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Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects

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ClinicalTrials.gov Identifier: NCT01252641
Recruitment Status : Completed
First Posted : December 3, 2010
Last Update Posted : September 20, 2019
Information provided by (Responsible Party):
Sangamo Therapeutics

Tracking Information
First Submitted Date  ICMJE November 29, 2010
First Posted Date  ICMJE December 3, 2010
Last Update Posted Date September 20, 2019
Study Start Date  ICMJE November 2010
Actual Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 24, 2011)
Evaluate the safety and tolerability of SB-728-T cells infusion in HIV-1 positive subjects [ Time Frame: 12 months ]
Evaluate the safety and tolerability of a single infusion of 5-30 billion SB-728-T cells in HIV-1 positive subjects
Original Primary Outcome Measures  ICMJE
 (submitted: December 1, 2010)
Treatment related adverse events [ Time Frame: 12 months ]
Number of participants who report adverse events as a measure of safety and tolerability
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 1, 2010)
  • Evaluate persistence of HIV as measured by HIV-1 RNA, [ Time Frame: 12 months ]
  • Change in CD4+ T-cell count [ Time Frame: 12 months ]
  • Persistence of SB-728-T in the peripheral blood [ Time Frame: 12 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects
Official Title  ICMJE A Phase 1/2, Open Label, Single Infusion Study of Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases (SB-728-T) in HIV Infected Subjects
Brief Summary

This research study is being carried out to study a new way to possibly treat human immunodeficiency virus (HIV). The agent is called SB-728-T which are CD4+ T-cells obtained from an individual that are genetically modified at the CCR5 gene by Zinc Finger Nucleases. The CCR5 gene is required for certain types of HIV to enter into and infect T-cells. T cells are one of the white blood cells used by the body to fight HIV. The most important of these are called "CD4+ T-cells"

Some people are born without the CCR5 gene on their T-Cells. These people remain healthy and are resistant to infection with HIV. Other people have a low number of CCR5 genes on their T-cells and their HIV disease is less severe and is slower to cause disease (AIDS).

The purpose of this research study is to find out whether SB-728-T is safe to give to humans and find out how this affects HIV.

Detailed Description

Laboratory studies have shown that when CD4+ T-cells are modified with Zinc Finger Nucleases SB-728, HIV is prevented from killing the CD4+ T-cells. On the basis of these laboratory results, there is the potential that this may work in humans infected with HIV and improve their immune system by allowing their CD4+ T-cells to survive longer.

The new treatment to be studied will involve removing white blood cells from the blood that contain CD4+ T-cells. The extracted CD4+ T-cells are then genetically modified by the Zinc finger Nucleases to be resistant to infection by removing the CCR5 gene from the surface of the CD4+ T-cell where HIV enters the cell. Additional genetically modified cells are manufactured and then re-infused back into the individual. Researchers hope that these genetically modified cells will be resistant to infection by HIV and will be able to reproduce additional resistant CD4+ T-cells in your body.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • HIV
  • HIV Infection
Intervention  ICMJE Biological: SB-728-T
Each infusion will be 5-30 billion modified CD4+ T-cells
Study Arms  ICMJE Experimental: SB-728-T
Subjects will receive one intravenous infusion of SB-728-T
Intervention: Biological: SB-728-T
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 20, 2014)
Original Estimated Enrollment  ICMJE
 (submitted: December 1, 2010)
Actual Study Completion Date  ICMJE May 2015
Actual Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Documented HIV infection
  • CD4+ T-cell count >500 cell per millimeter cubed (cells/mm3)
  • CD4+ T-cell nadir of >400 cells/mm3
  • HIV viral load >1,000 copies per milliliter (mL)

Exclusion Criteria:

  • Any viral hepatitis
  • Acute HIV infection
  • HIV viral load >1,000,000 copies/mL
  • Active or recent (prior 6 months) AIDS defining complication
  • Any HIV medications within the past 12 weeks
  • Cancer or malignancy that has not been in remission for at least 5 years with the exception of successfully treated basal cell carcinoma of the skin
  • Current diagnosis of NYHA grade 3 or 4 congestive heart failure or uncontrolled angina or arrhythmias
  • History of bleeding problems
  • Use of chronic steroids in past 30 days
  • Pregnant or breast feeding
  • Active drug or alcohol abuse
  • Serious illness in past 30 days
  • Currently participating in another clinical trail or any prior gene therapy
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT01252641
Other Study ID Numbers  ICMJE SB-728-1002
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Sangamo Therapeutics
Original Responsible Party Sangamo BioSciences, Inc., Sangamo BioSciences, inc.
Current Study Sponsor  ICMJE Sangamo Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Winson Tang, M.D. Sangamo BioSciences, Inc.
PRS Account Sangamo Therapeutics
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP