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

February 12, 2019 updated by: Juan Pascual

Treatment Development of Triheptanoin for Glucose Transporter Type I Deficiency

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 55 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline, 6 months, 9 months
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
Baseline, 6 months, 9 months
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
baseline, 6 months, 9 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Juan Pascual

Investigators

  • Principal Investigator: Juan Pascual, MD, PhD, UT Southwestern Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2015

Primary Completion (Anticipated)

June 1, 2020

Study Completion (Anticipated)

June 1, 2020

Study Registration Dates

First Submitted

December 12, 2013

First Submitted That Met QC Criteria

December 19, 2013

First Posted (Estimate)

December 27, 2013

Study Record Updates

Last Update Posted (Actual)

February 15, 2019

Last Update Submitted That Met QC Criteria

February 12, 2019

Last Verified

February 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PASCG1D2014

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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