<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/NCT03301532</url>
  </required_header>
  <id_info>
    <org_study_id>STU 102015-091</org_study_id>
    <secondary_id>R01NS094257-01A1</secondary_id>
    <nct_id>NCT03301532</nct_id>
  </id_info>
  <brief_title>Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D</brief_title>
  <official_title>Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Texas Southwestern Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Institute of Neurological Disorders and Stroke (NINDS)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>University of Texas Southwestern Medical Center</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and&#xD;
      seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a single site, open label proof of principle exploratory trial to investigate the&#xD;
      compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic&#xD;
      diet will have been previously described by the patient's treating physician independently of&#xD;
      this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from&#xD;
      fat, subjects, who are already tolerating over this much fat as part of their previously&#xD;
      prescribed ketogenic diet, will replace 45% of their daily caloric intake with the&#xD;
      triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous&#xD;
      EEG to monitor for any potential C7 related changes in seizure before, during, and after&#xD;
      triheptanoin oil ingestion.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 5, 2018</start_date>
  <completion_date type="Anticipated">July 5, 2024</completion_date>
  <primary_completion_date type="Anticipated">June 18, 2023</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Ketosis</measure>
    <time_frame>3 years</time_frame>
    <description>Changes in blood betahydroxybutyric acid level.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>EEG</measure>
    <time_frame>3 years</time_frame>
    <description>Changes in abnormal EEG discharges.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Seizure rate</measure>
    <time_frame>3 years</time_frame>
    <description>Changes in observable seizure numbers.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Glycemia</measure>
    <time_frame>3 years</time_frame>
    <description>Changes in blood glucose levels.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">10</enrollment>
  <condition>GLUT1DS1</condition>
  <arm_group>
    <arm_group_label>Patients on a ketogenic diet</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Triheptanoin</intervention_name>
    <description>Dietary supplementation with triheptanoin</description>
    <arm_group_label>Patients on a ketogenic diet</arm_group_label>
    <other_name>C7 oil</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical&#xD;
             genotyping at a CLIA-certified laboratory.&#xD;
&#xD;
          -  Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have&#xD;
             taken place for 2 months). The initiation of a ketogenic diet is previous to - and&#xD;
             thus is not part of this study.&#xD;
&#xD;
          -  Males and females 30 months to 35 years and 11 months old inclusive.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Subjects with evidence of independent, unrelated metabolic and/or genetic disease.&#xD;
&#xD;
          -  Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome,&#xD;
             crohn's disease, or colitis that could increase the subject's risk of developing&#xD;
             diarrhea or stomach pain.&#xD;
&#xD;
          -  Subjects with a BMI (body mass index) greater than or equal to 30.&#xD;
&#xD;
          -  Subjects currently not on ketogenic diet.&#xD;
&#xD;
          -  Women who are pregnant or breast feeding may not participate. Women who plan to become&#xD;
             pregnant during the course of the study, or who are unwilling to use birth control to&#xD;
             prevent pregnancy (including abstinence) may not participate. Females age 10 and over&#xD;
             will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects&#xD;
             will be asked to agree to abstinence or another form of birth control for the duration&#xD;
             of the study.&#xD;
&#xD;
          -  Allergy/sensitivity to C7&#xD;
&#xD;
          -  Previous use of triheptanoin less than 1 month prior to study initiation.&#xD;
&#xD;
          -  Treatment with medium chain triglycerides in the last 24 hours.&#xD;
&#xD;
          -  Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain&#xD;
             disorder (such as Alzheimer's disease) that would confound assessment of cognitive&#xD;
             changes, in the opinion of the investigator.&#xD;
&#xD;
          -  Active drug or alcohol use or dependence that, in the opinion of the investigator,&#xD;
             would interfere with adherence to study requirements.&#xD;
&#xD;
          -  Inability or unwillingness of subject or legal guardian/representative to give written&#xD;
             informed consent, or assent for children age 10-17,&#xD;
&#xD;
          -  Addition of a new antiseizure drug in the previous 3 months.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>30 Months</minimum_age>
    <maximum_age>35 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Juan Pascual, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Study Principal Investigator</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>UTexasSouthwestern</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75390</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2022</verification_date>
  <study_first_submitted>August 29, 2017</study_first_submitted>
  <study_first_submitted_qc>September 28, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">October 4, 2017</study_first_posted>
  <last_update_submitted>December 22, 2022</last_update_submitted>
  <last_update_submitted_qc>December 22, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 23, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>University of Texas Southwestern Medical Center</investigator_affiliation>
    <investigator_full_name>Juan Pascual</investigator_full_name>
    <investigator_title>Professor of Medicine</investigator_title>
  </responsible_party>
  <keyword>G1D, glucose, transporter</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/NCT03301532
org study id: STU 102015-091
secondary id: R01NS094257-01A1
nct id: NCT03301532
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and
      seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
This is a single site, open label proof of principle exploratory trial to investigate the
      compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic
      diet will have been previously described by the patient's treating physician independently of
      this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from
      fat, subjects, who are already tolerating over this much fat as part of their previously
      prescribed ketogenic diet, will replace 45% of their daily caloric intake with the
      triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous
      EEG to monitor for any potential C7 related changes in seizure before, during, and after
      triheptanoin oil ingestion.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Ketosis
time frame: 3 years
description: Changes in blood betahydroxybutyric acid level.
measure: EEG
time frame: 3 years
description: Changes in abnormal EEG discharges.
measure: Seizure rate
time frame: 3 years
description: Changes in observable seizure numbers.
measure: Glycemia
time frame: 3 years
description: Changes in blood glucose levels.
arm group label: Patients on a ketogenic diet
arm group type: Experimental
description: This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil.
intervention type: Drug
intervention name: Triheptanoin
description: Dietary supplementation with triheptanoin
arm group label: Patients on a ketogenic diet
other name: C7 oil
criteria: 
gender: All
minimum age: 30 Months
maximum age: 35 Years
healthy volunteers: No
last name: Juan Pascual, M.D.
role: Principal Investigator
affiliation: Study Principal Investigator
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: University of Texas Southwestern Medical Center
investigator full name: Juan Pascual
investigator title: Professor of Medicine