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Study to Evaluate the Safety and Clinical Activity of UCART123 in Patients With BPDCN (ABC123)

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.
 
ClinicalTrials.gov Identifier: NCT03203369
Recruitment Status : Terminated (The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.)
First Posted : June 29, 2017
Last Update Posted : July 31, 2019
Sponsor:
Information provided by (Responsible Party):
Cellectis S.A.

Tracking Information
First Submitted Date  ICMJE June 14, 2017
First Posted Date  ICMJE June 29, 2017
Last Update Posted Date July 31, 2019
Actual Study Start Date  ICMJE June 28, 2017
Actual Primary Completion Date June 27, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 29, 2019)
Incidence, nature, and severity of adverse events and serious adverse events [ Time Frame: Through day 84 ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 28, 2017)
Incidence, nature, and severity of adverse events and serious adverse events [ Time Frame: Through day 84 ]
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 28, 2017)
Assessment of Anti-tumor activity [ Time Frame: Between Day 28 and Day 35, then every 3 months during 1 year, then every 6 months for a total of 2 years ]
Anti-tumor activity following BPDCN composite criteria from 3 sources: 1) Assessment of anti-tumor activity by International Working Group (IWG) criteria for Acute Myeloid Leukemia (AML); 2) Assessment of skin lesions by Modified Severity Weights Assessment Tool (mSWAT); and 3) Assessment of anti-tumor activity using the Lugano criteria.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Safety and Clinical Activity of UCART123 in Patients With BPDCN
Official Title  ICMJE Phase 1, 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 Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Brief Summary A Phase 1 dose-finding study of Universal Chimeric Antigen Receptor T-cells targeting cluster of differentiation (CD) 123 (UCART123) administered intravenously to patients with relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), followed by a dose expansion phase in relapsed or refractory BPDCN patients or newly diagnosed BPDCN patients.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Intervention  ICMJE Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen.
Study Arms  ICMJE
  • Experimental: Part 1: Dose Escalation
    A single intravenous administration of UCART123. Dose escalation in Part 1 will include 3 doses ranging from 6.25 x 10^5 cells/kg to 6.25 x 10^6 cells/kg and continue until the Recommended Phase 2 Dose (RP2D) is identified.
    Intervention: Biological: UCART123
  • Experimental: Part 2: Dose Expansion
    A single intravenous administration of UCART123 at the RP2D. 2 Cohorts: Patients with Relapsed/Refractory BPDCN and Newly Diagnosed BPDCN.
    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 Terminated
Actual Enrollment  ICMJE
 (submitted: July 29, 2019)
1
Original Estimated Enrollment  ICMJE
 (submitted: June 28, 2017)
72
Actual Study Completion Date  ICMJE June 27, 2019
Actual Primary Completion Date June 27, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  • Patients with a diagnosis BPDCN according to World Health Organization (WHO) classification confirmed by hematopathology;
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1;
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03203369
Other Study ID Numbers  ICMJE UCART123_02
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
Study Director: Ioana Kloos, MD Cellectis S.A.
PRS Account Cellectis S.A.
Verification Date July 2019

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