Clinical Trial

Disease: Acute Myeloid Leukemia, AML, (NCT05309733)

Disease info:

Leukaemia is cancer of the white blood cells. White blood cells help your body to fight infection. Your blood cells form in your bone marrow. In leukaemia, 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 leukaemia (AML), there are too many of a specific type of white blood cell called a myeloblast.

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

The estimated number of new cases of acute myeloid leukaemia is 4.3 per 100,000 men and women per year. The estimated number of deaths is 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:
A Long-term Follow-up Study of Patients Who Received VOR33

United States, California

University of California San Diego Moores Cancer Center


United States, Florida

Miami Cancer Institute


United States, Missouri

Washington University School of Medicine Siteman Cancer Center


United States, New Jersey

John Theurer Cancer Center at Hackensack University Medical Center


United States, New York

Memorial Sloan Kettering Cancer Center


United States, Ohio

University Hospitals Seidman Cancer Center


United States, Washington

Fred Hutchinson Cancer Research Center


Canada, Quebec

Hôpital Maisonneuve-Rosemont

Study start:
Apr. 15, 2022
Gene editing method:
Type of edit:
Gene knock-out
CD33 molecule
Delivery method:
Undisclosed. - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


VBP201 study is the VOR33 long-term follow-up (LTFU) study focusing on assessing long-term safety and efficacy of VOR33. This study may last up to 15 years (counted from the date of VOR33 infusion completion). All patients who have participated in a study involving the drug product VOR33 and have completed the applicable study or prematurely discontinued, but have at least received the VOR33 infusion, will be asked to participate in the LTFU study.

Last updated: May. 4, 2023
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