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

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Intervention / Treatment
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* Biological: UCART123


Participation Criteria
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Eligibility Criteria
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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;

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Part 1: Dose Escalation<br>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<br>* Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen.<br>|
| Experimental: Part 2: Dose Expansion<br>A single intravenous administration of UCART123 at the RP2D. 2 Cohorts: Patients with Relapsed/Refractory BPDCN and Newly Diagnosed BPDCN. | Biological: UCART123<br>* Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence, nature, and severity of adverse events and serious adverse events |  | Through day 84 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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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)