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

This study has suspended participant recruitment.
(The protocol is being revised; the trial will resume once revisions are made.)
Sponsor:
ClinicalTrials.gov Identifier:
NCT03203369
First Posted: June 29, 2017
Last Update Posted: September 14, 2017
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.
Information provided by (Responsible Party):
Cellectis S.A.
  Purpose
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.

Condition Intervention Phase
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Biological: UCART123 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)

Resource links provided by NLM:


Further study details as provided by Cellectis S.A.:

Primary Outcome Measures:
  • 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)


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 72
Actual Study Start Date: June 28, 2017
Estimated Study Completion Date: June 15, 2022
Estimated Primary Completion Date: December 15, 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen.
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.
Biological: UCART123
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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;

Patients enrolled in the Dose Finding Portion of the Study:

  • Patients ≥18 years and ≤80 years
  • Patients with histological and/or cytological evidence of BPDCN in the peripheral blood, bone marrow, spleen, lymph nodes, skin, and/or other sites that is persistent/recurrent following prior treatment for BPDCN.

Patients enrolled in the Expansion Phase of the Study:

  • Age ≥18 years
  • Patients with histological and/or cytological evidence of BPDCN in the peripheral blood, bone marrow, spleen, lymph nodes, skin, and/or other sites that is either previously untreated or is persistent/recurrent following prior treatment for BPDCN.

Main Exclusion Criteria:

  • Previous treatment with investigational gene or chimeric antigen receptor therapy;
  • Active or previous central nervous system leukemia involvement
  • Presence of active and clinically relevant central nervous system (CNS) disorder;
  • Immunosuppression following a Hematopoietic Stem Cell Transplantation (HSCT) within 6 weeks prior to study entry;
  • Use of rituximab and other anti-cluster of differentiation antigen 20 (CD20) antibodies known to have the same epitope as rituximab or anti-cluster of differentiation antigen 20 (CD20) for which the epitope is unknown within 3 months prior to enrollment;
  • Active treatment with immunosuppressive agents that cannot be stopped;
  • Known infection with human immunodeficiency virus (HIV) or human T-lymphotrophic virus (HTLV-1);
  • Any known uncontrolled cardiovascular disease, pulmonary embolism, hypertension not adequately controlled by standard medications within 3 months prior to enrollment.
  • Active bacterial, fungal or viral infection not controlled by adequate treatment, at enrollment.
  Contacts and Locations
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): NCT03203369


Locations
United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Cellectis S.A.
Investigators
Study Director: loan Hoang-Sayag, MD Cellectis S.A.
  More Information

Responsible Party: Cellectis S.A.
ClinicalTrials.gov Identifier: NCT03203369     History of Changes
Other Study ID Numbers: UCART123_02
First Submitted: June 14, 2017
First Posted: June 29, 2017
Last Update Posted: September 14, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Cellectis S.A.:
Blastic plasmacytoid dendritic cell neoplasm
Natural Killer (NK) cell blastic lymphoma
Hematologic malignancies
Cluster of differentiation (CD) CD4+CD56+ hematodermic tumor
Acute leukemia and myeloid neoplasms
Chimeric Antigen Receptor T-cell (CAR-T) therapy
Allogeneic
Cluster of differentiation 4 (CD4)
Cluster of differentiation 56 (CD56)

Additional relevant MeSH terms:
Neoplasms