Clinical Trial

Disease: Acute Myeloid Leukaemia, AML, (NCT05309733)

Disease info:

Leukaemia is cancer of the white blood cells which are responsible for fighting infection. In leukaemia, the bone marrow produces abnormal levels of white blood cells. Acute myeloid leukaemia (AML) starts in the bone marrow but often quickly spreads into the bloodstream. AML sometimes spreads to other parts of the body such as the lymph nodes, liver, central nervous system and testicles. 

Commonly, AML develops from cells other than lymphocytes that would normally develop into white blood cells. The World Health Organization (WHO) divides AML into several subtypes based off genetic association. AML is also known as acute myelocytic leukaemia, acute myelogenous leukaemia, acute granulocytic leukaemia, and acute non-lymphocytic leukaemia.


The American Cancer Society estimates approximately 20,380 cases of AML will be diagnosed in the U.S. in 2023, and approximately 11,310 deaths. AML is predominantly found in adults and is one of the most common types of leukaemia in adults.
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
36 participants
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: Jan. 7, 2024
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