Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
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|ClinicalTrials.gov Identifier: NCT02018315|
Recruitment Status : Completed
First Posted : December 23, 2013
Results First Posted : October 31, 2019
Last Update Posted : October 31, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Glucose Transporter Type 1 Deficiency Syndrome GLUT1 Deficiency Syndrome||Drug: Triheptanoin||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||14 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Basic Science|
|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)|
|Study Start Date :||January 2012|
|Actual Primary Completion Date :||January 2013|
|Actual Study Completion Date :||March 2014|
Triheptanoin (C7 oil, liquid) dosed at 1 g/kg body weight divided and administered 4 times per day via mouth or g-tube for 3 months.
Triheptanoin is a 7-carbon medium chain triglyceride
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02018315
|United States, Texas|
|UT Southwestern Medical Center|
|Dallas, Texas, United States, 75390|
|Principal Investigator:||Juan M. Pascual, MD, PhD||UT Southwestern Medical Center|