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Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

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ClinicalTrials.gov Identifier: NCT03301532
Recruitment Status : Recruiting
First Posted : October 4, 2017
Last Update Posted : December 19, 2020
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Juan Pascual, University of Texas Southwestern Medical Center

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
GLUT1DS1 Drug: Triheptanoin Phase 2

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency
Actual Study Start Date : June 5, 2018
Estimated Primary Completion Date : July 5, 2021
Estimated Study Completion Date : July 5, 2023


Arm Intervention/treatment
Experimental: Patients on a ketogenic diet
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.
Drug: Triheptanoin
Dietary supplementation with triheptanoin
Other Name: C7 oil




Primary Outcome Measures :
  1. Ketosis [ Time Frame: 3 years ]
    Changes in blood betahydroxybutyric acid level.


Secondary Outcome Measures :
  1. EEG [ Time Frame: 3 years ]
    Changes in abnormal EEG discharges.

  2. Seizure rate [ Time Frame: 3 years ]
    Changes in observable seizure numbers.

  3. Glycemia [ Time Frame: 3 years ]
    Changes in blood glucose levels.



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Ages Eligible for Study:   30 Months to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory.
  • Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have taken place for 2 months). The initiation of a ketogenic diet is previous to - and thus is not part of this study.
  • Males and females 30 months to 35 years and 11 months old inclusive.

Exclusion Criteria:

  • Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
  • Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, crohn's disease, or colitis that could increase the subject's risk of developing diarrhea or stomach pain.
  • Subjects with a BMI (body mass index) greater than or equal to 30.
  • Subjects currently not on ketogenic diet.
  • Women who are pregnant or breast feeding may not participate. Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate. Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects will be asked to agree to abstinence or another form of birth control for the duration of the study.
  • Allergy/sensitivity to C7
  • Previous use of triheptanoin less than 1 month prior to study initiation.
  • Treatment with medium chain triglycerides in the last 24 hours.
  • Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain disorder (such as Alzheimer's disease) that would confound assessment of cognitive changes, in the opinion of the investigator.
  • Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements.
  • Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17,
  • Addition of a new antiseizure drug in the previous 3 months.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03301532


Contacts
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Contact: Adrian Avila, BS 214-648-4591 adrian.avila@utsouthwestern.edu
Contact: Tricia Plumb, R.N. 214-456-2464 patricia.plumb@utsouthwestern.edu

Locations
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United States, Texas
UTexasSouthwestern Recruiting
Dallas, Texas, United States, 75390
Contact: Adrian Avila, B.S.    214-648-4591    adrian.avila@utsouthwestern.edu   
Contact: Juan M Pascual, M.D.    214-648-3562    juan.pascual@utsouthwestern.edu   
Principal Investigator: Juan M Pascual, M.D.         
Sponsors and Collaborators
University of Texas Southwestern Medical Center
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
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Principal Investigator: Juan Pascual, M.D. Study Principal Investigator
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Responsible Party: Juan Pascual, Professor of Medicine, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT03301532    
Other Study ID Numbers: STU 102015-091
R01NS094257-01A1 ( U.S. NIH Grant/Contract )
First Posted: October 4, 2017    Key Record Dates
Last Update Posted: December 19, 2020
Last Verified: December 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Juan Pascual, University of Texas Southwestern Medical Center:
G1D, glucose, transporter