Overview

This trial is active, not recruiting.

Condition human immunodeficiency virus (hiv)
Treatments sb-728mr-t, cyclophosphamide
Phase phase 1/phase 2
Sponsor Sangamo Therapeutics
Start date August 2014
End date June 2018
Trial size 12 participants
Trial identifier NCT02225665, SB-728mR-1401

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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
sb-728mr-t
-SB-728mR-T infusions of 2 equal doses 14 days apart (total of up to 40 billion ZFN modified T-cells)
cyclophosphamide
- IV cyclophosphamide 1 g/m2 two days prior to the first SB-728mR-T infusion
(Experimental)
sb-728mr-t
- SB-728mR-T infusions of 3 equal doses 14 days apart (total of up to 40 billion ZFN modified T-cells)
cyclophosphamide
- IV cyclophosphamide 1 g/m2 two days prior to the first SB-728mR-T infusion

Primary Outcomes

Measure
Primary Outcome Measure
time frame: 12 months

Secondary Outcomes

Measure
Secondary Outcome Measure
time frame: 12 months
Secondary Outcome Measure
time frame: 12 months
Secondary Outcome Measure
time frame: 12 months

Eligibility Criteria

Male or female participants at least 18 years old.

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.

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in January 2017.
Information provided to ClinicalTrials.gov by Sangamo Therapeutics.