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

NCT03203369 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: UCART123_02
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Keywords

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)

Outcome Measures

Primary Outcomes (1)

• Incidence, nature, and severity of adverse events and serious adverse events

  Through day 84

Study Arms (2)

Part 1: Dose Escalation

EXPERIMENTAL

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

Part 2: Dose Expansion

EXPERIMENTAL

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

Biological: UCART123

Interventions

UCART123 BIOLOGICAL

Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor given as a single dose following a lymphodepleting regimen.

Part 1: Dose Escalation; Part 2: Dose Expansion

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

Sponsors & Collaborators

• Cellectis S.A.: lead

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Blastic Plasmacytoid Dendritic Cell Neoplasm; Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Histiocytic Disorders, Malignant; Neoplasms by Histologic Type; Neoplasms; Leukemia; Lymphoma; Neoplasms by Site; Skin Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Ioana Kloos, MD

  Cellectis S.A.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2017
• First Posted: June 29, 2017
• Study Start: June 28, 2017
• Primary Completion: June 27, 2019
• Study Completion: June 27, 2019
• Last Updated: July 31, 2019

Record last verified: 2019-07

Locations

US (1)