<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/NCT04455230</url>
  </required_header>
  <id_info>
    <org_study_id>FLT190-02</org_study_id>
    <nct_id>NCT04455230</nct_id>
  </id_info>
  <brief_title>A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190</brief_title>
  <official_title>A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Freeline Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Freeline Therapeutics</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>
      Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an&#xD;
      abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously&#xD;
      expressed throughout the body and is responsible for the breakdown of glycosphingolipids,&#xD;
      deficiency of which results in the accumulation of specific glycosphingolipids that are&#xD;
      associated with the pathophysiology of the disease. Current treatment for Fabry disease is&#xD;
      limited to the symptomatic management of pain, conventional management of complications, and&#xD;
      methods to increase the availability of functional αGLA.&#xD;
&#xD;
      This clinical study aims to investigate the long-term safety and durability of αGLA in&#xD;
      patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical&#xD;
      studies.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">September 8, 2020</start_date>
  <completion_date type="Anticipated">December 2030</completion_date>
  <primary_completion_date type="Anticipated">December 2030</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>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <intervention_model_description>A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease</intervention_model_description>
    <primary_purpose>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Safety endpoint will be assessed by the reporting of adverse events (AEs) according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.</measure>
    <time_frame>Entry to 5 years post dosing</time_frame>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">50</enrollment>
  <condition>Fabry Disease</condition>
  <condition>Lysosomal Storage Diseases</condition>
  <arm_group>
    <arm_group_label>Participants who have received gene therapy vector (FLT190)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Genetic</intervention_type>
    <intervention_name>FLT190</intervention_name>
    <description>FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).</description>
    <arm_group_label>Participants who have received gene therapy vector (FLT190)</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Subjects who have previously received FLT190&#xD;
&#xD;
          -  Provision of full informed consent and able to comply with all requirements of the&#xD;
             study including long-term follow-up for 60 months (5 years) post-treatment.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  N/A&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Charité - Universitätsmedizin Berlin</name>
      <address>
        <city>Berlin</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Free London</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Germany</country>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>June 29, 2020</study_first_submitted>
  <study_first_submitted_qc>June 29, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 2, 2020</study_first_posted>
  <last_update_submitted>June 27, 2023</last_update_submitted>
  <last_update_submitted_qc>June 27, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 28, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Gene Therapy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fabry Disease</mesh_term>
    <mesh_term>Lysosomal Storage Diseases</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT04455230
org study id: FLT190-02
nct id: NCT04455230
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an
      abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously
      expressed throughout the body and is responsible for the breakdown of glycosphingolipids,
      deficiency of which results in the accumulation of specific glycosphingolipids that are
      associated with the pathophysiology of the disease. Current treatment for Fabry disease is
      limited to the symptomatic management of pain, conventional management of complications, and
      methods to increase the availability of functional αGLA.

      This clinical study aims to investigate the long-term safety and durability of αGLA in
      patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical
      studies.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease
primary purpose: Other
masking: None (Open Label)
measure: Safety endpoint will be assessed by the reporting of adverse events (AEs) according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
time frame: Entry to 5 years post dosing
arm group label: Participants who have received gene therapy vector (FLT190)
arm group type: Experimental
intervention type: Genetic
intervention name: FLT190
description: FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).
arm group label: Participants who have received gene therapy vector (FLT190)
criteria: 
gender: Male
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
country: Germany
country: United Kingdom
responsible party type: Sponsor
mesh term: Fabry Disease
mesh term: Lysosomal Storage Diseases
sharing ipd: No