Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT01044654)

Disease info:

HIV stands for human immunodeficiency virus. It harms the immune system by destroying the white blood cells that fight infection. This puts at risk for serious infections and certain cancers.

AIDS stands for acquired immunodeficiency syndrome. It is the final stage of infection with HIV. Not everyone with HIV develops AIDS.

Worldwide, there were about 1.7 million new cases of HIV in 2018. About 37.9 million people were living with HIV around the world in 2018, and 23.3 million of them were receiving medicines to treat HIV, called antiretroviral therapy (ART).
Official title:
A Phase 1 Dose Escalation, Single Dose Study of Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-278 in HIV-Infected Patients Who Have Exhibited Suboptimal CD4+ T-Cell Gains During Long-Term Antiretroviral Therapy

Study Director: Winson Tang, M.D. Sangamo Therapeutics Inc.


United States, California

UCLA Center for AIDS Research and Education, Los Angeles, California, United States, 90035

Orange Coast Medical Group, Newport Beach, California, United States, 92663

Quest Clinical Research, San Francisco, California, United States, 94115


United States, Connecticut

Circle CARE Center, LLC, Norwalk, Connecticut, United States, 06851


United States, Florida

Orlando Immunology Center, Orlando, Florida, United States, 32803


United States, Missouri

Central West Clinical Research, Inc., St Louis, Missouri, United States, 63108


United States, New Mexico

Southwest CARE Center, Santa Fe, New Mexico, United States, 87505


United States, New York

Ricky K Hsu, MD, PC, New York, New York, United States, 10011


United States, Texas

Gordon Crofoot, MD, PA, Houston, Texas, United States, 77098

Study start:
Dec. 1, 2009
19 participants
Gene editing method:
ZFN- Zinc Finger Nucleases
Type of edit:
Gene knock-out
C-C motif chemokine receptor 5 (CCR5)
Delivery method:
Adenovirus (AV) - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed


This research study is being carried out to study a new way to possibly treat HIV. This agent is called a "Zinc Finger Nuclease" or ZFN for short. ZFNs are proteins that can delete another protein named CCR5. This CCR5 protein is required for certain types of HIV (CCR5 tropic) to enter into and infect your T-cells. T cells are one of the white blood cells used by the body to fight HIV. The most important of these are called "CD4 T-cells."

Some People are born without CCR5 on their T-cells. These people remain healthy and are resistant to infection with HIV. Other people have a low number of CCR5 on their T-cells, and their HIV disease is less severe and is slower to cause disease (AIDS).

Even with no detectable levels of HIV in the blood, HIV remains in some tissues in the body, primarily the gut tissue. HIV infects the CD4+ T-cells including in the blood and gut. The new treatment to be studied will involve removing white blood cell from the blood that contains CD4+ T-cells. The extracted CD4+ T-cells are then genetically modified by the ZFNs to be resistant to infection by HIV by removing the CCR5 gene from the surface of the CD4+ T cell where HIV enters the cell. Additional genetically modified cells are manufactured and then re-infused back into you. Researchers hope that these genetically modified cells will be resistant to infection by HIV and will be able to reproduce additional resistant CD4+ T-cells in your body.

Laboratory studies have shown that when CD4+ T-cells are modified with ZFNs, HIV is prevented from killing the CD4+ T-cells. On the basis of these laboratory results, thre is the potential that ZFNs may work in humans infected with HIV and improve their immune system by allowing their CD4+ T-cells to survive longer.

The purpose of this research study is to find out whether "zinc finger" modified CD4+ T-cells are safe to give to humans and find how "zinc finger" modified T-cell affects HIV.

Last updated: Jun. 13, 2023
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