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

Cellectis

Status and phase: Terminated; Phase 1

Conditions: Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Treatments: Biological: UCART123

Study type: Interventional

Funder types: Industry

Identifiers

NCT03203369

UCART123_02

Details and patient eligibility

About

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.

Enrollment: 1 patient

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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;

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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1 participants in 2 patient groups

Part 1: Dose Escalation

Experimental group

Description:

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.

Treatment:

Biological: UCART123

Part 2: Dose Expansion

Experimental group

Description:

A single intravenous administration of UCART123 at the RP2D. 2 Cohorts: Patients with Relapsed/Refractory BPDCN and Newly Diagnosed BPDCN.

Treatment:

Biological: UCART123