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Study Evaluating Safety and Efficacy of UCART123v1.2 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 : February 5, 2024
Sponsor:
Information provided by (Responsible Party):
Cellectis S.A.

Brief Summary:
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).

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 : December 2024
Estimated Study Completion Date : December 2024


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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

  2. Dose escalation and expansion part: Occurrence of DLTs [ Time Frame: Up to Day 28 post last UCART123v1.2 infusion ]

Secondary Outcome Measures :
  1. 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 ]
  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 ]
  5. 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 ]
  6. 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 ]
  7. 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 ]
  8. 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 ]
  9. 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 ]
  10. 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 ]
  11. 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 ]
  12. 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 ]
  13. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of T cells in peripheral blood [ Time Frame: From screening through Day 84 ]
  14. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of B cells in peripheral blood [ Time Frame: From screening through Day 84 ]
  15. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of natural killer (NK) cells in peripheral blood [ Time Frame: From screening through Day 84 ]
  16. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of total lymphocytes in peripheral blood [ Time Frame: From screening through Day 84 ]


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
Criteria

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

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 Withdrawn
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
Roswell Park Cancer Institute Recruiting
Buffalo, New York, United States, 14263
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: February 5, 2024
Last Verified: January 2024
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