NCT02018315

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Juan Pascual · Academic / Other
• Sex: All
• Age: 1 Month – 20 Years
• Healthy volunteers: Accepted

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 description

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.

Conditions

• Glucose Transporter Type 1 Deficiency Syndrome
• GLUT1 Deficiency Syndrome

Interventions

Type	Name	Description
DRUG	Triheptanoin	Triheptanoin is a 7-carbon medium chain triglyceride

Timeline

• Start date: 2012-01-01
• Primary completion: 2013-01-01
• Completion: 2014-03-01
• First posted: 2013-12-23
• Last updated: 2019-10-31
• Results posted: 2019-10-31

Locations

1 site across 1 country: United States