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

Treatment Development of Triheptanoin for Glucose Transporter Type I Deficiency

NCT02021526•Juan Pascual

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.

Eligibility

Age

2 - 55 years

Sex

ALL

Healthy Volunteers

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.
+1 more criteria

Locations (1)

UT Southwestern Medical Center

Dallas, Texas, United States

Key Dates

Start Date

December 1, 2015

Primary Completion Date

June 1, 2020

Completion Date

June 1, 2020

Last Updated

February 15, 2019

Conditions

Glucose Transporter Type 1 Deficiency SyndromeGlut1 Deficiency Syndrome

Interventions

Triheptanoin

DRUG

The Glucose Transporter Type I Deficiency (G1D) Registry

NCT02013583

GLUT1 Deficiency SyndromeGlucose Transporter Type 1 Deficiency Syndrome+2 more

View study

WITHDRAWNNA

Evaluation of Krio in Children and Adults With Epilepsy

NCT03202108

Ketogenic DietingEpilepsy+1 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 15, 2019Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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

Inclusion Criteria

Exclusion Criteria

The Glucose Transporter Type I Deficiency (G1D) Registry

Evaluation of Krio in Children and Adults With Epilepsy

Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)