Phase I, open label dose-escalation and dose-expansion study to evaluate the safety, expansion, persistence and clinical activity of UCART123 (allogeneic engineered T-cells expressing anti-CD123 chimeric antigen receptor), administered in patients with Relapsed/Refractory Acute Myeloid Leukemia

Protocol # :
Relapsed/Refractory Acute Myeloid Leukemia
Disease Sites
Myeloid and Monocytic Leukemia
Principal Investigator
DeAngelo, Daniel, J

Trial Description

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).

Eligibility Requirements

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

- Other criteria may apply

Main Exclusion Criteria:

- Patients with acute promyelocytic leukemia (APL) or central nervous system (CNS)

- 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