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Phase I Study of UCART123 in Patient With Adverse Genetic Risk Acute Myeloid Leukemia

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. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04106076
Recruitment Status : Withdrawn (The trial was discontinued due to sponsor's decision and not a consequence of any safety concern)
First Posted : September 26, 2019
Last Update Posted : July 14, 2020
Information provided by (Responsible Party):
Cellectis S.A.

Brief Summary:
This is a Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). The purpose of this study is to evaluate the safety and clinical activity of multiple infusions of UCART123 and to determine the Maximum Tolerated Dose (MTD).

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukaemia Biological: UCART123 Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I, Open Label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of Multiple Infusions of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor) in Patients With Adverse Genetic Risk Acute Myeloid Leukaemia
Actual Study Start Date : July 11, 2019
Actual Primary Completion Date : December 5, 2019
Actual Study Completion Date : December 5, 2019

Arm Intervention/treatment
Experimental: Dose escalation
Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified.
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 ]
    Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Main Inclusion Criteria:

  • Patients newly diagnosed with CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined as per ELN guidelines (Döhner et al., 2017)
  • Eastern Cooperative Oncology Group performance status of 0 or 1
  • No prior gene or experimental cellular therapy
  • No organ dysfunction that in the opinion of the investigator precludes intensive induction chemotherapy or cellular therapy

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 ClinicalTrials.gov identifier (NCT number): NCT04106076

Sponsors and Collaborators
Cellectis S.A.
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Principal Investigator: Ghulam Mufti, Pr Kings college London NHS Foundation Trust
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Responsible Party: Cellectis S.A.
ClinicalTrials.gov Identifier: NCT04106076    
Other Study ID Numbers: UCART123_03
First Posted: September 26, 2019    Key Record Dates
Last Update Posted: July 14, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cellectis S.A.:
Acute Myeloid Leukaemia
Newly diagnosed AML
ELN adverse genetic risk group
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