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

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ClinicalTrials.gov Identifier: NCT03190278
Recruitment Status : Recruiting
First Posted : June 16, 2017
Last Update Posted : September 22, 2021
Sponsor:
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 UCART123v1.2 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).

Condition or disease Intervention/treatment Phase
Relapsed/Refractory Acute Myeloid Leukemia Biological: UCART123v1.2 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 65 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 : October 2022
Estimated Study Completion Date : October 2022


Arm Intervention/treatment
Experimental: Part 1: Dose Escalation

Several tested doses of UCART123v1.2 with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D)

Dose Expansion: UCART123v1.2 administered at the selected dose determined from the dose escalation phase

Biological: UCART123v1.2
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 UCART123v1.2 - Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study


Secondary Outcome Measures :
  1. Response Assessment [ 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 ]
  2. 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 ]
  3. 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 ]
  4. Overall Survival [ Time Frame: From the first day of study treatment to the date of death from any cause, assessed up to Month 24 ]


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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
Criteria

Main Inclusion Criteria:

  • Patients with relapsed or primary refractory AML (as defined in 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 APL or CNS Leukemia
  • Previous investigation gene or cell therapy (including CAR)
  • ≥ 2 prior allogeneic SCTs
  • Prior treatment with rituximab or other anti-CD20 therapy within 3 months
  • Any known active or uncontrolled infection
  • 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 ClinicalTrials.gov identifier (NCT number): NCT03190278


Contacts
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Contact: Cellectis Central Contact 1-347-752-4044 clinicaltrials@cellectis.com

Locations
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United States, California
University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center Recruiting
San Francisco, California, United States, 94143
United States, Florida
Sylvester Comprehensive Cancer Center Recruiting
Miami, Florida, United States, 33136
H. Lee Moffitt Cancer Center & Research Institute Recruiting
Tampa, Florida, United States, 33612
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60201
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, Pennsylvania
University of Pennsylvania - Abramson Cancer Center Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Cellectis S.A.
Investigators
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Principal Investigator: Gail Roboz, Dr Weill Medical College of Cornell University
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Responsible Party: Cellectis S.A.
ClinicalTrials.gov Identifier: NCT03190278    
Other Study ID Numbers: UCART123_01
First Posted: June 16, 2017    Key Record Dates
Last Update Posted: September 22, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Allogeneic
Transcription Activator-Like Effector Nuclease (TALEN)
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Neoplasms