Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT02500849)

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 to Evaluate the Feasibility, Safety and Engraftment of Zinc Finger Nuclease (ZFN) CCR5 Modified CD34+ Hematopoietic Stem/Progenitor Cells (SB-728mR-HSPC) in HIV-1 (R5) Infected Patients
Who:

Principal Investigator: Amrita Y. Krishnan, MD City of Hope Medical Center

Sponsor:

City of Hope Medical Center

Partners:

Sangamo Therapeutics Inc.

California Institute for Regenerative Medicine (CIRM)

Locations:

United States, California

City of Hope Medical Center, Duarte, California, United States, 91010

UCLA CARE Center, Los Angeles, California, United States, 90035

Mills Clinical Research, Los Angeles, California, United States, 90069

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

 

United States, Connecticut

Circle CARE Center, LLC, Norwalk, Connecticut, United States, 06850

 

Study start:
Mar. 10, 2016
Enrollment:
12 participants
Gene editing method:
ZFN- Zinc Finger Nucleases
Type of edit:
Gene knock-out
Gene:
C-C motif chemokine receptor 5 (CCR5)
Delivery method:
Electroporation - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT02500849
Safety updates:

(09-12-2016) Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells

Other links:

LinkHIV around the worldLinkHIV Infection InformationLinkPreclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells.

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

Status: Active not recruiting

Description

The purpose of the study is to evaluate the safety and feasibility of administering SB-728mR-HSPC after conditioning with busulfan.

The objective of the study is to evaluate the safety and feasibility of giving autologous SB-728mR-HSPC to HIV-1 (R5) infected patients who are being treated with cART and have undetectable virus but suboptimal CD4+ cell levels. To strengthen the possibility that CCR5-disrupted HSPCs engraft, patients will receive either a two- or three-day (Cohort 1 or Cohort 2) course of busulfan (dose targeting AUC of 4000 µM/day) before being infused with the genetically modified cells. At 9-12 months after SB-728mR-HSPC infusion, subjects who are aviremic with CD4 cell counts ≥600 cells/µL and have ≥1% CCR5-modified CD4 cells within the peripheral blood detected by pentamer PCR will undergo an ATI.

Last updated: Dec. 28, 2023
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