Clinical Trial

Disease: Relapsed/Refractory Acute Myeloid Leukemia, AML, (NCT03190278)

Disease info:

Leukemia is cancer of the white blood cells. White blood cells help your body fight infection. White blood cells help your body fight infection. Your blood cells form in your bone marrow. In leukemia, however, the bone marrow produces abnormal white blood cells. These cells crowd out the healthy blood cells, making it hard for blood to do its work. In acute myeloid leukemia (AML), there are too many of a specific type of white blood cell called a myeloblast.

AML is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. 

Frequency:
The number of new cases of acute myeloid leukemia was 4.3 per 100,000 men and women per year. The number of deaths was 2.8 per 100,000 men and women per year. These rates are age-adjusted and based on 2012-2016 cases and deaths.
Official title:
Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia (AMELI-01)
Who:

Principal Investigator: Gail Roboz, Dr  Weill Medical College of Cornell University

Sponsor:

Cellectis S.A.

Partners:
Locations:

United States, Florida

United States, Massachusetts

United States, New York

United States, Texas

United States, Illinois

United States, Pennsylvania

United States, California

Study start:
Jun. 19, 2017
Enrollment:
65 participants
Gene editing method:
TALENs
Type of edit:
Gene knock-out
Gene:
Interleukin 3 receptor subunit alpha (CD123 or IL3RA), T-Cell Receptor (TCR)
Delivery method:
Lentivirus (LV) and electroporation - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/study/NCT03190278
Safety updates:

(28-05-2018) Approval of UCART123 Amendment in AML to Accelerate Clinical Development

Other links:

LinkAcute Myeloid Leukemia Disease InformationLinkWhat is UCART123?LinkAcute Myeloid Leukemia FrequencyLinkStraightforward Generation of Ultrapure Off-the-Shelf Allogeneic CAR-T CellsLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem Qasim

Note:
UCART123 is Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor. In the first engineering step, genes are added to the T-cell genome with lentiviral vectors. In the second T-cell engineering step transfection is achieved with electroporation.
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

Phase I, first-in-human, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of UCART targeting CD123 in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of one infusion of UCART123 and determine the Maximum Tolerated Dose (MTD).

UCART is Universal CAR-T cells. That is T Cells that have been taken from healthy donors (not from patients themselves). TALENS are used to disable the TCRαβ gene T cells use to recognize ’self’ to prevent them from attacking the host (graft vs host).

Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor.

Experimental: Part 1: Dose Escalation

Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified

Dose Expansion: UCART123 administered at the selected dose determined from the dose escalation phase

Last updated: Apr. 10, 2022
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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