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Study Evaluating Safety and Efficacy of UCART123 in Patients With Acute Myeloid Leukemia (AML123)

This study has suspended participant recruitment.
(The protocol is being revised; the trial will resume once revisions are made.)
Sponsor:
ClinicalTrials.gov Identifier:
NCT03190278
First Posted: June 16, 2017
Last Update Posted: September 14, 2017
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.
Information provided by (Responsible Party):
Cellectis S.A.
  Purpose
Phase I, first-in-human, open-label, dose-finding study of UCART123 administered intravenously to patients with Acute Myeloid Leukemia (AML), followed by a dose-escalation stage in Relapsed or Refractory AML patients and a dose expansion stage in Relapsed/Refractory AML patients, and in poor-prognosis, newly diagnosed AML patients in the European LeukemiaNet (ELN) Adverse genetic risk group.

Condition Intervention Phase
Acute Myeloid Leukemia Biological: UCART123 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel 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 a Single Dose of UCART123 (Allogeneic Engineered T Cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Relapsed/Refractory Acute Myeloid Leukemia, and Patients With Newly Diagnosed High-risk Acute Myeloid Leukemia

Resource links provided by NLM:


Further study details as provided by Cellectis S.A.:

Primary Outcome Measures:
  • Incidence, nature, and severity of adverse events and serious adverse events [ Time Frame: Through Day 84 (+/- 7 days), dose limiting toxicities are assessed up to 42 days after UCART123 infusion ]
    Adverse events assessed according to common terminology criteria for adverse events (CTCAE v4.03); cytokine release syndrome (CRS); tumor lysis syndrome (TLS); graft vs. host disease (GvHD)


Secondary Outcome Measures:
  • Assessment of Anti-leukemic activity [ Time Frame: Day 14, between Day 28 and Day 35, then every 3 months for one year and every 6 months during the second year after treatment ]
    Anti-leukemic activity, as measured by International Working Group (IWG) criteria for AML response criteria


Estimated Enrollment: 156
Actual Study Start Date: June 19, 2017
Estimated Study Completion Date: December 15, 2021
Estimated Primary Completion Date: June 15, 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Part 1: Dose Escalation
A single IV administration of UCART123 in the dose escalation phase will explore 3 doses of UCART123 ranging from 6.25x10^5 cells/kg to 6.25 x10^6 cells/kg and continue until the Recommended Phase 2 Dose (RP2D) is identified.
Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen
Experimental: Part 2: Dose Expansion
A single intravenous administration of UCART123. 2 Cohorts: Relapsed and Refractory AML and newly diagnosed AML in the ELN adverse genetic risk group.
Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • AML Blast cell expressing CD123 by flow cytometry performed as per standard practice
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1
  • Adequate organ function.

Patients enrolled in the Dose Finding Portion of the Study:

  • Patients ≥18 and ≤75 years old with relapsed or primary refractory acute myeloid leukemia.
  • Patients enrolled onto the dose-finding phase of the study must have an identified donor and transplant strategy prior to initiation of the lymphodepletion regimen.

Patients enrolled in the Expansion Phase of the Study: Relapsed/Refractory Cohort

• Patients ≥18 years old with relapsed or primary refractory acute myeloid leukemia.

Newly Diagnosed Cohort

• Patients with newly diagnosed, untreated Acute Myeloid Leukemia (as defined by World Health Organization (WHO) criteria,) who meet criteria for the European Leukemia Net (ELN) Adverse genetics prognostic group.

Main Exclusion Criteria:

  • Previous treatment with investigational gene or chimeric antigen receptor therapy.
  • Acute Promyelocytic Leukemia
  • Active or previous central nervous system leukemia involvement
  • Presence of active and clinically relevant Central Nervous System (CNS) disorder
  • Use of rituximab and other anti-cluster of differentiation 20 (CD20) antibodies known to have the same epitope as rituximab or anti-CD20 for which the epitope is unknown within 3 months prior to start of lymphodepletion
  • Active treatment with prednisone >20 mg or other immunosuppressive agents that cannot be stopped
  • Known infection with Human Immunodeficiency Virus (HIV) or Human T-lymphotropic Virus 1 (HTLV-1).
  • A known hypersensitivity to any of the test materials or related compounds including murine and bovine products;
  • Any known uncontrolled cardiovascular disease;
  • Active bacterial, fungal or viral infection not controlled by adequate treatment, at enrollment
  Contacts and Locations
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): NCT03190278


Locations
United States, New York
Weill Cornell Medical College
New York, New York, United States, 10065
Sponsors and Collaborators
Cellectis S.A.
Investigators
Study Director: Loan Hoang-Sayag, MD Cellectis S.A.
  More Information

Responsible Party: Cellectis S.A.
ClinicalTrials.gov Identifier: NCT03190278     History of Changes
Other Study ID Numbers: UCART123_01
First Submitted: June 14, 2017
First Posted: June 16, 2017
Last Update Posted: September 14, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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
Newly diagnosed AML
ELN adverse genetic risk group
Chimeric Antigen Receptor T-Cell (CAR-T) therapy
Allogeneic

Additional relevant MeSH terms:
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Neoplasms