Clinical trial NCT02225665: Repeat Doses of SB-728mR-T After Cyclophosphamide Conditioning in HIV-Infected Subjects on HAART
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Human Immunodeficiency Virus (HIV)
Lead sponsor: Sangamo Therapeutics
Sponsor class: INDUSTRY
First posted: 2014-08-26
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Primary Outcome Measure
timeFrame: 12 months
Secondary outcomes:
measure: Secondary Outcome Measure
timeFrame: 12 months
measure: Secondary Outcome Measure
timeFrame: 12 months
measure: Secondary Outcome Measure
timeFrame: Baseline and 12 months
Other outcomes:
Locations:
city: San Francisco
country: United States
state: California
city: Norwalk
country: United States
state: Connecticut
city: Orlando
country: United States
state: Florida
city: Austin
country: United States
state: Texas
city: Dallas
country: United States
state: Texas
Study officials:
References: