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

Study Status: Not yet recruiting
Recruit Status: Unknown status
Study Type: Interventional

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)

Brief Summary:

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.


Detailed Summary: 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.
Sponsor: Juan Pascual

Current Primary Outcome: Change in Brain Metabolic Rate [ Time Frame: Baseline, 90 minutes, 3 months ]

Magnetic Resonance Imaging (MRI) used to calculate brain metabolic rate before oil ingestion, 90 minutes after oil ingestion, and after 3 months of daily oil ingestion


Original Primary Outcome: Same as current

Current Secondary Outcome: Change in Fasting Glucose [ Time Frame: Baseline, 3 months ]

Original Secondary Outcome: Same as current

Information By: University of Texas Southwestern Medical Center

Dates:
Date Received: December 11, 2013
Date Started: January 2012
Date Completion: March 2014
Last Updated: December 26, 2013
Last Verified: December 2013