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

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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Recruitment Status : Withdrawn (NIH funding resulted in new clinical trial)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

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.

Condition or disease Intervention/treatment Phase
Glucose Transporter Type 1 Deficiency Syndrome Glut1 Deficiency Syndrome Drug: Triheptanoin Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: Treatment Development of Triheptanoin for Glucose Transporter Type I Deficiency
Study Start Date : December 2015
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
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
Drug: Triheptanoin
Triheptanoin (C7 oil) is a 7-carbon medium chain triglyceride.
Other Names:
  • C7
  • Heptanoate
  • Heptanoic Acid

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.
Drug: Triheptanoin
Triheptanoin (C7 oil) is a 7-carbon medium chain triglyceride.
Other Names:
  • C7
  • Heptanoate
  • Heptanoic Acid




Primary Outcome Measures :
  1. 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

  2. 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

  3. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   30 Months to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT02021526


Locations
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United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Juan Pascual
Investigators
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Principal Investigator: Juan Pascual, MD, PhD UT Southwestern Medical Center

Additional Information:
Publications:

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Responsible Party: Juan Pascual, Associate Professor, Director of the Rare Brain Disorders Program, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT02021526     History of Changes
Other Study ID Numbers: PASCG1D2014
First Posted: December 27, 2013    Key Record Dates
Last Update Posted: February 15, 2019
Last Verified: February 2019
Keywords provided by Juan Pascual, University of Texas Southwestern Medical Center:
G1D
Glut1 Deficiency
Glucose Transporter Type 1 Deficiency
Glucose Transporter Type I Deficiency
Additional relevant MeSH terms:
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Syndrome
Disease
Pathologic Processes