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

Sponsor

Juan Pascual (Other)

Overall Status

Withdrawn

CT.gov ID

NCT02021526

Collaborator

(none)

Enrollment

Location

Arms

Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

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

Study Design

Study Type:

Interventional

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 :

Dec 1, 2015

Anticipated Primary Completion Date :

Jun 1, 2020

Anticipated Study Completion Date :

Jun 1, 2020

Arms and Interventions

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

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:

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:

Heptanoate

Heptanoic Acid

Outcome Measures

Primary Outcome Measures

Change in risk for Metabolic Syndrome [Baseline, 6 months, 9 months]
Triglycerides, lipid levels, and cholesterol are measured to evaluate change in risk of metabolic syndrome
Change on Biomarkers [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 [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

Eligibility Criteria

Criteria

Ages Eligible for Study:

30 Months to 55 Years

Sexes Eligible for Study:

All

Accepts 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.
Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17.