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Repeat Doses of SB-728mR-T After Cyclophosphamide Conditioning in HIV-Infected Subjects on HAART

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ClinicalTrials.gov Identifier: NCT02225665
Recruitment Status : Completed
First Posted : August 26, 2014
Results First Posted : February 9, 2021
Last Update Posted : March 19, 2021
Information provided by (Responsible Party):
Sangamo Therapeutics

Brief Summary:

The purpose of this study is to evaluate the safety and tolerability of repeat doses of T-cell immunotherapy (SB-728mR-T) following cyclophosphamide conditioning.

CCR5 is a major co-receptor for HIV entry into T-cells. Disruption of CCR5 by zinc finger nuclease (SB-728mR), blocks HIV entry into the T-cells, therefore, protects the T-cells from HIV infection. Safety (primary outcome) and anti-viral effect (secondary outcome) of zinc finger nuclease-mediated CCR5 disrupted autologous T-cells (SB-728mR-T) will be evaluated in the study.

Condition or disease Intervention/treatment Phase
Human Immunodeficiency Virus (HIV) Genetic: SB-728mR-T Drug: Cyclophosphamide Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Open-Label Study to Assess the Safety and Tolerability of Repeat Doses of Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects Following Cyclophosphamide Conditioning
Actual Study Start Date : August 2014
Actual Primary Completion Date : June 2018
Actual Study Completion Date : June 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: HIV/AIDS

Arm Intervention/treatment
Experimental: Cohort 1 Genetic: SB-728mR-T
-SB-728mR-T infusions of 2 equal doses 14 days apart (total of up to 40 billion ZFN modified T-cells)

Drug: Cyclophosphamide
- IV cyclophosphamide 1 g/m2 two days prior to the first SB-728mR-T infusion

Experimental: Cohort 2 Genetic: SB-728mR-T
- SB-728mR-T infusions of 3 equal doses 14 days apart (total of up to 40 billion ZFN modified T-cells)

Drug: Cyclophosphamide
- IV cyclophosphamide 1 g/m2 two days prior to the first SB-728mR-T infusion

Primary Outcome Measures :
  1. Primary Outcome Measure [ Time Frame: 12 months ]
    Number of Participants with Treatment related Adverse Events in subjects who received any portion of the SB-728mR-T infusion

Secondary Outcome Measures :
  1. Secondary Outcome Measure [ Time Frame: 12 months ]
    Effect of repeat doses of SB-728mR-T on engraftment following cyclophosphamide conditioning as measured by CCR5 Modified CD4 Cells at Month 12.

  2. Secondary Outcome Measure [ Time Frame: 12 months ]
    Effect of SB-728mR-T on plasma HIV-1 RNA levels following HAART interruption

  3. Secondary Outcome Measure [ Time Frame: Baseline and 12 months ]
    Change from baseline to month 12 in CD4+ T-cell counts in peripheral blood after repeat treatments with SB-728mR-T. (i.e. month 12 value - baseline value)

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female, 18 years of age or older with documented HIV diagnosis.
  • Must be willing to comply with study-mandated evaluations; including discontinuation of current antiretroviral therapy during the treatment interruption.
  • Initiated HAART therapy within (≤) 1 year of HIV diagnosis or suspected infection.
  • Undetectable HIV-1 RNA for at least 2 months prior to screening and at screening.
  • CD4+ T-cell count ≥500 cells/µL.
  • Absolute neutrophil count (ANC) ≥ 2500/mm3.
  • Platelet count ≥ 200,000/mm3.

Exclusion Criteria:

  • Acute or chronic hepatitis B or hepatitis C infection.
  • Active or recent (in prior 6 months) AIDS defining complication.
  • Any cancer or malignancy within the past 5 years, with the exception of successfully treated basal cell or squamous cell carcinoma of the skin or low grade (0 or 1) anal or cervical dysplasia.
  • Current diagnosis of NYHA grade 3 or 4 CHF, uncontrolled angina or uncontrolled arrhythmias.
  • History or any features on physical examination indicative of a bleeding diathesis.
  • Received HIV experimental vaccine within 6 months prior to screening, or any previous gene therapy using an integrating vector.
  • Use of chronic corticosteroids, hydroxyurea, or immunomodulating agents within 30 days prior to screening.
  • Use of Aspirin, dipyridamole, warfarin or any other medication that is likely to affect platelet function or other aspects of blood coagulation during the 2 week period prior to leukapheresis.
  • Currently participating in another clinical trial or participation in such a trial within 30 days prior to screening visit.
  • Currently taking maraviroc or have received maraviroc within 6 months prior to screening.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02225665

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United States, California
San Francisco, California, United States, 94115
United States, Connecticut
Norwalk, Connecticut, United States, 06850
United States, Florida
Orlando, Florida, United States, 32803
United States, Texas
Austin, Texas, United States, 78705
Dallas, Texas, United States, 75246
Sponsors and Collaborators
Sangamo Therapeutics
  Study Documents (Full-Text)

Documents provided by Sangamo Therapeutics:
Study Protocol: Protocol Amendment 2  [PDF] October 10, 2014
Statistical Analysis Plan  [PDF] December 21, 2020

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Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT02225665    
Other Study ID Numbers: SB-728mR-1401
First Posted: August 26, 2014    Key Record Dates
Results First Posted: February 9, 2021
Last Update Posted: March 19, 2021
Last Verified: December 2020
Additional relevant MeSH terms:
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Acquired Immunodeficiency Syndrome
HIV Infections
Immunologic Deficiency Syndromes
Immune System Diseases
Blood-Borne Infections
Communicable Diseases
Sexually Transmitted Diseases, Viral
Sexually Transmitted Diseases
Lentivirus Infections
Retroviridae Infections
RNA Virus Infections
Virus Diseases
Slow Virus Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists