eligibility_summary
Adults 18–70 with CD123+ AML, ALL, BPDCN, or MDS‑IB2 lacking standard options. AML: refractory, ≥2nd relapse, or post‑allo‑HSCT relapse. ALL: post‑CAR19 R/R or CD19– relapse ineligible for TKI/inotuzumab. BPDCN: R/R ± prior allo‑SCT. MDS‑IB2: refractory/progressive on azacitidine, refractory to induction, or post‑transplant relapse. Requires suitable allo‑HSCT donor. Exclude: recent allo‑HSCT, severe infection/organ dysfunction, severe GVHD, pregnancy/breastfeeding, live vaccines, contraception required.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Intervention: CART123—an autologous, genetically modified chimeric antigen receptor (CAR) T-cell therapy (biologic, cell-based immunotherapy). Patients’ T cells are engineered ex vivo to express an anti-CD123 CAR with activation/costimulatory signaling domains, then infused once after lymphodepleting chemotherapy, dose escalated (BOIN) to determine MTD/MFD. Mechanism of action: Upon binding CD123, CAR T cells activate, proliferate, secrete cytokines, and kill target cells via perforin/granzyme pathways, independent of native TCR. Targets (cells/pathways): CD123 (IL-3 receptor alpha) expressed on malignant cells in AML, MDS-IB2, ALL, and BPDCN, also on plasmacytoid dendritic cells and some normal hematopoietic progenitors. The IL-3 receptor pathway component (CD123) is the molecular target. On-target myelosuppression risk underlies the requirement for HSCT eligibility.