CELL INTELLIGENCE · 肿瘤细胞治疗研究

Main Inclusion Criteria:

* Patients with relapsed or primary refractory AML (as defined in World Health Organization \[WHO\] criteria) with ≥5% bone marrow blasts
* Patients with CD123+ blast cells (verified by flow cytometry)
* Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of ≤1
* Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within screening period
* (Dose-escalation) Identified donor and transplant strategy prior to lymphodepletion (LD)
* Other criteria may apply

Main Exclusion Criteria:

* Patients with acute promyelocytic leukemia (APL) or central nervous system (CNS) Leukemia
* Previous investigation gene or cell therapy (including CAR)
* \> 1 prior allogeneic stem cell transplantations (SCTs)
* Prior treatment with rituximab or other anti-cluster of differentiation 20 (anti-CD20) therapy within 3 months
* Any known active or uncontrolled infection
* Other criteria may apply

主要终点：Incidence of adverse events (AE)/serious adverse events (SAE)/Dose Limiting Toxicities (DLT) [Safety and Tolerability] · Safety of UCART123v1.2 - Incidence, nature, and severity of AE and SAEs throughout the study · 24 Months；Dose escalation and expansion part: Occurrence of DLTs · Up to Day 28 post last UCART123v1.2 infusion
次要终点：Investigators assessed overall response rate according to the European Leukemia Net (ELN) Response Criteria；Duration of Response；Progression Free Survival；Overall Survival；Pharmacokinetic (PK) Analysis: Standard PK Analysis will be completed to obtain Maximum plasma concentration (Cmax)；Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain time to reach Cmax (Tmax)；Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain total area under curve from zero to infinity (AUC-infinity)；Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Rate

• Dose Escalation · EXPERIMENTAL · UCART123v1.2 tested at several dose levels with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART123v1.2 administered at the RP2D determined from the dose escalation phase

Phase I, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of Universal Chimeric Antigen Receptor T-cell (UCART) targeting the Cluster of Differentiation 123 (CD123) in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of Universal Chimeric Antigen Receptor T-cells targeting CD123 (UCART123v1.2) and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).