Clinical Trial

Disease: Blastic Plasmacytoid Dendritic Cell Neoplasm, BPDCN, (NCT03203369)

Disease info:

Blastic plasmacytoid dendritic cell neoplasm was previously known as natural killer (NK) cell leukaemia/lymphoma. This is a highly aggressive, historically difficult-to-diagnose haematologic malignancy with a poor prognosis. As understanding of the biology and origin of this type of cancer has improved, the World Health Organization (WHO) established the term blastic plasmacytoid dendritic cell neoplasm (BPDCN) in 2008. BPDCN is currently listed as its own category in the 2016 World Health Organization revision. Most often, BPDCN presents with features of both lymphoma and leukaemia. Little data is available about BPDCN and there is no standard approach to treatment. 

Source: The Leukemia & Lymphoma Society® (LLS)

According to LLS, BPDCN has an annual incidence of 1,000 to 1,400 (US and Europe combined). The median age at diagnosis is in the mid-60s, with approximately 75% of cases occurring in men.
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)

United States, Texas 
MD Anderson Cancer Center, Houston, Texas, United States, 77030

Study start:
Jun. 28, 2017
1 participant
Gene editing method:
Type of edit:
Gene knock-out
T Cell Receptor alpha constant (TCRαβ)
Delivery method:
Lentivirus - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated


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.

Last updated: Feb. 5, 2024
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