<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/NCT02221362</url>
  </required_header>
  <id_info>
    <org_study_id>701-901</org_study_id>
    <nct_id>NCT02221362</nct_id>
  </id_info>
  <brief_title>A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease</brief_title>
  <official_title>A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>BioMarin Pharmaceutical</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>BioMarin Pharmaceutical</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational&#xD;
      study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease&#xD;
      prospectively collects data to understand clinical progression in terms of respiratory&#xD;
      function, symptomology, genotype, biochemistry, endurance and selected subject-reported&#xD;
      measures for 24 weeks followed by a 240 week additional observation period for up to 100&#xD;
      subjects.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <start_date>December 2014</start_date>
  <completion_date type="Actual">September 2016</completion_date>
  <primary_completion_date type="Actual">September 2016</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Control</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease</measure>
    <time_frame>264 Weeks</time_frame>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">30</enrollment>
  <condition>Late-onset Pompe Patients Untreated or Treated With rhGAA</condition>
  <arm_group>
    <arm_group_label>Observational</arm_group_label>
    <description>No interventions</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Blood and urine samples to be collected for exploratory research, Genetic/Genomic testing is&#xD;
      optional requiring specific consent&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Late-onset Pompe Disease patients untreated or treated with rhGAA&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Willing and able to provide written informed consent after the nature of the study has&#xD;
             been explained and prior to any study-related procedure&#xD;
&#xD;
          -  Diagnosed with late-onset Pompe disease based on current or previous genomic testing&#xD;
             and/or endogenous GAA activity&#xD;
&#xD;
          -  At least 18 years of age at study entry&#xD;
&#xD;
          -  Willing and able to comply with all study procedures&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Requires non-invasive ventilatory support while awake and in the upright position&#xD;
&#xD;
          -  Concurrent disease, medical condition, or extenuating circumstance that, in the&#xD;
             opinion of the investigator, might compromise study completion or data collection&#xD;
&#xD;
          -  Unable to perform baseline efficacy assessments&#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>Medical Monitor, MD</last_name>
    <role>Study Director</role>
    <affiliation>BioMarin Pharmaceutical</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Westmead Hospital, Dept. of Genetic Medicine</name>
      <address>
        <city>Westmead</city>
        <state>New South Wales</state>
        <zip>2145</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Women's and Children's Hospital</name>
      <address>
        <city>Adelaide</city>
        <state>South Australia</state>
        <zip>5006</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Monash Medical Centre</name>
      <address>
        <city>Clayton</city>
        <state>Victoria</state>
        <zip>3168</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Porto Alegre</city>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Recife</city>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Ribeirao Preto</city>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Alberta Children's Hospital</name>
      <address>
        <city>Calgary</city>
        <state>Alberta</state>
        <zip>T3B 6A8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Alexandroupolis</city>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Athens</city>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Larissa</city>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Thessalonika</city>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St. Vincent's University Hospital</name>
      <address>
        <city>Dublin</city>
        <country>Ireland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seoul National University Hospital</name>
      <address>
        <city>Seoul</city>
        <zip>110-744</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Copernicus, St. Adalbert Hospital</name>
      <address>
        <city>Gdansk</city>
        <zip>80-462</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Diamond Clinic</name>
      <address>
        <city>Krakow</city>
        <zip>31-011</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Insitute of Psychiatry and Neurology</name>
      <address>
        <city>Warsaw</city>
        <zip>02-957</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Spitalul Clinic Judetean de Urgenta &quot;Sf. Apostol Andrei&quot;</name>
      <address>
        <city>Constanta</city>
        <zip>900951</zip>
        <country>Romania</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Belgrade</city>
        <country>Serbia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>National University Hospital</name>
      <address>
        <city>Singapore</city>
        <zip>119074</zip>
        <country>Singapore</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Ljubljana</city>
        <country>Slovenia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital de la Santa Creu I Sant Pau</name>
      <address>
        <city>Barcelona</city>
        <zip>08041</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital General Universitario Gregorio Maranon</name>
      <address>
        <city>Madrid</city>
        <zip>28007</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitari i Politecnic La Fe</name>
      <address>
        <city>Valencia</city>
        <zip>46026</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mackay Memorial Hospital</name>
      <address>
        <city>Taipei</city>
        <zip>10449</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Brazil</country>
    <country>Canada</country>
    <country>Greece</country>
    <country>Ireland</country>
    <country>Korea, Republic of</country>
    <country>Poland</country>
    <country>Romania</country>
    <country>Serbia</country>
    <country>Singapore</country>
    <country>Slovenia</country>
    <country>Spain</country>
    <country>Taiwan</country>
  </location_countries>
  <removed_countries>
    <country>Austria</country>
    <country>Turkey</country>
  </removed_countries>
  <verification_date>September 2015</verification_date>
  <study_first_submitted>August 6, 2014</study_first_submitted>
  <study_first_submitted_qc>August 19, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 20, 2014</study_first_posted>
  <last_update_submitted>October 27, 2016</last_update_submitted>
  <last_update_submitted_qc>October 27, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">October 31, 2016</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>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/NCT02221362
org study id: 701-901
nct id: NCT02221362
lead sponsor: 
has dmc: No
Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational
      study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease
      prospectively collects data to understand clinical progression in terms of respiratory
      function, symptomology, genotype, biochemistry, endurance and selected subject-reported
      measures for 24 weeks followed by a 240 week additional observation period for up to 100
      subjects.
observational model: Case-Control
time perspective: Prospective
measure: To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
time frame: 264 Weeks
arm group label: Observational
description: No interventions
Blood and urine samples to be collected for exploratory research, Genetic/Genomic testing is
      optional requiring specific consent
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Medical Monitor, MD
role: Study Director
affiliation: BioMarin Pharmaceutical
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country: Australia
country: Brazil
country: Canada
country: Greece
country: Ireland
country: Korea, Republic of
country: Poland
country: Romania
country: Serbia
country: Singapore
country: Slovenia
country: Spain
country: Taiwan
country: Austria
country: Turkey
responsible party type: Sponsor
mesh term: Glycogen Storage Disease Type II