Clinical Trial

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

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 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

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
12 participants
Gene editing method:
ZFN- Zinc Finger Nucleases
Type of edit:
Gene knock-out
C-C motif chemokine receptor 5 (CCR5)
Delivery method:
Electroporation - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting


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: Jun. 13, 2023
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