<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/NCT03775174</url>
  </required_header>
  <id_info>
    <org_study_id>UX003-EAP</org_study_id>
    <nct_id>NCT03775174</nct_id>
  </id_info>
  <brief_title>Expanded Access to Mepsevii</brief_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ultragenyx Pharmaceutical Inc</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Ultragenyx Pharmaceutical Inc</source>
  <brief_summary>
    <textblock>
      Individual patient expanded access requests may be considered for patients who have no other&#xD;
      treatment options&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Expanded access may be available outside of the US in countries prior to approval by the&#xD;
      local regulatory agencies. For full details, please visit the links provided below.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Available</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <expanded_access_info>
    <expanded_access_type_individual>Yes</expanded_access_type_individual>
  </expanded_access_info>
  <condition>MPS VII</condition>
  <condition>Mucopolysaccharidosis VII</condition>
  <condition>Sly Syndrome</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Mepsevii</intervention_name>
    <other_name>UX003</other_name>
    <other_name>recombinant human beta-glucuronidase</other_name>
    <other_name>rhGUS</other_name>
    <other_name>vestronidase alfa</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        -&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        -&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <overall_contact>
    <last_name>Early Access</last_name>
    <phone>1-415-483-8800</phone>
    <email>EarlyAccess@ultragenyx.com</email>
  </overall_contact>
  <link>
    <url>https://www.ultragenyx.com/our-purpose/supporting-access-for-patients/?#investigational-therapies</url>
    <description>Access to Investigational Therapies / Requesting Access</description>
  </link>
  <link>
    <url>https://www.mepsevii.com/en/</url>
    <description>Mepsevii Patient and Prescribing Information Page</description>
  </link>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>December 11, 2018</study_first_submitted>
  <study_first_submitted_qc>December 11, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 13, 2018</study_first_posted>
  <last_update_submitted>September 1, 2023</last_update_submitted>
  <last_update_submitted_qc>September 1, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 6, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Expanded Access</keyword>
  <keyword>Compassionate Use</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mucopolysaccharidoses</mesh_term>
    <mesh_term>Mucopolysaccharidosis VII</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/NCT03775174
org study id: UX003-EAP
nct id: NCT03775174
lead sponsor: 
Individual patient expanded access requests may be considered for patients who have no other
      treatment options
Expanded access may be available outside of the US in countries prior to approval by the
      local regulatory agencies. For full details, please visit the links provided below.
expanded access type individual: Yes
intervention type: Drug
intervention name: Mepsevii
other name: UX003
other name: recombinant human beta-glucuronidase
other name: rhGUS
other name: vestronidase alfa
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
last name: Early Access
phone: 1-415-483-8800
email: EarlyAccess@ultragenyx.com
url: https://www.ultragenyx.com/our-purpose/supporting-access-for-patients/?#investigational-therapies
description: Access to Investigational Therapies / Requesting Access
url: https://www.mepsevii.com/en/
description: Mepsevii Patient and Prescribing Information Page
responsible party type: Sponsor
mesh term: Mucopolysaccharidoses
mesh term: Mucopolysaccharidosis VII