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/NCT06019728</url>
    </required_header>
    <id_info>
      <org_study_id>LPS17726</org_study_id>
      <secondary_id>U1111-1287-8570</secondary_id>
      <nct_id>NCT06019728</nct_id>
    </id_info>
    <brief_title>A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme</brief_title>
    <acronym>SHORTEN</acronym>
    <official_title>A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Sanofi</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Sanofi</source>
    <oversight_info>
      <has_dmc>No</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>
        This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved&#xD;
        dose with increases in the infusion rate and reduced infusion volume. This study aims to&#xD;
        generate data to provide the guidance on how infusion rate can be safely increased and&#xD;
        minimize the burden of the life-long treatment with Fabrazyme.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        The total duration will be up to 6 months&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 29, 2023</start_date>
    <completion_date type="Anticipated">September 4, 2025</completion_date>
    <primary_completion_date type="Anticipated">September 4, 2025</primary_completion_date>
    <phase>Phase 4</phase>
    <study_type>Interventional</study_type>
    <study_design_info>
      <allocation>N/A</allocation>
      <intervention_model>Single Group Assignment</intervention_model>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Reduction of infusion duration from pretrial average of recent 3 infusions</measure>
      <time_frame>Baseline to month 4</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Reduction of infusion duration from initial 120 minutes</measure>
      <time_frame>Baseline to month 4</time_frame>
    </primary_outcome>
    <primary_outcome>
      <measure>Shortest infusion duration each participant tolerates</measure>
      <time_frame>Baseline to month 4</time_frame>
    </primary_outcome>
    <secondary_outcome>
      <measure>Number of participants achieving the shortest planned duration of infusion time</measure>
      <time_frame>Baseline to month 4</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of participants achieving the shortest planned duration of infusion time</measure>
      <time_frame>Baseline to month 4</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR</measure>
      <time_frame>Baseline to month 4</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR</measure>
      <time_frame>Baseline to month 4</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants achieving infusion duration without experiencing any IAR</measure>
      <time_frame>Baseline to month 4</time_frame>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">18</enrollment>
    <condition>Fabry's Disease</condition>
    <arm_group>
      <arm_group_label>agalsidase beta</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>agalsidase beta 1 mg/kg infusion once every other week</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>AGALSIDASE BETA (GZ419828)</intervention_name>
      <description>Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion</description>
      <arm_group_label>agalsidase beta</arm_group_label>
      <other_name>Fabrazyme</other_name>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Acetaminophen</intervention_name>
      <description>Tablet or solution; Oral</description>
      <arm_group_label>agalsidase beta</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Diphenhydramine</intervention_name>
      <description>Tablet or solution; Oral</description>
      <arm_group_label>agalsidase beta</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Dexamethasone</intervention_name>
      <description>Tablet or solution; Oral</description>
      <arm_group_label>agalsidase beta</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Montelukast</intervention_name>
      <description>Tablet or chewable tablet or oral granules; Oral</description>
      <arm_group_label>agalsidase beta</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
          - Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time&#xD;
          of signing the informed consent form (ICF) or assent, if applicable.&#xD;
  &#xD;
            -  Cohort 1: female participants with body weight ≥30 kg who have been treated with&#xD;
               Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.&#xD;
  &#xD;
            -  Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated&#xD;
               with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.&#xD;
  &#xD;
            -  Cohort 3: classic male participants with body weight ≥30 kg who have been treated with&#xD;
               Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.&#xD;
  &#xD;
            -  Cohort 4: participants with body weight &amp;lt;30 kg who have been treated with Fabrazyme&#xD;
               for at least 3 months without IARs during the most recent 3 infusions.&#xD;
  &#xD;
            -  Cohort 5: ERT-naïve participants. Women of childbearing potential must use a highly&#xD;
               effective method of contraception through the study.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Female participants who are pregnant or breastfeeding.&#xD;
  &#xD;
            -  History of significant allergic disease or hypersensitivity to Fabrazyme or other&#xD;
               medicinal products.&#xD;
  &#xD;
            -  Contraindication to Fabrazyme or any of the premedications or rescue medications&#xD;
               (diphenhydramine, acetaminophen, montelukast, dexamethasone).&#xD;
  &#xD;
            -  Any other medical condition considered to make the increased infusion rate not&#xD;
               tolerable at the Investigator's discretion. The above information is not intended to&#xD;
               contain all considerations relevant to a potential participation in a clinical trial.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>2 Years</minimum_age>
      <maximum_age>65 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Clinical Sciences &amp;amp; Operations</last_name>
      <role>Study Director</role>
      <affiliation>Sanofi</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Trial Transparency email recommended (Toll free for US &amp;amp; Canada)</last_name>
      <phone>800-633-1610</phone>
      <phone_ext>option 6</phone_ext>
      <email>Contact-US@sanofi.com</email>
    </overall_contact>
    <verification_date>September 2023</verification_date>
    <study_first_submitted>August 25, 2023</study_first_submitted>
    <study_first_submitted_qc>August 30, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 31, 2023</study_first_posted>
    <last_update_submitted>September 14, 2023</last_update_submitted>
    <last_update_submitted_qc>September 14, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 15, 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>Fabry Disease</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Acetaminophen</mesh_term>
      <mesh_term>Diphenhydramine</mesh_term>
      <mesh_term>Promethazine</mesh_term>
      <mesh_term>Dexamethasone</mesh_term>
      <mesh_term>Montelukast</mesh_term>
    </intervention_browse>
    <patient_data>
      <sharing_ipd>Yes</sharing_ipd>
      <ipd_description>Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org</ipd_description>
    </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/NCT06019728
  org study id: LPS17726
  secondary id: U1111-1287-8570
  nct id: NCT06019728
  lead sponsor: 
  has dmc: No
  is fda regulated drug: Yes
  is fda regulated device: No
  This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved
        dose with increases in the infusion rate and reduced infusion volume. This study aims to
        generate data to provide the guidance on how infusion rate can be safely increased and
        minimize the burden of the life-long treatment with Fabrazyme.
  The total duration will be up to 6 months
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Reduction of infusion duration from pretrial average of recent 3 infusions
  time frame: Baseline to month 4
  measure: Reduction of infusion duration from initial 120 minutes
  time frame: Baseline to month 4
  measure: Shortest infusion duration each participant tolerates
  time frame: Baseline to month 4
  measure: Number of participants achieving the shortest planned duration of infusion time
  time frame: Baseline to month 4
  measure: Percentage of participants achieving the shortest planned duration of infusion time
  time frame: Baseline to month 4
  measure: Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
  time frame: Baseline to month 4
  measure: Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
  time frame: Baseline to month 4
  measure: Number of participants achieving infusion duration without experiencing any IAR
  time frame: Baseline to month 4
  arm group label: agalsidase beta
  arm group type: Experimental
  description: agalsidase beta 1 mg/kg infusion once every other week
  intervention type: Drug
  intervention name: AGALSIDASE BETA (GZ419828)
  description: Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
  arm group label: agalsidase beta
  other name: Fabrazyme
  intervention type: Drug
  intervention name: Acetaminophen
  description: Tablet or solution; Oral
  arm group label: agalsidase beta
  intervention type: Drug
  intervention name: Diphenhydramine
  description: Tablet or solution; Oral
  arm group label: agalsidase beta
  intervention type: Drug
  intervention name: Dexamethasone
  description: Tablet or solution; Oral
  arm group label: agalsidase beta
  intervention type: Drug
  intervention name: Montelukast
  description: Tablet or chewable tablet or oral granules; Oral
  arm group label: agalsidase beta
  criteria: 
  gender: All
  minimum age: 2 Years
  maximum age: 65 Years
  healthy volunteers: No
  last name: Clinical Sciences &amp; Operations
  role: Study Director
  affiliation: Sanofi
  last name: Trial Transparency email recommended (Toll free for US &amp; Canada)
  phone: 800-633-1610
  phone ext: option 6
  email: Contact-US@sanofi.com
  responsible party type: Sponsor
  mesh term: Fabry Disease
  mesh term: Acetaminophen
  mesh term: Diphenhydramine
  mesh term: Promethazine
  mesh term: Dexamethasone
  mesh term: Montelukast
  sharing ipd: Yes
  ipd description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org