xml: <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/NCT06003387</url>
    </required_header>
    <id_info>
      <org_study_id>CSL222_3005</org_study_id>
      <nct_id>NCT06003387</nct_id>
    </id_info>
    <brief_title>Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)</brief_title>
    <official_title>Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>CSL Behring</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>CSL Behring</source>
    <oversight_info>
      <has_dmc>Yes</has_dmc>
      <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        The purpose of this study is to assess the risk of bleeding due to failure of expected&#xD;
        pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with&#xD;
        detectable pretreatment AAV5 Nabs.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">December 2023</start_date>
    <completion_date type="Anticipated">October 2028</completion_date>
    <primary_completion_date type="Anticipated">October 2028</primary_completion_date>
    <phase>Phase 3</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>N/A</allocation>
      <intervention_model>Single Group Assignment</intervention_model>
      <primary_purpose>Prevention</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Annualized Bleeding Rate (ABR)</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
      <description>The total, spontaneous, joint, and Factor IX (FIX)-treated bleeding episodes will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs (TESAEs), and TEAEs of Special Interest (TEAESIs)</measure>
      <time_frame>Post-dose: At Months 6,12, and 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants With TEAEs, TESAEs, and TEAESIs</measure>
      <time_frame>Post-dose: At Months 6,12, and 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of TEAEs, TESAEs, and TEAESIs</measure>
      <time_frame>Post-dose: At Months 6,12, and 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants with Change From Baseline in Abdominal Ultrasound</measure>
      <time_frame>Baseline up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants Who Develop Factor IX (FIX) Inhibitors</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants who Develop FIX Inhibitors</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants with Clinically Significant Hematology and Serum Chemistry Parameters</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants With Clinically Significant Hematology and Serum Chemistry Parameters</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants with Clinically Significant Change in Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST)</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants With Clinically Significant Change in ALT or AST</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants Treated With Corticosteroids For Change in ALT or AST</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants Treated With Corticosteroids For Change in ALT or AST</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants With Clinically Significant Alpha-fetoprotein (AFP)</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants With Clinically Significant AFP</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants with Infusion Related Reactions or Hypersensitivity Reactions</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants With Infusion Related Reactions or Hypersensitivity Reactions</measure>
      <time_frame>Up to 18 months post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants With the Endogenous FIX Activity</measure>
      <time_frame>Post-dose: At Months 6, 12, and 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change From Baseline in the Endogenous FIX Activity</measure>
      <time_frame>Baseline up to Months 6, 12, and 18 post dose</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Annualized Consumption of FIX Replacement Therapy</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Annualized Infusion Rate of FIX Replacement Therapy</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants Remaining Free of Previous Continuous Routine FIX Prophylaxis</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>ABR for Spontaneous Bleeding Episodes, Joint Bleeding Episodes, and FIX-treated Bleeding Episodes Separately</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Correlation Analysis of FIX Activity Levels</measure>
      <time_frame>Post-dose: Months 6 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants With New Target Joints and Resolved New or Preexisting Target Joints</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
      <description>Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of Participants With Zero Bleeds Following Stable FIX Expression</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of Participants With Zero Bleeds Following Stable FIX Expression</measure>
      <time_frame>Post-dose: Months 7 to 18</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score and Treatment Domain Score</measure>
      <time_frame>Post-dose: Months 6 to 18</time_frame>
      <description>The Hem-A-QoL (Hemophilia Quality of Life Questionnaire for Adults) consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a &quot;not applicable&quot; option). The Hem-A-QoL Total Score and Treatment Domain Score range from 0 to 100, with lower scores reflecting a better quality of life.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change From Baseline in the Hem-A-QoL Total Score and Treatment Domain Score</measure>
      <time_frame>Baseline, Months 6 to 18 post dose</time_frame>
      <description>The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a &quot;not applicable&quot; option). The Hem-A-QoL Total Score and Treatment Domain Score range from 0 to 100, with lower scores reflecting a better quality of life. The change from baseline in the Hem-A-QoL Total Score and Treatment Domain Score will be determined.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score</measure>
      <time_frame>Post-dose: Months 6 to 18</time_frame>
      <description>The EQ-5D-5L questionnaire visual analogue scale (VAS) measures overall health status on a vertical VAS ranging from 0 to 100. A higher score indicates better quality of life.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>EQ-5D-5L Index Scores</measure>
      <time_frame>Post-dose: Months 6 to 18</time_frame>
      <description>The EQ-5D-5L questionnaire descriptive system of health-related quality of life consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single index utility score (typically between -0.6 and 1). A higher score indicates better quality of life.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change from Baseline in the EQ-5D-5L VAS Score</measure>
      <time_frame>Baseline, Months 6 to 18 post dose</time_frame>
      <description>The EQ-5D-5L questionnaire visual analogue scale (VAS) measures overall health status on a vertical VAS ranging from 0 to 100. A higher score indicates better quality of life. The change from baseline in the EQ-5D-5L VAS score will be determined.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change From Baseline in the EQ-5D-5L Index Scores</measure>
      <time_frame>Baseline, Months 6 to 18 post dose</time_frame>
      <description>The EQ-5D-5L questionnaire descriptive system of health-related quality of life consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single index utility score (typically between -0.6 and 1). A higher score indicates better quality of life. The change from baseline in the EQ-5D-5L index score will be determined.</description>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">35</enrollment>
    <condition>Hemophilia B</condition>
    <arm_group>
      <arm_group_label>CSL222</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram (gc/kg) on Day D.</description>
    </arm_group>
    <intervention>
      <intervention_type>Genetic</intervention_type>
      <intervention_name>CSL222 (AAV5-hFIXco-Padua)</intervention_name>
      <description>Administered as a single IV infusion.</description>
      <arm_group_label>CSL222</arm_group_label>
      <other_name>Etranacogene dezaparvovec</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Has congenital hemophilia B with known severe or moderately severe FIX deficiency (≤&#xD;
               2% of normal circulating FIX) for which the participant is on continuous routine FIX&#xD;
               prophylaxis&#xD;
  &#xD;
            -  Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit&#xD;
               L-Final using a validated AAV5 NAb assay (based on central laboratory results)&#xD;
  &#xD;
            -  Has &gt; 150 previous exposure days to FIX replacement therapy&#xD;
  &#xD;
            -  Has been on stable FIX prophylaxis for at least 2 months before Screening&#xD;
  &#xD;
            -  Has demonstrated capability to independently, accurately, and in a timely manner&#xD;
               complete the eDiary during the Lead-in Period, as judged by the investigator&#xD;
  &#xD;
            -  Acceptance to barrier contraception protection for 1 year starting the day of CSL222&#xD;
               treatment&#xD;
  &#xD;
            -  Able to provide informed consent after receipt of verbal and written information about&#xD;
               the study&#xD;
  &#xD;
            -  Investigator believes that the participant (or the participant's legally acceptable&#xD;
               representative[s]) understands the nature, scope, and possible consequences of the&#xD;
               study and is able to adhere to the study procedures.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  History of FIX inhibitors or positive FIX inhibitor test at Screening or Visit L&#xD;
               (lead-in period)-Final (based on central laboratory results)&#xD;
  &#xD;
            -  Screening and Visit L-Final laboratory values that meet the definition of Severe&#xD;
               Hepatic Impairment per Common Terminology Criteria for Adverse Events (CTCAE) (based&#xD;
               on central laboratory results)&#xD;
  &#xD;
            -  ALT &gt; 2 × the upper limit of normal (ULN) at Screening and Visit L-Final (based on&#xD;
               central laboratory results)&#xD;
  &#xD;
            -  Any condition other than hemophilia B resulting in an increased bleeding tendency&#xD;
  &#xD;
            -  Any uncontrolled or untreated infection (human immunodeficiency virus [HIV], hepatitis&#xD;
               C, etc.) or any other significant concurrent, uncontrolled medical condition evaluated&#xD;
               by the investigator to interfere with adherence to the clinical study protocol or with&#xD;
               the degree of tolerance to CSL222.&#xD;
  &#xD;
            -  Thrombocytopenia, defined as a platelet count below 50 × 10^9/L, at Screening and&#xD;
               Visit L-Final (based on central laboratory results)&#xD;
  &#xD;
            -  Known history of allergy to corticosteroids or known medical condition that would&#xD;
               require chronic administration of steroids.&#xD;
  &#xD;
            -  Known uncontrolled allergic conditions or allergy / hypersensitivity to any component&#xD;
               of the CSL222 excipients&#xD;
  &#xD;
            -  Previous gene therapy treatment&#xD;
  &#xD;
            -  Receipt of an experimental agent or device within 60 days before Screening until the&#xD;
               end of the study.&#xD;
  &#xD;
            -  Note: Other protocol pre-specified exclusion criteria may apply.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Study Director</last_name>
      <role>Study Director</role>
      <affiliation>CSL Behring LLC</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Trial Registration Coordinator</last_name>
      <phone>1-610-878-4000</phone>
      <email>clinicaltrials@cslbehring.com</email>
    </overall_contact>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 1, 2023</study_first_submitted>
    <study_first_submitted_qc>August 14, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 22, 2023</study_first_posted>
    <last_update_submitted>August 14, 2023</last_update_submitted>
    <last_update_submitted_qc>August 14, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 22, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Hemophilia A</mesh_term>
      <mesh_term>Hemophilia B</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>Yes</sharing_ipd>
      <ipd_description>CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.&#xD;
  Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.&#xD;
  If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.</ipd_description>
      <ipd_info_type>Study Protocol</ipd_info_type>
      <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
      <ipd_time_frame>IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.</ipd_time_frame>
      <ipd_access_criteria>Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.&#xD;
  An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.&#xD;
  The requesting party must execute an appropriate data sharing agreement before IPD will be made available.</ipd_access_criteria>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06003387
  org study id: CSL222_3005
  nct id: NCT06003387
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  The purpose of this study is to assess the risk of bleeding due to failure of expected
        pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with
        detectable pretreatment AAV5 Nabs.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Prevention
  masking: None (Open Label)
  measure: Annualized Bleeding Rate (ABR)
  time frame: Post-dose: Months 7 to 18
  description: The total, spontaneous, joint, and Factor IX (FIX)-treated bleeding episodes will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk.
  measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs (TESAEs), and TEAEs of Special Interest (TEAESIs)
  time frame: Post-dose: At Months 6,12, and 18
  measure: Percentage of Participants With TEAEs, TESAEs, and TEAESIs
  time frame: Post-dose: At Months 6,12, and 18
  measure: Number of TEAEs, TESAEs, and TEAESIs
  time frame: Post-dose: At Months 6,12, and 18
  measure: Number of Participants with Change From Baseline in Abdominal Ultrasound
  time frame: Baseline up to 18 months post dose
  measure: Number of Participants Who Develop Factor IX (FIX) Inhibitors
  time frame: Up to 18 months post dose
  measure: Percentage of Participants who Develop FIX Inhibitors
  time frame: Up to 18 months post dose
  measure: Number of Participants with Clinically Significant Hematology and Serum Chemistry Parameters
  time frame: Up to 18 months post dose
  measure: Percentage of Participants With Clinically Significant Hematology and Serum Chemistry Parameters
  time frame: Up to 18 months post dose
  measure: Number of Participants with Clinically Significant Change in Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST)
  time frame: Up to 18 months post dose
  measure: Percentage of Participants With Clinically Significant Change in ALT or AST
  time frame: Up to 18 months post dose
  measure: Number of Participants Treated With Corticosteroids For Change in ALT or AST
  time frame: Up to 18 months post dose
  measure: Percentage of Participants Treated With Corticosteroids For Change in ALT or AST
  time frame: Up to 18 months post dose
  measure: Number of Participants With Clinically Significant Alpha-fetoprotein (AFP)
  time frame: Up to 18 months post dose
  measure: Percentage of Participants With Clinically Significant AFP
  time frame: Up to 18 months post dose
  measure: Number of Participants with Infusion Related Reactions or Hypersensitivity Reactions
  time frame: Up to 18 months post dose
  measure: Percentage of Participants With Infusion Related Reactions or Hypersensitivity Reactions
  time frame: Up to 18 months post dose
  measure: Number of Participants With the Endogenous FIX Activity
  time frame: Post-dose: At Months 6, 12, and 18
  measure: Change From Baseline in the Endogenous FIX Activity
  time frame: Baseline up to Months 6, 12, and 18 post dose
  measure: Annualized Consumption of FIX Replacement Therapy
  time frame: Post-dose: Months 7 to 18
  measure: Annualized Infusion Rate of FIX Replacement Therapy
  time frame: Post-dose: Months 7 to 18
  measure: Percentage of Participants Remaining Free of Previous Continuous Routine FIX Prophylaxis
  time frame: Post-dose: Months 7 to 18
  measure: ABR for Spontaneous Bleeding Episodes, Joint Bleeding Episodes, and FIX-treated Bleeding Episodes Separately
  time frame: Post-dose: Months 7 to 18
  measure: Correlation Analysis of FIX Activity Levels
  time frame: Post-dose: Months 6 to 18
  measure: Number of Participants With New Target Joints and Resolved New or Preexisting Target Joints
  time frame: Post-dose: Months 7 to 18
  description: Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint.
  measure: Number of Participants With Zero Bleeds Following Stable FIX Expression
  time frame: Post-dose: Months 7 to 18
  measure: Percentage of Participants With Zero Bleeds Following Stable FIX Expression
  time frame: Post-dose: Months 7 to 18
  measure: Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score and Treatment Domain Score
  time frame: Post-dose: Months 6 to 18
  description: The Hem-A-QoL (Hemophilia Quality of Life Questionnaire for Adults) consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The Hem-A-QoL Total Score and Treatment Domain Score range from 0 to 100, with lower scores reflecting a better quality of life.
  measure: Change From Baseline in the Hem-A-QoL Total Score and Treatment Domain Score
  time frame: Baseline, Months 6 to 18 post dose
  description: The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The Hem-A-QoL Total Score and Treatment Domain Score range from 0 to 100, with lower scores reflecting a better quality of life. The change from baseline in the Hem-A-QoL Total Score and Treatment Domain Score will be determined.
  measure: EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score
  time frame: Post-dose: Months 6 to 18
  description: The EQ-5D-5L questionnaire visual analogue scale (VAS) measures overall health status on a vertical VAS ranging from 0 to 100. A higher score indicates better quality of life.
  measure: EQ-5D-5L Index Scores
  time frame: Post-dose: Months 6 to 18
  description: The EQ-5D-5L questionnaire descriptive system of health-related quality of life consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single index utility score (typically between -0.6 and 1). A higher score indicates better quality of life.
  measure: Change from Baseline in the EQ-5D-5L VAS Score
  time frame: Baseline, Months 6 to 18 post dose
  description: The EQ-5D-5L questionnaire visual analogue scale (VAS) measures overall health status on a vertical VAS ranging from 0 to 100. A higher score indicates better quality of life. The change from baseline in the EQ-5D-5L VAS score will be determined.
  measure: Change From Baseline in the EQ-5D-5L Index Scores
  time frame: Baseline, Months 6 to 18 post dose
  description: The EQ-5D-5L questionnaire descriptive system of health-related quality of life consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single index utility score (typically between -0.6 and 1). A higher score indicates better quality of life. The change from baseline in the EQ-5D-5L index score will be determined.
  arm group label: CSL222
  arm group type: Experimental
  description: Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram (gc/kg) on Day D.
  intervention type: Genetic
  intervention name: CSL222 (AAV5-hFIXco-Padua)
  description: Administered as a single IV infusion.
  arm group label: CSL222
  other name: Etranacogene dezaparvovec
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Study Director
  role: Study Director
  affiliation: CSL Behring LLC
  last name: Trial Registration Coordinator
  phone: 1-610-878-4000
  email: clinicaltrials@cslbehring.com
  responsible party type: Sponsor
  mesh term: Hemophilia A
  mesh term: Hemophilia B
  sharing ipd: Yes
  ipd description: CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
  Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.
  If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
  ipd info type: Study Protocol
  ipd info type: Statistical Analysis Plan (SAP)
  ipd time frame: IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
  ipd access criteria: Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.
  An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.
  The requesting party must execute an appropriate data sharing agreement before IPD will be made available.