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Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02021526
Recruitment Status : Withdrawn (NIH funding resulted in new clinical trial)
First Posted : December 27, 2013
Last Update Posted : February 15, 2019
Sponsor:
Information provided by (Responsible Party):
Juan Pascual, University of Texas Southwestern Medical Center

Tracking Information
First Submitted Date  ICMJE December 12, 2013
First Posted Date  ICMJE December 27, 2013
Last Update Posted Date February 15, 2019
Study Start Date  ICMJE December 2015
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 19, 2013)
  • Change in risk for Metabolic Syndrome [ Time Frame: Baseline, 6 months, 9 months ]
    Triglycerides, lipid levels, and cholesterol are measured to evaluate change in risk of metabolic syndrome
  • Change on Biomarkers [ Time Frame: Baseline, 6 months, 9 months ]
    EEG and brain metabolic rate will be measured at three time points. Changes in these biomarkers indicate the utilization of triheptanoin in brain metabolism
  • Change in Ketosis [ Time Frame: baseline, 6 months, 9 months ]
    Safety blood work (described in the first outcome measure) is measured along with ketone levels and EEG to confirm that triheptanoin is safe and does not break ketosis in patients on the ketogenic diet
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)
Official Title  ICMJE Treatment Development of Triheptanoin for Glucose Transporter Type I Deficiency
Brief Summary This is a single-site, open-label, phase II trial of C7, a food supplement or medical food, for the development of treatment outcome measures for glucose transporter type I deficiency (G1D). The primary outcome measures are: 1. Safety and tolerability of C7 as measured by changes in comprehensive blood work, including lipid and free fatty acid panels, self-reported side effects and clinical exam; 2. Changes in brain metabolic rate by MRI and EEG measurements during C7 treatment; and 3. Maintenance of ketosis in G1D patients on ketogenic diet, as measured by serial ketone levels during treatment initiation.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE
  • Glucose Transporter Type 1 Deficiency Syndrome
  • Glut1 Deficiency Syndrome
Intervention  ICMJE Drug: Triheptanoin
Triheptanoin (C7 oil) is a 7-carbon medium chain triglyceride.
Other Names:
  • C7
  • Heptanoate
  • Heptanoic Acid
Study Arms  ICMJE
  • Experimental: No Dietary Therapy
    Patients currently on no dietary therapy will receive triheptanoin (C7 oil), dosed at 1 g/kg body weight and divided into 4 doses daily, administered for 6 months
    Intervention: Drug: Triheptanoin
  • Experimental: Ketogenic Diet
    Patients on ketogenic diet will receive triheptanoin (C7 oil) in place of their usual fat intake, at a dose sufficient to maintain their ketogenic diet ratio (based on patient weight and current ratio). Patients will receive triheptanoin for 6 months.
    Intervention: Drug: Triheptanoin
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: February 12, 2019)		 0
Original Estimated Enrollment  ICMJE
 (submitted: December 19, 2013)		 50
Estimated Study Completion Date  ICMJE June 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis or suspected diagnosis of glucose transporter type I deficiency (G1D).
  • On stable ketogenic diet at a ratio between 1:2.5 and 1:4 OR Stable on no dietary therapy
  • Males and females 30 months to 55 years old, inclusive.

Exclusion Criteria:

  • Subjects with a history of life-threatening seizure episodes, including but not limited to status epilepticus and cardiac arrest.
  • Subjects with a BMI (body mass index) greater than or equal to 30 will be excluded.
  • Subjects currently on dietary therapy other than ketogenic diet (i.e., medium chain triglyceride-supplemented diets, Atkins diet, low glycemic index diet, etc.).
  • 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.
  • Allergy/sensitivity to triheptanoin.
  • Previous treatment with triheptanoin.
  • Treatment with medium chain triglycerides in the last 30 days.
  • 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.
  • Patients with metal implants, experience claustrophobia, or who are behaviorally unable to be still for MRS (magnetic resonance spectroscopy) imaging (not due to seizures) will be excluded from the imaging portion of the research.
  • Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 30 Months to 55 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02021526
Other Study ID Numbers  ICMJE PASCG1D2014
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Juan Pascual, University of Texas Southwestern Medical Center
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Juan Pascual
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Juan Pascual, MD, PhD UT Southwestern Medical Center
PRS Account University of Texas Southwestern Medical Center
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP