<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/NCT03768817</url>
  </required_header>
  <id_info>
    <org_study_id>UX007-CL003</org_study_id>
    <nct_id>NCT03768817</nct_id>
  </id_info>
  <brief_title>Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program</brief_title>
  <official_title>A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ultragenyx Pharmaceutical Inc</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Ultragenyx Pharmaceutical Inc</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The primary objective of the study is to evaluate the clinical outcomes for the trigger event&#xD;
      of patients who receive triheptanoin in the emergency Investigational New Drug (eIND)&#xD;
      program.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The study is a retrospective chart review of patients in the USA with LC-FAOD who received&#xD;
      triheptanoin via eIND request from their metabolic or treating physician before 01 September&#xD;
      2018. Participating study sites will provide the medical records of subjects who meet&#xD;
      eligibility criteria, and relevant data regarding the trigger event (the reason the patient&#xD;
      was placed on triheptanoin) and key details about hospitalizations during the study period&#xD;
      will be extracted.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 30, 2019</start_date>
  <completion_date type="Actual">June 8, 2020</completion_date>
  <primary_completion_date type="Actual">June 8, 2020</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Duration of Hospitalization for Trigger Event</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Disposition on Discharge of Trigger Event</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Duration of Important Interventions for Trigger Event</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Duration of Hospitalization for Other Major Clinical Events</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Disposition on Discharge of Other Major Clinical Events</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Important Medical Interventions for Other Major Clinical Events</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events</measure>
    <time_frame>Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment</time_frame>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">20</enrollment>
  <condition>Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)</condition>
  <arm_group>
    <arm_group_label>Patients treated with triheptanoin</arm_group_label>
    <description>Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>No Intervention</intervention_name>
    <description>This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.</description>
    <arm_group_label>Patients treated with triheptanoin</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Medical records of patients in the USA with LC-FAOD who received triheptanoin via eIND&#xD;
        request from their metabolic or treating physician before 01 September 2018.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male or female patients of any age with confirmed diagnosis of one of the LC-FAOD&#xD;
             disorders including: mitochondrial trifunctional protein (TFP) deficiency, carnitine&#xD;
             palmitoyltransferase deficiencies (CPT I and CPT II), very long chain acyl-CoA&#xD;
             dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxy-acyl-CoA dehydrogenase (LCHAD)&#xD;
             deficiency, and carnitine acylcarnitine translocase (CATR) deficiency&#xD;
&#xD;
          -  Treatment initiation with triheptanoin was via eIND before 01 September 2018&#xD;
&#xD;
          -  Treated with triheptanoin in the USA&#xD;
&#xD;
          -  Willing and able to provide written, signed informed consent, or where appropriate for&#xD;
             subjects under the age of 18, or provide written assent and written informed consent&#xD;
             by a legally authorized representative after the nature of the study has been&#xD;
             explained and prior to any research-related procedures. To obtain and review medical&#xD;
             records of deceased individuals, informed consent from next of kin or appropriate&#xD;
             legal entity will be obtained, as applicable.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Unwilling to sign informed consent or assent to release of medical records&#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>
  <location>
    <facility>
      <name>Children's Hospital of Los Angeles</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Orange County</name>
      <address>
        <city>Orange</city>
        <state>California</state>
        <zip>92868</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Yale University</name>
      <address>
        <city>New Haven</city>
        <state>Connecticut</state>
        <zip>06511</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's National Hospital</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of South Florida</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33606</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Emory University</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Minnesota Masonic Children's Hospital</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Duke University</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Health and Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UPMC Children's Hospital of Pittsburgh</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15224</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2020</verification_date>
  <study_first_submitted>December 5, 2018</study_first_submitted>
  <study_first_submitted_qc>December 5, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 7, 2018</study_first_posted>
  <last_update_submitted>August 26, 2020</last_update_submitted>
  <last_update_submitted_qc>August 26, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 28, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)</keyword>
  <keyword>Triheptanoin</keyword>
  <!-- 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/NCT03768817
org study id: UX007-CL003
nct id: NCT03768817
lead sponsor: 
has dmc: No
The primary objective of the study is to evaluate the clinical outcomes for the trigger event
      of patients who receive triheptanoin in the emergency Investigational New Drug (eIND)
      program.
The study is a retrospective chart review of patients in the USA with LC-FAOD who received
      triheptanoin via eIND request from their metabolic or treating physician before 01 September
      2018. Participating study sites will provide the medical records of subjects who meet
      eligibility criteria, and relevant data regarding the trigger event (the reason the patient
      was placed on triheptanoin) and key details about hospitalizations during the study period
      will be extracted.
observational model: Case-Only
time perspective: Retrospective
measure: Duration of Hospitalization for Trigger Event
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Disposition on Discharge of Trigger Event
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Duration of Important Interventions for Trigger Event
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Duration of Hospitalization for Other Major Clinical Events
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Disposition on Discharge of Other Major Clinical Events
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Number of Important Medical Interventions for Other Major Clinical Events
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
measure: Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events
time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
arm group label: Patients treated with triheptanoin
description: Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND
intervention type: Other
intervention name: No Intervention
description: This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.
arm group label: Patients treated with triheptanoin
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
last name: Medical Director
role: Study Director
affiliation: Ultragenyx Pharmaceutical Inc
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country: United States
responsible party type: Sponsor