<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/NCT03773770</url>
  </required_header>
  <id_info>
    <org_study_id>UX007-EAP</org_study_id>
    <secondary_id>UX007G-EAP102</secondary_id>
    <nct_id>NCT03773770</nct_id>
  </id_info>
  <brief_title>Expanded Access to Triheptanoin</brief_title>
  <official_title>An Open-label Intermediate-size Treatment Protocol for the Urgent Treatment of Seriously Ill Patients With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) With Triheptanoin (UX007)</official_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>
      Expanded access may be provided for qualified patients who have limited treatment options and&#xD;
      are not eligible for a clinical trial.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS): Available through&#xD;
      Intermediate-Size Population Expanded Access in US only.&#xD;
&#xD;
      The intermediate-size expanded access treatment protocol is intended to provide rapid access&#xD;
      to triheptanoin for the treatment of seriously ill patients with Glut1 DS.&#xD;
&#xD;
      Consideration for access is for patients with previous exposure to triheptanoin.&#xD;
&#xD;
      Patients will be treated under this protocol for the duration of one year, with consideration&#xD;
      on a yearly basis for extension of treatment based on the risk-benefit ratio assessed in the&#xD;
      Treating Physician's quarterly progress reports. Patients may continue to receive&#xD;
      triheptanoin under this intermediate-size treatment protocol until commercial availability of&#xD;
      triheptanoin, should the drug receive regulatory approval for the specific disease&#xD;
      indication.&#xD;
&#xD;
      Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Non-FAOD conditions: Expanded access&#xD;
      may be available outside of the US in countries prior to approval by the local regulatory&#xD;
      agencies.&#xD;
&#xD;
      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_type_intermediate>Yes</expanded_access_type_intermediate>
  </expanded_access_info>
  <condition>Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Triheptanoin</intervention_name>
    <description>Liquid for oral (PO) or enteral tube administration</description>
    <other_name>UX007</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Criteria per Intermediate-Size Population Protocol for Glut1 DS&#xD;
&#xD;
          -  Confirmed diagnosis of Glut1 DS by documented SLC2A1 mutation or documented&#xD;
             improvement on other forms of tripheptanoin administered based on a clinical&#xD;
             presentation consistent with Glut1 DS diagnosis, including cerebrospinal fluid glucose&#xD;
             levels.&#xD;
&#xD;
          -  Patients of any age who are seriously ill and, in the Treating Physician's opinion,&#xD;
             experiencing clinical manifestations of Glut1 DS despite other management.&#xD;
&#xD;
          -  Willing and able to comply with all aspects of the treatment, including visits and&#xD;
             tests specified by the Treating Physician, documentation of symptoms and diet, and&#xD;
             administration of triheptanoin. If a minor, have a caregiver(s) willing and able to&#xD;
             assist in all applicable treatment requirements.&#xD;
&#xD;
          -  Provide written informed consent (patients aged ≥ 18 years), or provide written assent&#xD;
             (where appropriate) and have a legally authorized representative willing and able to&#xD;
             provide written informed consent, after the nature of the treatment program has been&#xD;
             explained and prior to any treatment-related procedures.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Criteria per Intermediate-Size Population Protocol for Glut1 DS&#xD;
&#xD;
          -  Patient qualifies for any other clinical trial designed to progressively evaluate the&#xD;
             safety and efficacy of tripheptanoin in Glut1 DS.&#xD;
&#xD;
          -  Any known hypersensitivity to triheptanoin that, in the judgement of the Treating&#xD;
             Physician, places the patient at an increased risk for adverse events.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <overall_official>
    <last_name>Medical Director</last_name>
    <role>Study Director</role>
    <affiliation>Ultragenyx Pharmaceutical Inc</affiliation>
  </overall_official>
  <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.dojolvi.com/</url>
    <description>Dojolvi Patient and Prescribing Information Page</description>
  </link>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>December 10, 2018</study_first_submitted>
  <study_first_submitted_qc>December 10, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 12, 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>
  <keyword>Carnitine Palmitoyltransferase Deficiency</keyword>
  <keyword>CPT I</keyword>
  <keyword>CPT II</keyword>
  <keyword>Very Long Chain acyl-CoA Dehydrogenase Deficiency</keyword>
  <keyword>VLCAD</keyword>
  <keyword>Long Chain 3-hydroxy-acyl-CoA Dehydrogenase Deficiency</keyword>
  <keyword>LHCAD</keyword>
  <keyword>Trifunctional Protein Deficiency</keyword>
  <keyword>TFP</keyword>
  <keyword>Carnitine-acylcarnitine Translocase Deficiency</keyword>
  <keyword>CACT</keyword>
  <keyword>Long Chain Fatty Acid Oxidation Disorders</keyword>
  <keyword>LC-FAOD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Carbohydrate Metabolism, Inborn Errors</mesh_term>
    <mesh_term>Syndrome</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/NCT03773770
org study id: UX007-EAP
secondary id: UX007G-EAP102
nct id: NCT03773770
lead sponsor: 
Expanded access may be provided for qualified patients who have limited treatment options and
      are not eligible for a clinical trial.
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS): Available through
      Intermediate-Size Population Expanded Access in US only.

      The intermediate-size expanded access treatment protocol is intended to provide rapid access
      to triheptanoin for the treatment of seriously ill patients with Glut1 DS.

      Consideration for access is for patients with previous exposure to triheptanoin.

      Patients will be treated under this protocol for the duration of one year, with consideration
      on a yearly basis for extension of treatment based on the risk-benefit ratio assessed in the
      Treating Physician's quarterly progress reports. Patients may continue to receive
      triheptanoin under this intermediate-size treatment protocol until commercial availability of
      triheptanoin, should the drug receive regulatory approval for the specific disease
      indication.

      Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Non-FAOD conditions: 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
expanded access type intermediate: Yes
intervention type: Drug
intervention name: Triheptanoin
description: Liquid for oral (PO) or enteral tube administration
other name: UX007
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
last name: Medical Director
role: Study Director
affiliation: Ultragenyx Pharmaceutical Inc
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.dojolvi.com/
description: Dojolvi Patient and Prescribing Information Page
responsible party type: Sponsor
mesh term: Carbohydrate Metabolism, Inborn Errors
mesh term: Syndrome