NCT04318678

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

Recruiting now Phase 1 Last updated 30 March 2026

What this trial tests

Phase 1 trial testing CD123-CAR T in AML/MDS in 108 participants. Currently enrolling.

Timeline

29 July 2020

Primary endpoint
29 July 2029

29 July 2030

Quick facts

Lead sponsor	St. Jude Children's Research Hospital
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	108
Start date	29 July 2020
Primary completion	29 July 2029
Estimated completion	29 July 2030
Sites	2 locations across United States

Drugs / interventions tested

• CD123-CAR T
• Cyclophosphamide (cyclophosphamide) — full drug profile →
• Fludarabine (FLUDARABINE) — full drug profile →
• Mesna (MESNA) — full drug profile →
• Rituximab — full drug profile →

Conditions studied

• AML/MDS — all drugs for AML/MDS →
• B-ALL — all drugs for B-ALL →
• T-ALL — all drugs for T-ALL →
• BPDCN — all drugs for BPDCN →

Sponsor

St. Jude Children's Research Hospital

Who can join

Under 21, any sex, with AML/MDS or B-ALL. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. CAR-T cell therapy for cancer: current challenges and future directions.
   Zugasti I, Espinosa-Aroca L, Fidyt K, Mulens-Arias V, et al · · 2025 · cited 81× · PMID 40610404 · DOI 10.1038/s41392-025-02269-w
2. Acute Myeloid Leukemia in Children: Emerging Paradigms in Genetics and New Approaches to Therapy.
   Conneely SE, Stevens AM. · · 2021 · cited 55× · PMID 33439382 · DOI 10.1007/s11912-020-01009-3
3. Efficacy, Safety, and Challenges of CAR T-Cells in the Treatment of Solid Tumors.
   Chen Q, Lu L, Ma W. · · 2022 · cited 44× · PMID 36497465 · DOI 10.3390/cancers14235983
4. Blastic plasmacytoid dendritic cell neoplasm: a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies.
   Cuglievan B, Connors J, He J, Khazal S, et al · · 2023 · cited 41× · PMID 37452102 · DOI 10.1038/s41375-023-01968-z
5. Recent Advances in Immune-Based Therapies for Acute Myeloid Leukemia.
   Restelli C, Ruella M, Paruzzo L, Tarella C, et al · · 2024 · cited 36× · PMID 38904305 · DOI 10.1158/2643-3230.bcd-23-0202
6. Challenges and Advances in Chimeric Antigen Receptor Therapy for Acute Myeloid Leukemia.
   Marvin-Peek J, Savani BN, Olalekan OO, Dholaria B. · · 2022 · cited 30× · PMID 35158765 · DOI 10.3390/cancers14030497
7. Novel Therapeutic Targets in Acute Myeloid Leukemia (AML).
   Wysota M, Konopleva M, Mitchell S. · · 2024 · cited 29× · PMID 38502417 · DOI 10.1007/s11912-024-01503-y
8. CAR-T therapy: Prospects in targeting cancer stem cells.
   Cui X, Liu R, Duan L, Cao D, et al · · 2021 · cited 29× · PMID 34585512 · DOI 10.1111/jcmm.16939