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Dose Escalation Study of Cyclophosphamide in HIV-Infected Subjects on HAART Receiving SB-728-T

Sponsor
Sangamo Therapeutics (Industry)
Overall Status
Completed
CT.gov ID
NCT01543152
Collaborator
(none)
26
Enrollment
11
Locations
5
Arms
67.2
Duration (Months)
2.4
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of the study is to evaluate the safety, tolerability and effect on HIV viral load, of escalating doses of cyclophosphamide administered 1 day prior to SB-728-T infusion.

Condition or Disease Intervention/Treatment Phase
  • Genetic: SB-728-T
  • Genetic: SB-728-T
  • Genetic: SB-728-T
  • Genetic: SB-728-T
  • Genetic: SB-728-T
 Phase 1/Phase 2

Detailed Description

The objectives of the study are to augment HIV-specific T-cells and to reverse or decrease the progressive destruction of CD4+ T-cells that leads to clinical AIDS. Levels of engraftment vary from negligible to about 10% of the CD4+ T-cells in the vascular compartment. Preliminary analyses of HAART TI suggest that an anti-HIV effect may correlate with the level of SB-728-T engraftment. Concurrently, non-myeloablative lymphodepletion with cyclophosphamide has been demonstrated to enhance engraftment of adoptively transferred T-cells through a variety of mechanisms. The study is being undertaken to increase SB-728-T engraftment through the administration of low non-myeloablative doses of cyclophosphamide.

Study Design

Study Type:
Interventional
Actual Enrollment :
26 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase I, Open-Label Study to Assess the Effect of Escalating Doses of Cyclophosphamide on the Engraftment of SB-728-T in Aviremic HIV-Infected Subjects on HAART
Study Start Date :
Dec 1, 2011
Actual Primary Completion Date :
Jul 7, 2017
Actual Study Completion Date :
Jul 7, 2017

Arms and Interventions

Arm Intervention/Treatment
Experimental: Cohort 1 - IV cyclophosphamide 200 mg

Genetic: SB-728-T
Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg
Other Names:
  • cyclophosphamide

    		•
    Experimental: Cohort 2 - IV cyclophosphamide 0.5 g/m2

    Genetic: SB-728-T
    Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2
    Other Names:
  • cyclophosphamide

    		•
    Experimental: Cohort 3 - IV cyclophosphamide 1.0 g/m2

    Genetic: SB-728-T
    Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2
    Other Names:
  • cyclophosphamide

    		•
    Experimental: Cohort 4 - IV cyclophosphamide 2.0 g/m2

    Genetic: SB-728-T
    Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2
    Other Names:
  • cyclophosphamide

    		•
    Experimental: Cohort 5 - IV cyclophosphamide 1.5 g/m2

    Genetic: SB-728-T
    Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    Other Names:
  • cyclophosphamide

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Treatment-emergent Adverse Events [28 days after the SB-728-T infusion of the last subject in each Cohort and up to 12 months]

      Number of Participants with Treatment related Adverse Events in subjects who received any portion of the SB-728-T infusion

    Secondary Outcome Measures

    1. Effect of Escalating Doses of Cyclophosphamide on SB-728-T Engraftment as Measured by CCR5 Modified CD4 Cells in Blood. [Up to 12 months after the last SB-728-T infusion]

      Effect of repeat doses of SB-728-T on engraftment following cyclophosphamide conditioning as measured by CCR5 Modified CD4 Cells in blood at Month 12.

    2. Effect of SB-728-T on Plasma HIV-1 RNA Levels Following HAART Interruption [Up to 12 months after the last SB-728-T infusion]

      Effect of SB-728-T on plasma HIV-1 RNA levels following HAART interruption. The unit is log copies/mL, except for the Cohort 1, the unit is " copies/mL". Cohort 1 mean and SD are 0. All 3 subjects had NO HIV-1 RNA DETECTED.

    3. Change From Baseline to Month 12 in CD4+ T-cell Counts in Peripheral Blood After Repeat Treatments With SB-728-T. (i.e. Month 12 Value - Baseline Value) [Up to 12 months after the last SB-728-T infusion]

      Change from baseline to month 12 in CD4+ T-cell counts in peripheral blood after repeat treatments with SB-728-T. (i.e. month 12 value - baseline value)

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Male or female, 18 years of age or older with documented HIV diagnosis within 10 years of screening.

    • Must be willing to comply with study-mandated evaluations; including discontinuation of current antiretroviral therapy during the treatment interruption.

    • Must have received at least 6 months of continuous HAART therapy and have had undetectable VLs for the preceding 3 months.

    • On stable antiretroviral medication (no changes to treatment within 4 weeks of screening.

    • CD4+ T-cell count ≥500 cells/µL.

    • Undetectable HIV-1 RNA obtained at screening.

    • ANC ≥2500/µL

    • Platelet count ≥200,000/µL

    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 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.

    • Subjects who are currently taking maraviroc or have received maraviroc within 6 months prior to screening.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 UCLA Care Center Los Angeles California United States 90035
    2 Quest Clinical Research San Francisco California United States 94115
    3 Circle CARE Center, LLC Norwalk Connecticut United States 06850
    4 Orlando Immunology Center Orlando Florida United States 32803
    5 Central West Clinical Research, Inc. Saint Louis Missouri United States 63108
    6 Southwest CARE Center Santa Fe New Mexico United States 87505
    7 Ricky K Hsu, MD, PC New York New York United States 10011
    8 Central Texas Clinical Research Austin Texas United States 78705
    9 North Texas Infectious Diseases Consultants Dallas Texas United States 75246
    10 Gordon Crofoot, MD, PA Houston Texas United States 77098
    11 Clinical Research Puerto Rico San Juan Puerto Rico 00909

    Sponsors and Collaborators

    • Sangamo Therapeutics

    Investigators

    None specified.

    Study Documents (Full-Text)

    More Information

    Publications

    None provided.
    Responsible Party:
    Sangamo Therapeutics
    ClinicalTrials.gov Identifier:
    NCT01543152
    Other Study ID Numbers:
    • SB-728-1101
    First Posted:
    Mar 2, 2012
    Last Update Posted:
    May 24, 2021
    Last Verified:
    Oct 1, 2017
    Keywords provided by Sangamo Therapeutics
    HIV
    autologous cell therapy
    cyclophosphamide
    Additional relevant MeSH terms:
    Cyclophosphamide

    Study Results

    Participant Flow

    Recruitment Details
    Pre-assignment Detail
    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    Period Title: Overall Study
    STARTED 3 6 11 3 3
    COMPLETED 3 4 11 3 3
    NOT COMPLETED 0 2 0 0 0

    Baseline Characteristics

    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2 Total
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2 Total of all reporting groups
    Overall Participants 3 6 11 3 3 26
    Age (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    44.7
    (8.08)
    43.2
    (6.11)
    41.5
    (12.13)
    35.3
    (10.21)
    50.0
    (1.00)
    42.5
    (9.70)
    Sex: Female, Male (Count of Participants)
    Female
    0
    0%
    1
    16.7%
    1
    9.1%
    0
    0%
    0
    0%
    2
    7.7%
    Male
    3
    100%
    5
    83.3%
    10
    90.9%
    3
    100%
    3
    100%
    24
    92.3%
    Ethnicity (NIH/OMB) (Count of Participants)
    Hispanic or Latino
    0
    0%
    2
    33.3%
    3
    27.3%
    0
    0%
    1
    33.3%
    6
    23.1%
    Not Hispanic or Latino
    3
    100%
    4
    66.7%
    8
    72.7%
    3
    100%
    2
    66.7%
    20
    76.9%
    Unknown or Not Reported
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Race (NIH/OMB) (Count of Participants)
    American Indian or Alaska Native
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Asian
    0
    0%
    0
    0%
    1
    9.1%
    1
    33.3%
    0
    0%
    2
    7.7%
    Native Hawaiian or Other Pacific Islander
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Black or African American
    1
    33.3%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    1
    3.8%
    White
    2
    66.7%
    6
    100%
    9
    81.8%
    2
    66.7%
    2
    66.7%
    21
    80.8%
    More than one race
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    0
    0%
    Unknown or Not Reported
    0
    0%
    0
    0%
    1
    9.1%
    0
    0%
    1
    33.3%
    2
    7.7%

    Outcome Measures

    1. Primary Outcome
    Title Treatment-emergent Adverse Events
    Description Number of Participants with Treatment related Adverse Events in subjects who received any portion of the SB-728-T infusion
    Time Frame 28 days after the SB-728-T infusion of the last subject in each Cohort and up to 12 months

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    Measure Participants 3 6 11 3 3
    Number [participants]
    3
    100%
    6
    100%
    11
    100%
    3
    100%
    3
    100%
    2. Secondary Outcome
    Title Effect of Escalating Doses of Cyclophosphamide on SB-728-T Engraftment as Measured by CCR5 Modified CD4 Cells in Blood.
    Description Effect of repeat doses of SB-728-T on engraftment following cyclophosphamide conditioning as measured by CCR5 Modified CD4 Cells in blood at Month 12.
    Time Frame Up to 12 months after the last SB-728-T infusion

    Outcome Measure Data

    Analysis Population Description
    Safety Analysis Set
    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    Measure Participants 3 4 10 3 3
    Mean (Standard Deviation) [cells 10^9/L]
    0.038
    (0.0409)
    0.061
    (0.0447)
    0.143
    (0.1055)
    0.085
    (0.0197)
    0.079
    (0.0155)
    3. Secondary Outcome
    Title Effect of SB-728-T on Plasma HIV-1 RNA Levels Following HAART Interruption
    Description Effect of SB-728-T on plasma HIV-1 RNA levels following HAART interruption. The unit is log copies/mL, except for the Cohort 1, the unit is " copies/mL". Cohort 1 mean and SD are 0. All 3 subjects had NO HIV-1 RNA DETECTED.
    Time Frame Up to 12 months after the last SB-728-T infusion

    Outcome Measure Data

    Analysis Population Description
    Safety Analysis Set
    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    Measure Participants 3 4 11 3 3
    Mean (Standard Deviation) [log copies/mL]
    0
    (0)
    0.250
    (0.5000)
    1.537
    (1.3955)
    0.667
    (0.5774)
    3.442
    (1.1545)
    4. Secondary Outcome
    Title Change From Baseline to Month 12 in CD4+ T-cell Counts in Peripheral Blood After Repeat Treatments With SB-728-T. (i.e. Month 12 Value - Baseline Value)
    Description Change from baseline to month 12 in CD4+ T-cell counts in peripheral blood after repeat treatments with SB-728-T. (i.e. month 12 value - baseline value)
    Time Frame Up to 12 months after the last SB-728-T infusion

    Outcome Measure Data

    Analysis Population Description
    Safety Analysis Set
    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    Measure Participants 3 4 10 3 3
    Mean (Standard Deviation) [cells 10^9/L]
    0.064
    (0.1309)
    0.149
    (0.3736)
    -0.043
    (0.1712)
    0.153
    (0.3247)
    0.099
    (0.3279)

    Adverse Events

    Time Frame 1 year
    Adverse Event Reporting Description
    Arm/Group Title Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Arm/Group Description SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells 1 day following IV cyclophosphamide 200 mg SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 0.5 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 2.0 g/m2 SB-728-T: Infusion will be 5 to 30 billion ZFN modified CD4+ T cells up to 3 days following IV cyclophosphamide 1.5 g/m2
    All Cause Mortality
    Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 0/3 (0%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Serious Adverse Events
    Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Infections and infestations
    Cellulitis staphylococcal 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Psychiatric disorders
    Substance abuse 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Other (Not Including Serious) Adverse Events
    Cohort 1 - IV Cyclophosphamide 200 mg Cohort 2 - IV Cyclophosphamide 0.5 g/m2 Cohort 3 - IV Cyclophosphamide 1.0 g/m2 Cohort 4 - IV Cyclophosphamide 2.0 g/m2 Cohort 5 - IV Cyclophosphamide 1.5 g/m2
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 3/3 (100%) 6/6 (100%) 11/11 (100%) 3/3 (100%) 3/3 (100%)
    Blood and lymphatic system disorders
    Neutropenia 0/3 (0%) 0/6 (0%) 0/11 (0%) 3/3 (100%) 2/3 (66.7%)
    Lymphadenopathy 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Ear and labyrinth disorders
    Tinnitus 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Endocrine disorders
    Hypogonadism 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Eye disorders
    Scintillating scotoma 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Vision blurred 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Gastrointestinal disorders
    Nausea 2/3 (66.7%) 4/6 (66.7%) 4/11 (36.4%) 1/3 (33.3%) 1/3 (33.3%)
    Vomiting 1/3 (33.3%) 2/6 (33.3%) 2/11 (18.2%) 1/3 (33.3%) 0/3 (0%)
    Diarrhoea 0/3 (0%) 1/6 (16.7%) 2/11 (18.2%) 1/3 (33.3%) 1/3 (33.3%)
    Abdominal discomfort 1/3 (33.3%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Abdominal pain 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Anal skin tags 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Aphthous stomatitis 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Constipation 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Flatulence 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    General disorders
    Chills 2/3 (66.7%) 4/6 (66.7%) 3/11 (27.3%) 3/3 (100%) 0/3 (0%)
    Pyrexia 1/3 (33.3%) 2/6 (33.3%) 6/11 (54.5%) 1/3 (33.3%) 0/3 (0%)
    Fatigue 1/3 (33.3%) 2/6 (33.3%) 3/11 (27.3%) 0/3 (0%) 2/3 (66.7%)
    Pain 1/3 (33.3%) 1/6 (16.7%) 2/11 (18.2%) 1/3 (33.3%) 0/3 (0%)
    Asthenia 0/3 (0%) 1/6 (16.7%) 1/11 (9.1%) 1/3 (33.3%) 0/3 (0%)
    Malaise 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 1/3 (33.3%)
    Chest pain 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Temperature intolerance 0/3 (0%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 1/3 (33.3%)
    Hepatobiliary disorders
    Hepatotoxicity 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Immune system disorders
    Food allergy 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Seasonal allergy 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Infections and infestations
    Upper respiratory tract infection 2/3 (66.7%) 2/6 (33.3%) 3/11 (27.3%) 2/3 (66.7%) 0/3 (0%)
    Sinusitis 0/3 (0%) 1/6 (16.7%) 2/11 (18.2%) 1/3 (33.3%) 1/3 (33.3%)
    Furuncle 1/3 (33.3%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Nasopharyngitis 0/3 (0%) 1/6 (16.7%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Balanitis candida 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Bronchitis 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Cellulitis staphylococcal 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Chlamydial infection 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Fungal skin infection 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Gonorrhoea 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Influenza 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Laryngitis 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Paronychia 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Pharyngitis streptococcal 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Syphilis 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Tinea pedis 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Urinary tract infection 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Injury, poisoning and procedural complications
    Exposure to communicable disease 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Laceration 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Muscle strain 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Procedural pain 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Investigations
    Alanine aminotransferase increased 0/3 (0%) 0/6 (0%) 2/11 (18.2%) 0/3 (0%) 0/3 (0%)
    Aspartate aminotransferase increased 0/3 (0%) 0/6 (0%) 2/11 (18.2%) 0/3 (0%) 0/3 (0%)
    Blood phosphorus decreased 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Blood pressure decreased 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Metabolism and nutrition disorders
    Decreased appetite 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Dehydration 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Hypertriglyceridaemia 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Hyponatraemia 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Musculoskeletal and connective tissue disorders
    Arthralgia 1/3 (33.3%) 1/6 (16.7%) 1/11 (9.1%) 1/3 (33.3%) 0/3 (0%)
    Back pain 1/3 (33.3%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Bone pain 0/3 (0%) 0/6 (0%) 0/11 (0%) 2/3 (66.7%) 0/3 (0%)
    Muscle spasms 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Neck pain 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Anogenital warts 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Fibrous histiocytoma 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Nervous system disorder 2/3 (66.7%) 6/6 (100%) 4/11 (36.4%) 1/3 (33.3%) 2/3 (66.7%)
    Headache 2/3 (66.7%) 5/6 (83.3%) 4/11 (36.4%) 1/3 (33.3%) 1/3 (33.3%)
    Migraine 0/3 (0%) 1/6 (16.7%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Dizziness 0/3 (0%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 1/3 (33.3%)
    Psychiatric disorders 0/3 (0%) 1/6 (16.7%) 1/11 (9.1%) 1/3 (33.3%) 0/3 (0%)
    Agitation 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Insomnia 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Substance abuse 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Renal and urinary disorders
    Urinary hesitation 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Respiratory, thoracic and mediastinal disorders 0/3 (0%) 1/6 (16.7%) 4/11 (36.4%) 1/3 (33.3%) 1/3 (33.3%)
    Cough 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 1/3 (33.3%) 1/3 (33.3%)
    Sinus congestion 0/3 (0%) 1/6 (16.7%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Oropharyngeal pain 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Respiratory tract congestion 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Respiratory, thoracic and mediastinal disorders
    Skin odour abnormal 1/3 (33.3%) 2/6 (33.3%) 5/11 (45.5%) 2/3 (66.7%) 1/3 (33.3%)
    Skin and subcutaneous tissue disorders
    Alopecia 0/3 (0%) 0/6 (0%) 3/11 (27.3%) 3/3 (100%) 3/3 (100%)
    Hyperhidrosis 0/3 (0%) 1/6 (16.7%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Night sweats 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 1/3 (33.3%)
    Acne 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Actinic keratosis 0/3 (0%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 1/3 (33.3%)
    Alopecia areata 0/3 (0%) 0/6 (0%) 1/11 (9.1%) 0/3 (0%) 0/3 (0%)
    Dry skin 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Hair colour changes 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Pityriasis rubra pilaris 0/3 (0%) 0/6 (0%) 0/11 (0%) 1/3 (33.3%) 0/3 (0%)
    Pruritus generalised 0/3 (0%) 1/6 (16.7%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Vascular disorders
    Hypertension 0/3 (0%) 0/6 (0%) 2/11 (18.2%) 0/3 (0%) 0/3 (0%)
    Pallor 0/3 (0%) 2/6 (33.3%) 0/11 (0%) 0/3 (0%) 0/3 (0%)
    Venous thrombosis 1/3 (33.3%) 0/6 (0%) 0/11 (0%) 0/3 (0%) 0/3 (0%)

    Limitations/Caveats

    [Not Specified]

    More Information

    Certain Agreements

    Principal Investigators are NOT employed by the organization sponsoring the study.

    All proposed written materials related to the study or an outline of any proposed oral presentations, shall be submitted to Sangamo for approval at least 45 days prior to submission of materials for publication or any oral disclosure to a third party. If Sangamo determines that a description of patentable subject matter is contained in such written material or outline, it shall notify the clinical site within 1 month after receipt and Sangamo will have an additional 90 days for review.

    Results Point of Contact

    Name/Title Medical Monitor
    Organization Sangamo Therapeutics
    Phone 510-970-6000
    Email clinicaltrials@sangamo.com
    Responsible Party:
    Sangamo Therapeutics
    ClinicalTrials.gov Identifier:
    NCT01543152
    Other Study ID Numbers:
    • SB-728-1101
    First Posted:
    Mar 2, 2012
    Last Update Posted:
    May 24, 2021
    Last Verified:
    Oct 1, 2017