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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 : June 7, 2022
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 June 7, 2022
Actual Study Start Date  ICMJE June 19, 2017
Estimated Primary Completion Date March 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 3, 2021)
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
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
 (submitted: February 3, 2021)
  • 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 ]
  • 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 ]
  • 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 ]
  • Overall Survival [ Time Frame: From the first day of study treatment to the date of death from any cause, assessed up to Month 24 ]
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 UCART123v1.2 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).
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: UCART123v1.2
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor
Study Arms  ICMJE 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

Intervention: Biological: UCART123v1.2
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: February 3, 2021)
65
Original Estimated Enrollment  ICMJE
 (submitted: June 15, 2017)
156
Estimated Study Completion Date  ICMJE March 2023
Estimated Primary Completion Date March 2023   (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
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Cellectis Central Contact 1-347-752-4044 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: No
Current Responsible Party Cellectis S.A.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Cellectis S.A.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gail Roboz, Dr Weill Medical College of Cornell University
PRS Account Cellectis S.A.
Verification Date June 2022

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