Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT03617198)

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 Pilot Study of T Cells Genetically Modified by Zinc Finger Nucleases SB-728mR and CD4 Chimeric Antigen Receptor in HIV-infected Subjects
Who:

Principal Investigator: Pablo Tebas, MD University of Pennsylvania

 

Sponsor:

University of Pennsylvania

Partners:
Locations:

United States, Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Study start:
Jul. 31, 2019
Enrollment:
12 participants
Gene editing method:
ZFN- Zinc Finger Nucleases
Type of edit:
Gene disruption
Gene:
CD4 molecule CAR and C-C motif chemokine receptor 5 (CCR5)
Delivery method:
Adeno-associated virus (AAV) - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT03617198
Other links:

LinkHIV infection informationLinkHIV around the worldLinkZFN Technology PlatformLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem Qasim

Note:
Infusion of autologous T cells genetically modified to express a CD4 chimeric antigen receptor while also having zinc finger nuclease-mediated disruption of the CCR5 gene
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting

Description

This research study is being carried out to study a new way to possibly treat HIV. As part of this study, doctors will take some of your own white blood cells, called T-cells, and modify them so that they can identify and target your HIV cells. The purpose of the study is to evaluate the safety of these modified T cells and determine whether they have any effect on HIV infection.

Biological: CD4 CAR+CCR5 ZFN T-cells

A dual cohort, open-label, randomized study of the safety and tolerability of a single infusion of autologous T cells genetically modified to express a CD4 chimeric antigen receptor while also having zinc finger nuclease-mediated disruption of the CCR5 gene

Study Arm:

  • Experimental:Cohort 1 subjects will begin treatment interruption approximately 24 hours after they receive the modified T-cells. All other study procedures are the same as Cohort 2.

Intervention: Biological: CD4 CAR+CCR5 ZFN T-cells

  • Experimental:Cohort 2 subjects will begin treatment interruption approximately 8 weeks after they receive the modified T-cells. All other study procedures are the same as Cohort 1.

    Intervention: Biological: CD4 CAR+CCR5 ZFN T-cells

Last updated: Dec. 28, 2023
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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