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Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia (AMELI-01)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03190278
Recruitment Status : Recruiting
First Posted : June 16, 2017
Last Update Posted : May 18, 2020
Information provided by (Responsible Party):
Cellectis S.A.

Brief Summary:
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 one infusion of UCART123 and determine the Maximum Tolerated Dose (MTD).

Condition or disease Intervention/treatment Phase
Relapsed/Refractory Acute Myeloid Leukemia Biological: UCART123 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 59 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 19, 2017
Estimated Primary Completion Date : November 18, 2021
Estimated Study Completion Date : November 18, 2021

Arm Intervention/treatment
Experimental: Part 1: Dose Escalation

Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified

Dose Expansion: UCART123 administered at the selected dose determined from the dose escalation phase

Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor

Primary Outcome Measures :
  1. Incidence of AE/SAE/DLT [Safety and Tolerability] [ Time Frame: 24 Months ]
    Safety of UCART123 - Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Main Inclusion Criteria:

  • Patients with relapsed or primary refractory AML (as defined in WHO criteria) with >5% bone marrow blasts
  • Eastern Cooperative Oncology Group Performance Status of 0 or 1
  • Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within screening period
  • Other criteria may apply

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03190278

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Contact: Cellectis Central Contact 1-347-579-0935

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United States, Florida
H. Lee Moffitt Cancer Center & Research Institute Recruiting
Tampa, Florida, United States, 33612
United States, Massachusetts
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
United States, New York
Weill Medical College of Cornell University Recruiting
New York, New York, United States, 10021
United States, Texas
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Cellectis S.A.
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Principal Investigator: Gail Roboz, Dr Weill Medical College of Cornell University
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Responsible Party: Cellectis S.A. Identifier: NCT03190278    
Other Study ID Numbers: UCART123_01
First Posted: June 16, 2017    Key Record Dates
Last Update Posted: May 18, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cellectis S.A.:
Acute Myeloid Leukemia
Relapsed/Refractory Acute Myeloid Leukemia
Chimeric Antigen Receptor T-Cell (CAR-T) therapy
Transcription Activator-Like Effector Nuclease (TALEN)
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type