Phase I/II Trial to Determine Safety and Efficacy of non-Viral Allogeneic Dual CD123/33-Chimeric Antigen Receptor CIK (CARCIK-CD123/33) Cells in Adult and Pediatric Patients with Relapse/Refractory Acute Myeloid Leukemia (AML) and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Condition studied

Acute myeloid leukemia and Blastic plasmacytoid dendritic cell neoplasm

Investigational medicinal product(s)

Eligibility

Primary endpoint

Rate of enrolled patients that eventually receive CARCIK-CD123/33 infusion, Rate of dose Limiting toxicity (DLT) as per investigator assessment during the first 28 days after CARCIK-CD123/33 infusion, Incidence of Adverse event (AE) and laboratory abnormalities continuously assessed throughout the study and reported at each scheduled visit

Endpoint detail

Investigator-assessed overall response rate (CR+CRh+CRi+MLFS+PR) at 28 days after the first CARCIK-CD123/33 infusion, Persistence and kinetics of CARCIK-CD123/33 cells in terms of absolute number of cells using flowcytometry and vector copy number (VCN) by polymerase chain reaction (PCR), in target tissues, Persistence and kinetics of peripheral blood lymphoid chimerism, Investigator assessed best objective response (CR+CRh+CRi+MLFS+PR) after CARCIK-CD123/33 infusion, according to the defined criteria above, DOR, calculated as time from first documentation of a response to relapse, new anticancer therapies, progression or death from any cause, DFS, defined as time from first documentation of a CR to relapse, new anticancer therapies or death from any cause, PFS, defined as the time from CARCIK-CD123/33 infusion until leukemia progression, new anticancer therapies or leukemia-related death, OS, calculated as time from CARCIK-CD123/33 infusion to death from any cause