Study Evaluating Safety and Efficacy of UCART123v1.2 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AMELI-01)

August 4, 2025 updated by: Cellectis S.A.

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

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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
29

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94143
        • University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center
    • Florida
      • Tampa, Florida, United States, 33612
        • H. Lee Moffitt Cancer Center & Research Institute
    • Illinois
      • Chicago, Illinois, United States, 60201
        • Northwestern University
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Dana-Farber Cancer Institute
    • New York
      • Buffalo, New York, United States, 14263
        • Roswell Park Cancer Institute
      • New York, New York, United States, 10021
        • Weill Medical College of Cornell University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania - Abramson Cancer Center
    • Texas
      • Houston, Texas, United States, 77030
        • MD Anderson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years to 61 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Dose Escalation

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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incidence of adverse events (AE)/serious adverse events (SAE)/Dose Limiting Toxicities (DLT) [Safety and Tolerability]
Time Frame: 24 Months
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
Time Frame: Up to Day 28 post last UCART123v1.2 infusion
Up to Day 28 post last UCART123v1.2 infusion

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
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
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
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
From the first day of study treatment to the date of death from any cause, assessed up to Month 24
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
At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24
Pharmacokinetic (PK) Analysis: Standard PK Analysis will be completed to obtain Maximum plasma concentration (Cmax)
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain time to reach Cmax (Tmax)
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain total area under curve from zero to infinity (AUC-infinity)
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Rate
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Half-life
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Clearance
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Volume of Distribution
Time Frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose
Pharmacodynamic Analysis: Pharmacodynamics Monitoring of the incidence of anti-cluster of differentiation 52 (anti-CD52; alemtuzumab) antibodies (ADA) in serum Pre-alemtuzumab administration and through Day 84
Time Frame: From screening through Day 84
From screening through Day 84
Pharmacodynamic Analysis: Pharmacodynamics Quantitation of T cells in peripheral blood
Time Frame: From screening through Day 84
From screening through Day 84
Pharmacodynamic Analysis: Pharmacodynamics Quantitation of B cells in peripheral blood
Time Frame: From screening through Day 84
From screening through Day 84
Pharmacodynamic Analysis: Pharmacodynamics Quantitation of natural killer (NK) cells in peripheral blood
Time Frame: From screening through Day 84
From screening through Day 84
Pharmacodynamic Analysis: Pharmacodynamics Quantitation of total lymphocytes in peripheral blood
Time Frame: From screening through Day 84
From screening through Day 84

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Cellectis S.A.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Gail Roboz, Dr, Weill Medical College of Cornell University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 19, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 24, 2024

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 14, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 15, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 16, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 7, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 4, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • UCART123_01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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