<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/NCT03293810</url>
  </required_header>
  <id_info>
    <org_study_id>REG-uQ-Glyb-001</org_study_id>
    <nct_id>NCT03293810</nct_id>
  </id_info>
  <brief_title>Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients</brief_title>
  <acronym>GENIALL</acronym>
  <official_title>Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>UniQure Biopharma B.V.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>UniQure Biopharma B.V.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Lipoprotein lipase deficiency (LPLD) is a rare autosomal recessive disorder, characterized by&#xD;
      loss-of function mutations in the LPL gene, leading to the inability to produce functionally&#xD;
      active lipoprotein lipase (LPL). LPL is the key enzyme in the metabolism of triglyceride&#xD;
      (TG)-rich lipoproteins (chylomicrons (CM) and very low-density lipoproteins (VLDL)). LPLD&#xD;
      results in extremely high concentrations of circulating TG-rich lipoproteins.&#xD;
&#xD;
      No drug therapy for LPLD is currently available. Clinical management of LPLD patients&#xD;
      consists of severe dietary fat restriction and the use of medium-chain triglycerides to&#xD;
      substitute for normal dietary fats.&#xD;
&#xD;
      Alipogene tiparvovec (Glybera®) received marketing authorisation from the European commission&#xD;
      on 25 October 2012. Glybera® aims to correct lipoprotein lipase deficiency sufficiently to&#xD;
      decrease the morbidity and lower the risk of inherent complications of LPLD, in adult&#xD;
      patients genetically diagnosed with LPLD.&#xD;
&#xD;
      The Glybera Registry is designed to collect the long-term safety and efficacy data of&#xD;
      GLYBERA®&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      All patients treated with GLYBERA®, in a clinical trial and when GLYBERA® was commercially&#xD;
      available who are currently participating in the LPLD Registry (Long term follow up of safety&#xD;
      and efficacy in LPLD), will be asked to continue their participation in this Glybera Registry&#xD;
      to collect long-term safety and efficacy data.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 27, 2014</start_date>
  <completion_date type="Anticipated">October 2033</completion_date>
  <primary_completion_date type="Anticipated">September 2033</primary_completion_date>
  <study_type>Observational [Patient Registry]</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Other</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <target_duration>15 Years</target_duration>
  <primary_outcome>
    <measure>Long-term collection of Safety and Efficacy of GLYBERA®, as measured by collection of Adverse Events, Immunological responses and information on Pancreatitis-events</measure>
    <time_frame>15 years</time_frame>
    <description>Adverse Events will be collected as reported by the patients during routine visits/contacts.&#xD;
Immunological responses defined as antibody formation and T-cell responses against the AAV1-capsid and against the LPLS447X transgene product, measured just before dosing and at 6 and 12 months post-dosing.&#xD;
Pancreatitis-events will be collected as reported by the patients during routine visits/contacts</description>
  </primary_outcome>
  <enrollment type="Actual">16</enrollment>
  <condition>Lipoprotein Lipase Deficiency</condition>
  <condition>Familial Hyperlipoproteinemia Type 1</condition>
  <condition>Familial Hyperchylomicronemia</condition>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Observational study</intervention_name>
    <description>Post-Authorization Safety Study</description>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Patients treated with GLYBERA®, either during their participation in a clinical trial or in&#xD;
        the commercial setting till October 25th, 2017&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  All patients treated with GLYBERA®, either during their participation in a clinical&#xD;
             trial or in the commercial setting till October 25th, 2017 (= expiration date of&#xD;
             Marketing Authorization of GLYBERA®), and&#xD;
&#xD;
          -  Who are currently participating in the LPLD Registry&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  None&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Maurizio Averna, Prof</last_name>
    <role>Study Chair</role>
    <affiliation>Universitaria Policlinico Paolo Giaccone, Palermo</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin</name>
      <address>
        <city>Berlin</city>
        <zip>13353</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Germany</country>
  </location_countries>
  <verification_date>May 2022</verification_date>
  <study_first_submitted>May 20, 2015</study_first_submitted>
  <study_first_submitted_qc>September 20, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 26, 2017</study_first_posted>
  <last_update_submitted>May 9, 2022</last_update_submitted>
  <last_update_submitted_qc>May 9, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">May 10, 2022</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>Hyperlipoproteinemia Type I</mesh_term>
    <mesh_term>Hyperlipoproteinemias</mesh_term>
    <mesh_term>Hyperlipidemias</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</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/NCT03293810
org study id: REG-uQ-Glyb-001
nct id: NCT03293810
lead sponsor: 
has dmc: No
Lipoprotein lipase deficiency (LPLD) is a rare autosomal recessive disorder, characterized by
      loss-of function mutations in the LPL gene, leading to the inability to produce functionally
      active lipoprotein lipase (LPL). LPL is the key enzyme in the metabolism of triglyceride
      (TG)-rich lipoproteins (chylomicrons (CM) and very low-density lipoproteins (VLDL)). LPLD
      results in extremely high concentrations of circulating TG-rich lipoproteins.

      No drug therapy for LPLD is currently available. Clinical management of LPLD patients
      consists of severe dietary fat restriction and the use of medium-chain triglycerides to
      substitute for normal dietary fats.

      Alipogene tiparvovec (Glybera®) received marketing authorisation from the European commission
      on 25 October 2012. Glybera® aims to correct lipoprotein lipase deficiency sufficiently to
      decrease the morbidity and lower the risk of inherent complications of LPLD, in adult
      patients genetically diagnosed with LPLD.

      The Glybera Registry is designed to collect the long-term safety and efficacy data of
      GLYBERA®
All patients treated with GLYBERA®, in a clinical trial and when GLYBERA® was commercially
      available who are currently participating in the LPLD Registry (Long term follow up of safety
      and efficacy in LPLD), will be asked to continue their participation in this Glybera Registry
      to collect long-term safety and efficacy data.
observational model: Other
time perspective: Prospective
measure: Long-term collection of Safety and Efficacy of GLYBERA®, as measured by collection of Adverse Events, Immunological responses and information on Pancreatitis-events
time frame: 15 years
description: Adverse Events will be collected as reported by the patients during routine visits/contacts.
Immunological responses defined as antibody formation and T-cell responses against the AAV1-capsid and against the LPLS447X transgene product, measured just before dosing and at 6 and 12 months post-dosing.
Pancreatitis-events will be collected as reported by the patients during routine visits/contacts
intervention type: Other
intervention name: Observational study
description: Post-Authorization Safety Study
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Maurizio Averna, Prof
role: Study Chair
affiliation: Universitaria Policlinico Paolo Giaccone, Palermo
facility: 
country: Germany
responsible party type: Sponsor
mesh term: Hyperlipoproteinemia Type I
mesh term: Hyperlipoproteinemias
mesh term: Hyperlipidemias
sharing ipd: Undecided