Glucose Transporter Type 1 Deficiency Syndrome Clinical Trial
Official title:
Clinical Trial of Citric Acid Cycle Stimulation in Energy-deficiency States: Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D) (NMTUT 2010B)
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.
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. ;
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