CD123-CAR T cell therapy
    For patients who relapsed following allogeneic transplant and whose CAR T-cells will be manufactured from the previous transplant donor, when available. CD123-CAR T-cell dose and infusion Up to 4 Dose levels will be evaluated with a maximum dose of 2.5 x 10\^8 CAR+ T cells. If dose limiting toxicities (DLTs) are observed on Dose level 1 then the cell dose is de- escalated.

Primary Outcome Measure

Maximum tolerated dose of CD123-CAR T cells (CATCHAML) [ Time Frame: 4 weeks after CD123-CAR T-cell infusion ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
St Jude Children's Research HospitalMemphisTennessee38105
Swati Naik, MD
[email protected]
888-226-4343
Swati Naik, MD (PRINCIPAL_INVESTIGATOR)
St. Jude Children's Research HospitalMemphisTennessee38105
Paulina Velasquez, MD
[email protected]
888-226-4343
Paulina Velasquez, MD (PRINCIPAL_INVESTIGATOR)

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