<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT01734850</url>
  </required_header>
  <id_info>
    <org_study_id>CAL-USA-11</org_study_id>
    <nct_id>NCT01734850</nct_id>
  </id_info>
  <brief_title>Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection</brief_title>
  <official_title>An Adaptive Phase I/II Study of the Safety of CD4+ T Lymphocytes and CD34+ Hematopoietic Stem/Progenitor Cells Transduced With LVsh5/C46, a Dual Anti-HIV Gene Transfer Construct, With and Without Conditioning With Busulfan in HIV-1 Infected Adults Previously Exposed to ART</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Calimmune, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Calimmune, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is an early phase research study looking at whether an experimental gene transfer,&#xD;
      LVsh5/C46 (also known as Cal-1), is safe and if it can protect the immune system from the&#xD;
      effects of HIV without the use of antiretroviral drugs.&#xD;
&#xD;
      Cal-1 is an experimental gene transfer agent designed to inhibit HIV infection through 2&#xD;
      active parts:&#xD;
&#xD;
        1. Removing a protein named CCR5 from bone marrow and white blood cells&#xD;
&#xD;
        2. Producing a protein named C46 on bone marrow and white blood cells&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      It is estimated that 33 million individuals are currently infected with HIV. HIV/AIDS is a&#xD;
      disease that impairs immune function, primarily by decreasing CD4+ T lymphocytes. The&#xD;
      progression can be contained by daily dosing with antiretroviral therapy (ART) but there are&#xD;
      side effects that can be treatment limiting, and the development of HIV drug resistance can&#xD;
      force the physician to modify the ART regimen. There are no effective vaccines currently&#xD;
      available for HIV.&#xD;
&#xD;
      LVsh5/C46 (also known as Cal-1) is a dual therapeutic, self-inactivating lentiviral vector&#xD;
      that encodes for both a short hairpin RNA against the HIV-1 co-receptor CCR5 (sh5) and a&#xD;
      HIV-1 fusion inhibitor, C46 and inhibits two processes required for HIV-1 infection:&#xD;
&#xD;
        1. Binding of the virus to the cellular CCR5 co-receptor and&#xD;
&#xD;
        2. Fusion of the virus with the host cell&#xD;
&#xD;
      The rationale is that Cal-1 introduced into hematopoietic progenitor/stem cells (HSPC) and&#xD;
      mature CD4+ T lymphocytes will protect these cells and their progeny cells from HIV-1&#xD;
      infection and its pathogenic sequelae. This may provide a continuous means of controlling&#xD;
      HIV-1 after a single or infrequent dose(s), thereby decreasing or delaying (partially or&#xD;
      completely) the need for antiretroviral drug therapy.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 2013</start_date>
  <completion_date type="Actual">November 2017</completion_date>
  <primary_completion_date type="Actual">September 2017</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Severe and Life-threatening Adverse Events (AEs)</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Severe or Life-threatening AEs Related to CSL202</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With the Presence of Replication-competent Retrovirus</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Predominant Integration Site Analysis</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>Vector Integration Site Analysis performed only when Cal-1 Marking is &gt;= 1%.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean Cell Dose for CD4+ Cells (Ttn)</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean Cell Dose for CD34+ Cells (HSPCtn)</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Total Area Under the Curve (AUC) for Busulfan</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>Cohort 3: Total AUC = first dose AUC value + second dose AUC value</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percent Cal-1 Marking in Peripheral Blood</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>Samples were collected via endoscopic biopsy from the sigmoid colon: 10-15 cm from the anal margin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cal-1 Marking in GALT (25-35 cm)</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>Samples were collected via endoscopic biopsy from the sigmoid colon: 25-35 cm from the anal margin</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cal-1 Marking in Bone Marrow</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cal-1 C46 Expression in Peripheral Blood</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>C46 relative expression will be analyzed by reverse transcriptase-quantitative polymerase chain reaction (RT-qPCR) and normalized to the expression of β2-microglobulin (β2M) mRNA</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cal-1 sh5 Expression in Peripheral Blood</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>sh5 relative expression will be analyzed by RT-qPCR and normalized to the expression of RNU38B microRNA</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement</measure>
    <time_frame>Up to 48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With HIV-1 Tropism Shift</measure>
    <time_frame>Up to 48 weeks</time_frame>
    <description>Shift from R5 to X4 or dual/mixed tropism</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">13</enrollment>
  <condition>Human Immunodeficiency Virus</condition>
  <arm_group>
    <arm_group_label>No busulfan pre-conditioning</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes without busulfan preconditioning</description>
  </arm_group>
  <arm_group>
    <arm_group_label>1 x 4mg/kg busulfan preconditioning</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes, with single 4mg/kg busulfan dose administered as pre-conditioning for transplant</description>
  </arm_group>
  <arm_group>
    <arm_group_label>2 x 4mg/kg busulfan pre-conditioning</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes, with two 4mg/kg busulfan doses administered as pre-conditioning for transplant</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Busulfan</intervention_name>
    <description>Intravenous busulfan</description>
    <arm_group_label>1 x 4mg/kg busulfan preconditioning</arm_group_label>
    <arm_group_label>2 x 4mg/kg busulfan pre-conditioning</arm_group_label>
    <other_name>Busulfex</other_name>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Cal-1 modified HSPC</intervention_name>
    <description>Hematopoietic progenitor/stem cells (HSPC) modified with LVsh5/C46 (Cal-1)</description>
    <arm_group_label>1 x 4mg/kg busulfan preconditioning</arm_group_label>
    <arm_group_label>2 x 4mg/kg busulfan pre-conditioning</arm_group_label>
    <arm_group_label>No busulfan pre-conditioning</arm_group_label>
    <other_name>LVsh5/C46 modified HSPC</other_name>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Cal-1 modified CD4+ T lymphocytes</intervention_name>
    <description>CD4+ T lymphocytes modified with LVsh5/C46 (Cal-1)</description>
    <arm_group_label>1 x 4mg/kg busulfan preconditioning</arm_group_label>
    <arm_group_label>2 x 4mg/kg busulfan pre-conditioning</arm_group_label>
    <arm_group_label>No busulfan pre-conditioning</arm_group_label>
    <other_name>LVsh5/C46 modified CD4+ T lymphocytes</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Prior to any study-related procedures, signed informed consent indicating that they&#xD;
             understand the purpose, risks and procedures required for the study and are willing to&#xD;
             participate in the study&#xD;
&#xD;
          -  Individuals aged 18 to 65 years of age (inclusive) at time of consent&#xD;
&#xD;
          -  Documented HIV-1 infection ≥ 6 months prior to Screening 1&#xD;
&#xD;
          -  Previous treatment with antiretroviral agents that had a demonstrated suppressive&#xD;
             effect (defined as plasma HIV RNA ≤ 50 copies/ml)&#xD;
&#xD;
          -  A documented viable ART regimen option, as determined by the Investigator, taking into&#xD;
             account prior ART experience and HIV geno/phenotyping analyses&#xD;
&#xD;
          -  Not taking antiretroviral therapy for ≥ 6 weeks prior to Screening 1, for one or more&#xD;
             of the following reasons:&#xD;
&#xD;
             i) Concerns over short-term or long-term toxicities associated with antiretroviral&#xD;
             agents, or ii) Treatment fatigue from the daily regimen of life-long therapy&#xD;
&#xD;
          -  Plasma HIV-1 viral RNA ≥ 5,000 copies/mL and ≤ 100,000 copies/ml at Screening 1 and&#xD;
             Screening 2&#xD;
&#xD;
          -  CD4+ T lymphocyte count ≥ 500 cells/µl at Screening 1 and Screening 2&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Abnormal hematology at Screening 1: Absolute neutrophil count (ANC) &lt; 1.5 x 109/L,&#xD;
             Platelet count &lt; 100 x 109/L, Hemoglobin &lt; 10 g/dL&#xD;
&#xD;
          -  Abnormal biochemistry at Screening 1: Alanine aminotransferase (ALT) &gt; 2.5 x Upper&#xD;
             Limit of Normal (ULN), Total bilirubin &gt; 1.5 x ULN, Serum creatinine &gt; 1.5 x ULN&#xD;
&#xD;
          -  Detection of any CXCR4-tropic HIV-1 at Screening 1&#xD;
&#xD;
          -  Evidence of co-infection with hepatitis B virus, hepatitis C virus, West Nile Virus,&#xD;
             or HTLV-1 as detected at Screening 2&#xD;
&#xD;
          -  Evidence of active TB infection determined by positive QuantiFERON®-TB Gold/IGRA test&#xD;
             result and clinical confirmation at Screening 2&#xD;
&#xD;
          -  ART or other antiretroviral therapy within 6 weeks of Screening 1 or any time during&#xD;
             the pre-infusion period&#xD;
&#xD;
          -  Documented history of CD4+ T lymphocyte count &lt; 250 cells/µl&#xD;
&#xD;
          -  Any previous or current AIDS-defining illnesses (CDC Category C), including&#xD;
             AIDS-related dementia, with the exception of Kaposi's sarcoma confined to the skin&#xD;
&#xD;
          -  History of malignancy or systemic chemotherapy within the last 5 years (i.e., subjects&#xD;
             with prior malignancy must be disease-free for 5 years), except curatively-treated&#xD;
             basal cell carcinoma, cutaneous squamous cell carcinoma, or cervical or anal&#xD;
             intra-epithelial neoplasia&#xD;
&#xD;
          -  History of steroid-dependent asthma in the past 5 years&#xD;
&#xD;
          -  History of seizure&#xD;
&#xD;
          -  Any clinical history of hematologic diseases including leukemia, myelodysplasia,&#xD;
             myeloproliferative disease, thromboembolic disease, sickle cell disorder,&#xD;
             thrombocytopenia or leukopenia&#xD;
&#xD;
          -  Class II-IV heart failure, according to the New York Heart Association classification&#xD;
&#xD;
          -  Inadequate venous access for apheresis, as assessed at Screening 1&#xD;
&#xD;
          -  Current or planned systemic immunosuppressive or immunomodulatory medication&#xD;
&#xD;
          -  Taking warfarin, aspirin or any medication that is likely to affect platelet function&#xD;
             or other aspects of blood coagulation, and unable to safely cease this medication for&#xD;
             a period of 1 week prior, during, and 1 week after administration of G-CSF (a total&#xD;
             period of 19 days)&#xD;
&#xD;
          -  Participation in any study involving any investigational drug or medical device within&#xD;
             30 days prior to Screening 1&#xD;
&#xD;
          -  Receipt of a vaccine for HIV-1 or any gene transfer product at any time&#xD;
&#xD;
          -  Prior treatment with recombinant G-CSF or busulfan or other stem-cell mobilizing or&#xD;
             modulating agent within the previous 12 months&#xD;
&#xD;
          -  Known hypersensitivity to busulfan, G-CSF (Neupogen™) or E. coli-derived proteins&#xD;
&#xD;
          -  Subjects who will not accept transfusions of blood products&#xD;
&#xD;
          -  Pregnant or breast-feeding at any time between Screening 1 and Baseline (infusion)&#xD;
&#xD;
          -  History of alcohol or drug abuse within the 12 months prior to Screening 1&#xD;
&#xD;
          -  Inability to understand and provide informed consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Ronald Mitsuyasu, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of California, Los Angeles</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>UCLA CARE Center</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90035</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Quest Clinical Research</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2020</verification_date>
  <study_first_submitted>November 19, 2012</study_first_submitted>
  <study_first_submitted_qc>November 22, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 28, 2012</study_first_posted>
  <results_first_submitted>June 25, 2020</results_first_submitted>
  <results_first_submitted_qc>July 22, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">August 6, 2020</results_first_posted>
  <disposition_first_submitted>September 17, 2018</disposition_first_submitted>
  <disposition_first_submitted_qc>September 17, 2018</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">September 24, 2018</disposition_first_posted>
  <last_update_submitted>July 22, 2020</last_update_submitted>
  <last_update_submitted_qc>July 22, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 6, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>HIV-1</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Acquired Immunodeficiency Syndrome</mesh_term>
    <mesh_term>HIV Infections</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Busulfan</mesh_term>
  </intervention_browse>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>July 8, 2015</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/50/NCT01734850/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>December 15, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/50/NCT01734850/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Cohort 1 (CSL202 With No Busulfan)</title>
          <description>Cal-1 modified Hematopoietic stem/progenitor cells (HSPC) and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
        </group>
        <group group_id="P2">
          <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
        </group>
        <group group_id="P3">
          <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
                <participants group_id="P2" count="4"/>
                <participants group_id="P3" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="4"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Continued to Week 48 but recommenced ART</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Not infused due to manufacturing failure</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Cohort 1 (CSL202 With No Busulfan)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
        </group>
        <group group_id="B2">
          <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
        </group>
        <group group_id="B3">
          <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
        </group>
        <group group_id="B4">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="4"/>
            <count group_id="B2" value="4"/>
            <count group_id="B3" value="5"/>
            <count group_id="B4" value="13"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="5"/>
                    <measurement group_id="B4" value="13"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="43.0" spread="11.7"/>
                    <measurement group_id="B2" value="48.5" spread="12.8"/>
                    <measurement group_id="B3" value="46.8" spread="3.1"/>
                    <measurement group_id="B4" value="46.2" spread="9.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="5"/>
                    <measurement group_id="B4" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="5"/>
                    <measurement group_id="B4" value="12"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="5"/>
                    <measurement group_id="B4" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Severe and Life-threatening Adverse Events (AEs)</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>Safety Population (SP): The safety population includes all enrolled subjects signing informed consent who started the interventional phase of the trial (i.e. CD4+ Apheresis and discontinued subjects who re-commenced ART)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Severe and Life-threatening Adverse Events (AEs)</title>
          <population>Safety Population (SP): The safety population includes all enrolled subjects signing informed consent who started the interventional phase of the trial (i.e. CD4+ Apheresis and discontinued subjects who re-commenced ART)</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Severe</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="4"/>
                    <measurement group_id="O3" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Life-threatening</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="3"/>
                    <measurement group_id="O3" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Severe or Life-threatening AEs Related to CSL202</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Severe or Life-threatening AEs Related to CSL202</title>
          <population>SP</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With the Presence of Replication-competent Retrovirus</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With the Presence of Replication-competent Retrovirus</title>
          <population>SP</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Predominant Integration Site Analysis</title>
        <description>Vector Integration Site Analysis performed only when Cal-1 Marking is &gt;= 1%.</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Predominant Integration Site Analysis</title>
          <description>Vector Integration Site Analysis performed only when Cal-1 Marking is &gt;= 1%.</description>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="NA">Not done because Cal-1 Marking was &lt;1% at the corresponding time point required</measurement>
                    <measurement group_id="O2" value="NA">Not done because Cal-1 Marking was &lt;1% at the corresponding time point required</measurement>
                    <measurement group_id="O3" value="NA">Not done because Cal-1 Marking was &lt;1% at the corresponding time point required</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean Cell Dose for CD4+ Cells (Ttn)</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Mean Cell Dose for CD4+ Cells (Ttn)</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>Number (10^8 cells)</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16.940" lower_limit="14.61" upper_limit="20.62"/>
                    <measurement group_id="O2" value="81.855" lower_limit="38.94" upper_limit="138.68"/>
                    <measurement group_id="O3" value="49.553" lower_limit="20.70" upper_limit="79.29"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean Cell Dose for CD34+ Cells (HSPCtn)</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Mean Cell Dose for CD34+ Cells (HSPCtn)</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>Number (10^6 cells/kg body weight)</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.1" lower_limit="0.35" upper_limit="5.0"/>
                    <measurement group_id="O2" value="2.4" lower_limit="1.0" upper_limit="4.0"/>
                    <measurement group_id="O3" value="10.6" lower_limit="7.4" upper_limit="12.5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>Percent transduction efficiency</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>CD4+ Cells (Ttn)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="41.0" lower_limit="21.0" upper_limit="60.0"/>
                    <measurement group_id="O2" value="29.2" lower_limit="13.0" upper_limit="39.0"/>
                    <measurement group_id="O3" value="66.8" lower_limit="39.0" upper_limit="84.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>CD34+ Cells (HSPCtn)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="58.6" lower_limit="51.0" upper_limit="77.0"/>
                    <measurement group_id="O2" value="26.3" lower_limit="12.0" upper_limit="53.0"/>
                    <measurement group_id="O3" value="31.3" lower_limit="21.0" upper_limit="49.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Total Area Under the Curve (AUC) for Busulfan</title>
        <description>Cohort 3: Total AUC = first dose AUC value + second dose AUC value</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O2">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Total Area Under the Curve (AUC) for Busulfan</title>
          <description>Cohort 3: Total AUC = first dose AUC value + second dose AUC value</description>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>micromolar*min</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6521.5" lower_limit="5863" upper_limit="7259"/>
                    <measurement group_id="O2" value="8296.8" lower_limit="7893" upper_limit="8601"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percent Cal-1 Marking in Peripheral Blood</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Percent Cal-1 Marking in Peripheral Blood</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>percent Cal-1 marking</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Peak marking</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.3500" lower_limit="0.0000" upper_limit="1.1600"/>
                    <measurement group_id="O2" value="0.7650" lower_limit="0.6100" upper_limit="0.8900"/>
                    <measurement group_id="O3" value="3.1200" lower_limit="0.1800" upper_limit="7.4600"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O2" value="0.1275" lower_limit="0.0000" upper_limit="0.3800"/>
                    <measurement group_id="O3" value="0.1275" lower_limit="0.0000" upper_limit="0.4900"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)</title>
        <description>Samples were collected via endoscopic biopsy from the sigmoid colon: 10-15 cm from the anal margin</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)</title>
          <description>Samples were collected via endoscopic biopsy from the sigmoid colon: 10-15 cm from the anal margin</description>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>copies/cell</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Peak marking</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.8121" lower_limit="0.0" upper_limit="2.7054"/>
                    <measurement group_id="O2" value="0.0007" lower_limit="0.0" upper_limit="0.0019"/>
                    <measurement group_id="O3" value="0.0022" lower_limit="0.0006" upper_limit="0.0060"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.8121" lower_limit="0.0" upper_limit="2.7054"/>
                    <measurement group_id="O2" value="0.0004" lower_limit="0.0" upper_limit="0.0010"/>
                    <measurement group_id="O3" value="0.0001" lower_limit="0.0" upper_limit="0.0004"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Cal-1 Marking in GALT (25-35 cm)</title>
        <description>Samples were collected via endoscopic biopsy from the sigmoid colon: 25-35 cm from the anal margin</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Cal-1 Marking in GALT (25-35 cm)</title>
          <description>Samples were collected via endoscopic biopsy from the sigmoid colon: 25-35 cm from the anal margin</description>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>copies/cell</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Peak marking</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0255" lower_limit="0.0" upper_limit="0.0677"/>
                    <measurement group_id="O2" value="0.0003" lower_limit="0.0" upper_limit="0.0011"/>
                    <measurement group_id="O3" value="0.0068" lower_limit="0.0020" upper_limit="0.0186"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0255" lower_limit="0.0" upper_limit="0.0677"/>
                    <measurement group_id="O2" value="0.0003" lower_limit="0.0" upper_limit="0.0011"/>
                    <measurement group_id="O3" value="0.0055" lower_limit="0.0" upper_limit="0.0186"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Cal-1 Marking in Bone Marrow</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Cal-1 Marking in Bone Marrow</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>copies/cell</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Peak marking</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0007" lower_limit="0.0" upper_limit="0.0026"/>
                    <measurement group_id="O2" value="0.0539" lower_limit="0.0005" upper_limit="0.2027"/>
                    <measurement group_id="O3" value="0.0021" lower_limit="0.0009" upper_limit="0.0030"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0007" lower_limit="0.0" upper_limit="0.0026"/>
                    <measurement group_id="O2" value="0.0015" lower_limit="0.0005" upper_limit="0.0044"/>
                    <measurement group_id="O3" value="0.0009" lower_limit="0.0003" upper_limit="0.0025"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Cal-1 C46 Expression in Peripheral Blood</title>
        <description>C46 relative expression will be analyzed by reverse transcriptase-quantitative polymerase chain reaction (RT-qPCR) and normalized to the expression of β2-microglobulin (β2M) mRNA</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Cal-1 C46 Expression in Peripheral Blood</title>
          <description>C46 relative expression will be analyzed by reverse transcriptase-quantitative polymerase chain reaction (RT-qPCR) and normalized to the expression of β2-microglobulin (β2M) mRNA</description>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>units of relative expression</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Peak marking</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O2" value="1.0496" lower_limit="0.0000" upper_limit="2.9977"/>
                    <measurement group_id="O3" value="2.1701" lower_limit="0.0000" upper_limit="6.4800"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O2" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O3" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Cal-1 sh5 Expression in Peripheral Blood</title>
        <description>sh5 relative expression will be analyzed by RT-qPCR and normalized to the expression of RNU38B microRNA</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Cal-1 sh5 Expression in Peripheral Blood</title>
          <description>sh5 relative expression will be analyzed by RT-qPCR and normalized to the expression of RNU38B microRNA</description>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>units of relative expression</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Peak marking</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O2" value="0.2027" lower_limit="0.0000" upper_limit="0.6489"/>
                    <measurement group_id="O3" value="1.6933" lower_limit="0.1930" upper_limit="4.6736"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O2" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                    <measurement group_id="O3" value="0.0000" lower_limit="0.0000" upper_limit="0.0000"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>Log10 (copies/mL)</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline screening</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.59" lower_limit="4.06" upper_limit="4.87"/>
                    <measurement group_id="O2" value="4.51" lower_limit="3.98" upper_limit="4.82"/>
                    <measurement group_id="O3" value="4.28" lower_limit="3.45" upper_limit="4.62"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 or at ART re-commencement</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.56" lower_limit="4.00" upper_limit="4.93"/>
                    <measurement group_id="O2" value="4.70" lower_limit="3.88" upper_limit="4.96"/>
                    <measurement group_id="O3" value="4.61" lower_limit="4.20" upper_limit="4.95"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement</title>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement</title>
          <population>SP (1 participant in Cohort 3 withdrew consent and did not receive busulfan or Ttn and HSPCtn)</population>
          <units>CD4+ cells/cubic mm</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline screening</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="680.75" lower_limit="522.5" upper_limit="809.5"/>
                    <measurement group_id="O2" value="575.13" lower_limit="537.0" upper_limit="677.5"/>
                    <measurement group_id="O3" value="668.63" lower_limit="529.0" upper_limit="878.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 or at ART re-commencement</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="553.3" lower_limit="450" upper_limit="759"/>
                    <measurement group_id="O2" value="383.5" lower_limit="274" upper_limit="549"/>
                    <measurement group_id="O3" value="245.5" lower_limit="152" upper_limit="385"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With HIV-1 Tropism Shift</title>
        <description>Shift from R5 to X4 or dual/mixed tropism</description>
        <time_frame>Up to 48 weeks</time_frame>
        <population>SP</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1 (CSL202 With No Busulfan)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
            <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC)administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With HIV-1 Tropism Shift</title>
          <description>Shift from R5 to X4 or dual/mixed tropism</description>
          <population>SP</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Up to 48 weeks per participant</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Cohort 1 (CSL202 With No Busulfan)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202) without busulfan preconditioning</description>
        </group>
        <group group_id="E2">
          <title>Cohort 2 (CSL202 With 1 Busulfan Dose)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), with single 4mg/kg busulfan dose administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
        </group>
        <group group_id="E3">
          <title>Cohort 3 (CSL202 With 2 Busulfan Doses)</title>
          <description>Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes (CSL202), first busulfan dose = 3mg/kg and second busulfan dose adjusted (total target exposure of 8,000 μmolar/min AUC) administered as pre-conditioning for transplant&#xD;
Busulfan: Intravenous busulfan</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>gastroenteritis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="4"/>
                <counts group_id="E2" subjects_affected="4" subjects_at_risk="4"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Neutropenia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="38" subjects_affected="4" subjects_at_risk="4"/>
                <counts group_id="E3" events="21" subjects_affected="5" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Thrombocytopenia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="19" subjects_affected="4" subjects_at_risk="4"/>
                <counts group_id="E3" events="40" subjects_affected="4" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Leukopenia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="18" subjects_affected="4" subjects_at_risk="4"/>
                <counts group_id="E3" events="4" subjects_affected="3" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Leukocytosis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>ear congestion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Eye irritation</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Eye pruritus</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Vision blurred</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="3" subjects_affected="2" subjects_at_risk="4"/>
                <counts group_id="E3" events="5" subjects_affected="5" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" events="3" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="5" subjects_affected="4" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="3" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Retching</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Toothache</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Abdominal distension</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Dry mouth</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Faeces soft</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Food poisoning</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Haematochezia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Stomatitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Tongue discolouration</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Gastroenteritis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="4"/>
                <counts group_id="E2" events="5" subjects_affected="3" subjects_at_risk="4"/>
                <counts group_id="E3" events="4" subjects_affected="3" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Chills</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Malaise</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Night Sweats</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Catheter site pain</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Influenza like illness</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" events="5" subjects_affected="3" subjects_at_risk="4"/>
                <counts group_id="E2" events="2" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Cellulitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Acarodermatitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Bronchitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Diverticulitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Gingivitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Secondary syphilis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Staphylococcal infection</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Tinea pedis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Contusion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Head injury</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Road traffic accident</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Alanine aminotransferase increased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Aspartate aminotransferase increased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>CD4 lymphocytes decreased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="4"/>
                <counts group_id="E2" events="5" subjects_affected="3" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" events="4" subjects_affected="2" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Bone pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Insomnia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="3" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Confusional state</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Hypoaesthesia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Migraine</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Paraesthesia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Somnolence</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Depression</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Irritability</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Somnambulism</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Haematospermia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="2" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" events="2" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Wheezing</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Epistaxis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Paranasal sinus hypersecretion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Sinus congestion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Sneezing</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Throat irritation</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Tonsillar disorder</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Upper-airway cough syndrome</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Skin odour abnormal</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="4"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Alopecia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="3" subjects_affected="2" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Dermatitis allergic</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Rash vesicular</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Acne</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Dry skin</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Eosinophilic pustular folliculitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Excoriation</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Photosensitivity reaction</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pruritus</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Rash pruritic</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Seborrhoeic dermatitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Skin erosion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Skin mass</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Urticaria</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Hot flush</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="4"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Trial Registration Coordinator</name_or_title>
      <organization>CSL Behring</organization>
      <phone>484-878-4000</phone>
      <email>clinicaltrials@cslbehring.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01734850
org study id: CAL-USA-11
nct id: NCT01734850
lead sponsor: 
has dmc: Yes
This is an early phase research study looking at whether an experimental gene transfer,
      LVsh5/C46 (also known as Cal-1), is safe and if it can protect the immune system from the
      effects of HIV without the use of antiretroviral drugs.

      Cal-1 is an experimental gene transfer agent designed to inhibit HIV infection through 2
      active parts:

        1. Removing a protein named CCR5 from bone marrow and white blood cells

        2. Producing a protein named C46 on bone marrow and white blood cells
It is estimated that 33 million individuals are currently infected with HIV. HIV/AIDS is a
      disease that impairs immune function, primarily by decreasing CD4+ T lymphocytes. The
      progression can be contained by daily dosing with antiretroviral therapy (ART) but there are
      side effects that can be treatment limiting, and the development of HIV drug resistance can
      force the physician to modify the ART regimen. There are no effective vaccines currently
      available for HIV.

      LVsh5/C46 (also known as Cal-1) is a dual therapeutic, self-inactivating lentiviral vector
      that encodes for both a short hairpin RNA against the HIV-1 co-receptor CCR5 (sh5) and a
      HIV-1 fusion inhibitor, C46 and inhibits two processes required for HIV-1 infection:

        1. Binding of the virus to the cellular CCR5 co-receptor and

        2. Fusion of the virus with the host cell

      The rationale is that Cal-1 introduced into hematopoietic progenitor/stem cells (HSPC) and
      mature CD4+ T lymphocytes will protect these cells and their progeny cells from HIV-1
      infection and its pathogenic sequelae. This may provide a continuous means of controlling
      HIV-1 after a single or infrequent dose(s), thereby decreasing or delaying (partially or
      completely) the need for antiretroviral drug therapy.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants With Severe and Life-threatening Adverse Events (AEs)
time frame: Up to 48 weeks
measure: Number of Participants With Severe or Life-threatening AEs Related to CSL202
time frame: Up to 48 weeks
measure: Number of Participants With the Presence of Replication-competent Retrovirus
time frame: Up to 48 weeks
measure: Number of Participants With Predominant Integration Site Analysis
time frame: Up to 48 weeks
description: Vector Integration Site Analysis performed only when Cal-1 Marking is >= 1%.
measure: Mean Cell Dose for CD4+ Cells (Ttn)
time frame: Up to 48 weeks
measure: Mean Cell Dose for CD34+ Cells (HSPCtn)
time frame: Up to 48 weeks
measure: Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product
time frame: Up to 48 weeks
measure: Total Area Under the Curve (AUC) for Busulfan
time frame: Up to 48 weeks
description: Cohort 3: Total AUC = first dose AUC value + second dose AUC value
measure: Percent Cal-1 Marking in Peripheral Blood
time frame: Up to 48 weeks
measure: Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)
time frame: Up to 48 weeks
description: Samples were collected via endoscopic biopsy from the sigmoid colon: 10-15 cm from the anal margin
measure: Cal-1 Marking in GALT (25-35 cm)
time frame: Up to 48 weeks
description: Samples were collected via endoscopic biopsy from the sigmoid colon: 25-35 cm from the anal margin
measure: Cal-1 Marking in Bone Marrow
time frame: Up to 48 weeks
measure: Cal-1 C46 Expression in Peripheral Blood
time frame: Up to 48 weeks
description: C46 relative expression will be analyzed by reverse transcriptase-quantitative polymerase chain reaction (RT-qPCR) and normalized to the expression of β2-microglobulin (β2M) mRNA
measure: Cal-1 sh5 Expression in Peripheral Blood
time frame: Up to 48 weeks
description: sh5 relative expression will be analyzed by RT-qPCR and normalized to the expression of RNU38B microRNA
measure: HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement
time frame: Up to 48 weeks
measure: CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement
time frame: Up to 48 weeks
measure: Number of Participants With HIV-1 Tropism Shift
time frame: Up to 48 weeks
description: Shift from R5 to X4 or dual/mixed tropism
arm group label: No busulfan pre-conditioning
arm group type: Experimental
description: Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes without busulfan preconditioning
arm group label: 1 x 4mg/kg busulfan preconditioning
arm group type: Experimental
description: Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes, with single 4mg/kg busulfan dose administered as pre-conditioning for transplant
arm group label: 2 x 4mg/kg busulfan pre-conditioning
arm group type: Experimental
description: Cal-1 modified HSPC and Cal-1 modified CD4+ T lymphocytes, with two 4mg/kg busulfan doses administered as pre-conditioning for transplant
intervention type: Drug
intervention name: Busulfan
description: Intravenous busulfan
arm group label: 1 x 4mg/kg busulfan preconditioning
arm group label: 2 x 4mg/kg busulfan pre-conditioning
other name: Busulfex
intervention type: Biological
intervention name: Cal-1 modified HSPC
description: Hematopoietic progenitor/stem cells (HSPC) modified with LVsh5/C46 (Cal-1)
arm group label: 1 x 4mg/kg busulfan preconditioning
arm group label: 2 x 4mg/kg busulfan pre-conditioning
arm group label: No busulfan pre-conditioning
other name: LVsh5/C46 modified HSPC
intervention type: Biological
intervention name: Cal-1 modified CD4+ T lymphocytes
description: CD4+ T lymphocytes modified with LVsh5/C46 (Cal-1)
arm group label: 1 x 4mg/kg busulfan preconditioning
arm group label: 2 x 4mg/kg busulfan pre-conditioning
arm group label: No busulfan pre-conditioning
other name: LVsh5/C46 modified CD4+ T lymphocytes
criteria: 
gender: All
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
last name: Ronald Mitsuyasu, M.D.
role: Principal Investigator
affiliation: University of California, Los Angeles
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Acquired Immunodeficiency Syndrome
mesh term: HIV Infections
mesh term: Busulfan
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 