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Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients | Clinical Trials | Clareo Health

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Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients

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

NCT02500849•City of Hope Medical Center

View on ClinicalTrials.gov

Summary

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

Detailed Description

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.

Eligibility

Age

18 - 75 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Maximum age 75 years for cohort 1 and 65 years for cohort 2.
•HIV-1 R5 seropositive with no evidence of CXCR4-tropic virus.
•On cART with undetectable HIV-1 (\<20 gc/ml HIV-1 RNA) for at least 12 months prior to screening evaluations.
•CD4+ T-cell counts ≥200 cells/µL and ≤750 cells/µL.
•No psychosocial conditions that would hinder study compliance and follow-up.
•Absence of clinically significant cardiomyopathy, congestive heart failure.
•Secondary Eligibility Criteria (for registration):
•Complete G-CSF/Plerixafor mobilization of HSPC.
•Collect ≥7.5 x 10\^6 CD34+ cells/kg in two aphereses.
•The SB-728mR-HSPC product passed all release testing

Exclusion Criteria

•Use of AZT or maraviroc in the cART regimen.
•History of significant hematologic diseases such as leukemia, myelodysplasia, coagulopathy, and thromboembolism.
•Any AIDS-related opportunistic infection occurring within the past year such as tuberculosis, cryptococcosis and for which treatment has been unsuccessful as determined by the Principal Investigator.
•AIDS-related syndromes, infectious or otherwise, if perceived to cause excessive risk for morbidity post-HSPC infusion, as determined by the Principal Investigator.
•Patients with active HBV or HCV infection, i.e., HBV DNA and HCV RNA in blood, are excluded. Those with inactive, but past infection with HBV (positive HBV surface antigen or HBV surface antibody) or inactive HCV (positive HCV antibody), must have no cirrhosis, as determined by abdominal ultrasound with elastography.
•Active CMV retinitis or other active CMV-related organ dysfunction.
•CXCR4-tropic virus.
•Pregnant or nursing women.
•Any history of HIV-associated encephalopathy; dementia of any kind; seizures in the past 12 months; any perceived inability to directly provide informed consent.
•Participants may not be receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy. Participation in prior investigational drug or medical device study within the previous 45 days.
+4 more criteria

Locations (5)

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

Circle CARE Center, LLC

Norwalk, Connecticut, United States

Key Dates

Start Date

March 10, 2016

Primary Completion Date

August 24, 2026

Completion Date

August 24, 2026

Last Updated

October 9, 2025

Enrollment

ESTIMATED participants

Conditions

HIV

Interventions

SB-728mR-HSPC Infusion 3 days following busulfan conditioning

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 9, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients

Inclusion Criteria

Exclusion Criteria

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