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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.
 
ClinicalTrials.gov Identifier: NCT03190278
Recruitment Status : Recruiting
First Posted : June 16, 2017
Last Update Posted : May 18, 2020
Sponsor:
Information provided by (Responsible Party):
Cellectis S.A.

Tracking Information
First Submitted Date  ICMJE June 14, 2017
First Posted Date  ICMJE June 16, 2017
Last Update Posted Date May 18, 2020
Actual Study Start Date  ICMJE June 19, 2017
Estimated Primary Completion Date November 18, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 4, 2019)
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
Original Primary Outcome Measures  ICMJE
 (submitted: June 15, 2017)
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)
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: June 15, 2017)
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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Relapsed/Refractory Acute Myeloid Leukemia
Intervention  ICMJE Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor
Study Arms  ICMJE 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

Intervention: Biological: UCART123
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 4, 2019)
59
Original Estimated Enrollment  ICMJE
 (submitted: June 15, 2017)
156
Estimated Study Completion Date  ICMJE November 18, 2021
Estimated Primary Completion Date November 18, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 64 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Cellectis Central Contact 1-347-579-0935 clinicaltrials@cellectis.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03190278
Other Study ID Numbers  ICMJE UCART123_01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Cellectis S.A.
Study Sponsor  ICMJE Cellectis S.A.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gail Roboz, Dr Weill Medical College of Cornell University
PRS Account Cellectis S.A.
Verification Date May 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP