Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia
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
Lead SponsorCellectis S.A.
Indication/ConditionRelapsed/Refractory Acute Myeloid Leukemia
Phase I, first-in-human, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of UCART targeting CD123 in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of UCART123v1.2 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor
Several tested doses of UCART123v1.2 with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART123v1.2 administered at the selected dose determined from the dose escalation phase
Main Inclusion Criteria: Patients with relapsed or primary refractory AML (as defined in 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 APL or CNS Leukemia Previous investigation gene or cell therapy (including CAR) ≥ 2 prior allogeneic SCTs Prior treatment with rituximab or other anti-CD20 therapy within 3 months Any known active or uncontrolled infection Other criteria may apply