Study Evaluating Safety and Efficacy of UCART123v1.2 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Phase 1Active Not RecruitingNCT03190278

Cellectis S.A.29 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Relapsed/Refractory Acute Myeloid Leukemia

Interventions

UCART123v1.2

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (8)

University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center

San Francisco, California, United States

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, United States

Northwestern University

Chicago, Illinois, United States

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Roswell Park Cancer Institute

Buffalo, New York, United States

Weill Medical College of Cornell University

New York, New York, United States

University of Pennsylvania - Abramson Cancer Center

Philadelphia, Pennsylvania, United States

MD Anderson Cancer Center

Houston, Texas, United States

Outcomes

Primary Outcomes

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

Time frame: 24 Months

Dose escalation and expansion part: Occurrence of DLTs

Time frame: Up to Day 28 post last UCART123v1.2 infusion

Secondary Outcomes

Investigators assessed overall response rate according to the European Leukemia Net (ELN) Response Criteria

Time frame: At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24

Duration of Response

Time frame: From the date of the initial response to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24

Progression Free Survival

Time frame: From the first day of study treatment to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24

Overall Survival

Time frame: From the first day of study treatment to the date of death from any cause, assessed up to Month 24

Pharmacokinetic (PK) Analysis: Standard PK Analysis will be completed to obtain Maximum plasma concentration (Cmax)