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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
City of Hope Medical Center
Duarte, California, United States
UCLA CARE Center
Los Angeles, California, United States
Mills Clinical Research
Los Angeles, California, United States
Quest Clinical Research
San Francisco, California, United States
Toxicity in subjects who received SB-728mR-HSPC after each busulfan dose level
Time frame: 18 months
Number of CD34+ HSPC collected, gene modified, and released throughout the manufacturing process
Time frame: Approximately first 1-2 months on study
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Circle CARE Center, LLC
Norwalk, Connecticut, United States