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

NCT03190278 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: UCART123_01
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 8

Brief Summary

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).

Conditions

Relapsed/Refractory Acute Myeloid Leukemia

Keywords

Acute Myeloid Leukemia; Relapsed/Refractory Acute Myeloid Leukemia; Chimeric Antigen Receptor T-Cell (CAR-T) therapy; Allogeneic; Transcription Activator-Like Effector Nuclease (TALEN)

Outcome Measures

Primary Outcomes (2)

• 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

Secondary Outcomes (16)

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

  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

  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

  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

  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)

  alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

Study Arms (1)

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

Biological: UCART123v1.2

Interventions

UCART123v1.2 BIOLOGICAL

Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor Biological/vaccine: CLLS52 A monoclonal antibody that recognizes the CD52 antigen Other Names: Alemtuzumab

Dose Escalation

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cellectis S.A.: lead

Study Sites (8)

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

San Francisco, California, 94143, United States

Location

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, 33612, United States

Location

Northwestern University

Chicago, Illinois, 60201, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

University of Pennsylvania - Abramson Cancer Center

Philadelphia, Pennsylvania, 19104, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Gail Roboz, Dr

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2017
• First Posted: June 16, 2017
• Study Start: June 19, 2017
• Primary Completion: December 24, 2024
• Study Completion: December 1, 2025
• Last Updated: August 7, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)