Phase I Study of UCART123 in Patient With Adverse Genetic Risk Acute Myeloid Leukemia

Phase I, Open Label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of Multiple Infusions of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor) in Patients With Adverse Genetic Risk Acute Myeloid Leukaemia

This is a Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). The purpose of this study is to evaluate the safety and clinical activity of multiple infusions of UCART123 and to determine the Maximum Tolerated Dose (MTD).

Study Overview

• Status: Withdrawn
• Conditions: Acute Myeloid Leukaemia
• Intervention / Treatment: Biological: UCART123

• Study Type: Interventional
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 63 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Main Inclusion Criteria:

• Patients newly diagnosed with CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined as per ELN guidelines (Döhner et al., 2017)
• Eastern Cooperative Oncology Group performance status of 0 or 1
• No prior gene or experimental cellular therapy
• No organ dysfunction that in the opinion of the investigator precludes intensive induction chemotherapy or cellular therapy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose escalation; Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified.	Biological: UCART123; Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of AE/SAE/DLT [Safety and Tolerability]	Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study	24 months

Collaborators and Investigators

• Sponsor: Cellectis S.A.
• Investigators: Principal Investigator: Ghulam Mufti, Pr, Kings college London NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Actual): July 11, 2019
• Primary Completion (Actual): December 5, 2019
• Study Completion (Actual): December 5, 2019

Study Registration Dates

• First Submitted: September 25, 2019
• First Submitted That Met QC Criteria: September 25, 2019
• First Posted (Actual): September 26, 2019

Study Record Updates

• Last Update Posted (Actual): July 14, 2020
• Last Update Submitted That Met QC Criteria: July 13, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Keywords: Allogeneic; Acute Myeloid Leukaemia; Newly diagnosed AML; Chimeric Antigen Receptor T-Cell (CAR-T) therapy; Transcription Activator-Like Effector Nuclease (TALEN); ELN adverse genetic risk group
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: UCART123_03

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No