Clinical Trial

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

Disease info:

Human immunodeficiency virus (HIV) is a virus that attacks the immune system by infecting and killing white blood cells known as CD4+ T-cells. CD4+ T-cells represent a vital part of the immune system and untreated HIV infections render patients more vulnerable to dangerous infections by other pathogens, including bacteria and fungi. If the virus remains untreated it can lead to patients developing the life-threatening disease acquired immunodeficiency syndrome (AIDS). Untreated HIV can leave people vulnerable to life-threatening infections. HIV symptoms often manifest as flu-like symptoms such as fever, chills, rash, night sweats, muscle aches, sore throat, and fatigue. Today, antiviral medications can allow people living with HIV to live healthy lives.

Frequency:
In 2021, 36,136 people received an HIV diagnosis in the United States and dependent areas. An estimated 1.2 million people in the United States had HIV at the end of 2021.
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
Who:

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

Sponsor:

Sangamo Therapeutics Inc.

Partners:
Locations:

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
Enrollment:
19 participants
Gene editing method:
ZFN- Zinc Finger Nucleases
Type of edit:
Gene knock-out
Gene:
C-C motif chemokine receptor 5 (CCR5)
Delivery method:
Adenovirus (AV) - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT01044654
Safety updates:

(11-12-2015) Preliminary Data Suggest Adenoviral Delivery Method Superior for Immune Stimulation and Control of Viral Load in the Absence of Antiretroviral Therapy (ART)

Other links:

LinkHIV Infection InformationLinkHIV around the worldLinkZFN Technology Platform

Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed

Description

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: Dec. 28, 2023
close
Search CRISPR Medicine