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Completed

NCT Number: NCT02018315

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

The purpose of this trial is to determine if an alternative energy source will impact brain metabolism in a disorder characterized by glucose metabolism failure in the brain.

The central hypothesis tested in this investigation is whether circumventing impaired glucose metabolism is feasible, safe and potentially promising by supplying anaplerotic precursors through metabolism of odd-carbon fatty acids that can enter the citric acid cycle (CAC) through alternative metabolic pathways.

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Key information

Conditions

Glucose Transporter Type 1 Deficiency Syndrome GLUT1 Deficiency Syndrome

Age range

1 month–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

About this study

Triheptanoin, a nutritional supplement long used in other metabolic disorders and also added to foods and cosmetics, will be used to complement any diet that G1D patients may be receiving at enrollment with the exception of the ketogenic diet.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or Female
  • Ages 1 month to <21 years of age
  • Diagnosed with glucose transporter type I deficiency.
  • Age matched (within 1 year) controls not diagnosed with G1D.

Exclusion criteria

  • All subjects carrying body metal implants incompatible with the exposure to a magnetic field
  • Subjects unable to tolerate the MRI and MRS procedures due to anxiety
  • Subjects receiving oxygen supplementation or those confined to a bed or stretcher
  • Subjects currently receiving a ketogenic diet, due to a high risk of seizure recurrence while transitioning off ketosis.
  • Patients behaviorally unable to hold still for imaging procedures (rather than limited by seizure activity) will be excluded.

Treatment and study plan

Triheptanoin

Drug

Triheptanoin is a 7-carbon medium chain triglyceride

Other names: C7, Heptanoate

Primary outcomes

  1. Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time

    Time frame: 1 day

    Visual analysis of EEG recording to determine the fraction of spike-range within the area of recording.

Secondary outcomes

  1. Number of Participants With Change in Brain Metabolic Rate After 3 Months

    Time frame: 3 months

    Magnetic Resonance Imaging (MRI) used to calculate brain metabolic rate. Brain metabolic rate compared before oil ingestion (Baseline), 90 minutes after oil ingestion, and after 3 months of daily oil ingestion in each participant. Triheptanoin metabolism may lead to increased oxygen consumption only while the brain undergoes a reduction of ictogenesis. We hypothesize that when ictogenesis is abolished by triheptanoin or absent at baseline, triheptanoin exerts little or no effect on CMR02.

Sponsors and collaborators

Lead sponsor

Juan Pascual

Other

Registry information

Official study title

Clinical Trial of Citric Acid Cycle Stimulation in Energy-deficiency States: Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D) (NMTUT 2010B)

Important dates

Study start
2012
Primary completion
2013
Study completion
2014
First posted
Dec 23, 2013
Registry last updated
Oct 31, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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