Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT01252641)

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 1/2, Open Label, Single Infusion Study of Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases (SB-728-T) in HIV Infected Subjects
Who:

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

Sponsor:

Sangamo Therapeutics Inc.

Partners:
Locations:

United States, California

UCLA CARE Center, 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, Florida

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

Study start:
Nov. 1, 2010
Enrollment:
21 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/study/NCT01252641
Safety updates:

(11-12-2015) Sangamo BioSciences Presents Phase 2 Clinical Data From Two SB-728-T HIV Studies

(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

IndicatorIndicator
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 human immunodeficiency virus (HIV). The agent is called SB-728-T which are CD4+ T-cells obtained from an individual that are genetically modified at the CCR5 gene by Zinc Finger Nucleases. The CCR5 gene is required for certain types of HIV to enter into and infect 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 the CCR5 gene on their T-Cells. These people remain healthy and are resistant to infection with HIV. Other people have a low number of CCR5 genes on their T-cells and their HIV disease is less severe and is slower to cause disease (AIDS).

The purpose of this research study is to find out whether SB-728-T is safe to give to humans and find out how this affects HIV.

Laboratory studies have shown that when CD4+ T-cells are modified with Zinc Finger Nucleases SB-728, HIV is prevented from killing the CD4+ T-cells. On the basis of these laboratory results, there is the potential that this may work in humans infected with HIV and improve their immune system by allowing their CD4+ T-cells to survive longer

The new treatment to be studied will involve removing white blood cells from the blood that contain CD4+ T-cells. The extracted CD4+ T-cells are then genetically modified by the Zinc finger Nucleases to be resistant to infection 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 the individual. 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.

Last updated: Dec. 28, 2023
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