<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/NCT01710813</url>
  </required_header>
  <id_info>
    <org_study_id>AGLU06909</org_study_id>
    <secondary_id>LTS13930</secondary_id>
    <nct_id>NCT01710813</nct_id>
  </id_info>
  <brief_title>Alglucosidase Alfa Pompe Safety Sub-Registry</brief_title>
  <official_title>A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Genzyme, a Sanofi Company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sanofi</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      To collect uniform and meaningful data on patients with Pompe disease who experience&#xD;
      anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic&#xD;
      immune complex-mediated reactions following treatment with alglucosidase alfa.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">March 20, 2015</start_date>
  <completion_date type="Actual">April 20, 2021</completion_date>
  <primary_completion_date type="Actual">April 20, 2021</primary_completion_date>
  <study_type>Observational [Patient Registry]</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <target_duration>4 Years</target_duration>
  <primary_outcome>
    <measure>number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions</measure>
    <time_frame>4 Years</time_frame>
    <description>collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">110</enrollment>
  <condition>Pompe Disease</condition>
  <arm_group>
    <arm_group_label>pompe safety sub-registry</arm_group_label>
    <description>patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>alglucosidase alfa</intervention_name>
    <description>Alglucosidase alfa IV infusion of 20 mg/kg; qow</description>
    <arm_group_label>pompe safety sub-registry</arm_group_label>
    <other_name>Myozyme; Lumizyme</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Patients who are enrolled in the Pompe Registry will be enrolled in this sub-registry&#xD;
        include patients with infantile-onset Pompe disease, as well as those with late-onset Pompe&#xD;
        disease.&#xD;
&#xD;
        An approximately equal proportion of currently treated and treatment-naïve patients is&#xD;
        targeted for enrollment at each site.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  The patient must be enrolled in the Pompe Registry;&#xD;
&#xD;
          -  Provide a signed patient information and authorization form;&#xD;
&#xD;
          -  Have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as&#xD;
             documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA&#xD;
             gene mutations);&#xD;
&#xD;
          -  Be naïve to and plan to be treated with alglucosidase alfa at or prior to enrollment,&#xD;
             or are being treated with alglucosidase alfa.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients will be excluded if they have received an investigational drug (excluding&#xD;
             alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient&#xD;
             Information and Authorization form, or if they are taking or plan to take any&#xD;
             investigational product while enrolled in the Safety Sub-Registry.&#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>Medical Monitor</last_name>
    <role>Study Director</role>
    <affiliation>Genzyme, a Sanofi Company</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Investigational Site Number 840016</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85013</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840002</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840004</name>
      <address>
        <city>Hershey</city>
        <state>Pennsylvania</state>
        <zip>17033</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840014</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15213</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840008</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84113</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840001</name>
      <address>
        <city>Fairfax</city>
        <state>Virginia</state>
        <zip>22030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 056001</name>
      <address>
        <city>Gent</city>
        <zip>9000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 056002</name>
      <address>
        <city>Leuven</city>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 056003</name>
      <address>
        <city>Leuven</city>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 203001</name>
      <address>
        <city>Praha 2</city>
        <zip>12808</zip>
        <country>Czechia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 276002</name>
      <address>
        <city>Halle (Saale)</city>
        <zip>06120</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 380001</name>
      <address>
        <city>Brescia</city>
        <zip>25123</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 380002</name>
      <address>
        <city>Cagliari</city>
        <zip>09126</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 380006</name>
      <address>
        <city>Messina</city>
        <zip>98125</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 380005</name>
      <address>
        <city>Milano</city>
        <zip>20133</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 380004</name>
      <address>
        <city>Padova</city>
        <zip>35128</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 380003</name>
      <address>
        <city>Roma</city>
        <zip>00168</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 158001</name>
      <address>
        <city>Taipei</city>
        <zip>10043</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Czechia</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Taiwan</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Czech Republic</country>
  </removed_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>October 17, 2012</study_first_submitted>
  <study_first_submitted_qc>October 17, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 19, 2012</study_first_posted>
  <last_update_submitted>July 12, 2021</last_update_submitted>
  <last_update_submitted_qc>July 12, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 13, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>pompe disease</keyword>
  <keyword>alglucosidase alfa</keyword>
  <keyword>anaphylaxis</keyword>
  <keyword>severe cutaneous</keyword>
  <keyword>systemic immune complex-mediated reactions</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Glycogen Storage Disease Type II</mesh_term>
  </condition_browse>
  <!-- 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/NCT01710813
org study id: AGLU06909
secondary id: LTS13930
nct id: NCT01710813
lead sponsor: 
has dmc: Yes
To collect uniform and meaningful data on patients with Pompe disease who experience
      anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic
      immune complex-mediated reactions following treatment with alglucosidase alfa.
observational model: Cohort
time perspective: Prospective
measure: number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
time frame: 4 Years
description: collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa
arm group label: pompe safety sub-registry
description: patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry
intervention type: Biological
intervention name: alglucosidase alfa
description: Alglucosidase alfa IV infusion of 20 mg/kg; qow
arm group label: pompe safety sub-registry
other name: Myozyme; Lumizyme
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Medical Monitor
role: Study Director
affiliation: Genzyme, a Sanofi Company
facility: 
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country: Belgium
country: Czechia
country: Germany
country: Italy
country: Taiwan
country: United States
country: Czech Republic
responsible party type: Sponsor
mesh term: Glycogen Storage Disease Type II